Serving managed care, health-system and specialty decision makers Volume 10 • Number 4 • July/August 2021 • specialtypharmacycontinuum.com
UP FRONT
Rx delays, financial burdens cited in AMA survey
Connect with SPC online......3
Prior Authorization Ills: No Rest During COVID
OPERATIONS & MGMT Top 11 triggers for Medicare audits ............. 4 The per-patient cost of CAR T-cell Rx tops $2 million ....................... 7
As payors push patients out of hospitals …
It’s Prime Time For Ambulatory Infusion Centers
T
he time is ripe for ambulatory infusion centers (AICs) to gain a stronger foothold in U.S. health care, a panel of experts said during the MHA 2021 Business Summit, held virtually. “There’s a lot of tailwinds in this industry,” said Reece Norris, JD, a cofounder and the CEO of WeInfuse, an infusion software and consulting company. Payors and health plans are looking for sites of care (SOC) for infusion therapies that are more cost-effective than hospitals without compromising
Medicare loses billions when generic drug use falls short ................................ 14
Continued on page 9
POLICY Manufacturers and payors: finding a safe harbor for compliance .....................18 Some providers, payors push back on Alzheimer’s Rx ............ 20
REVIEW ARTICLE
The New Frontier Of Hemophilia Treatment See page 22.
Amid challenges … A
N
Health Systems Still Fighting for Specialty Access
ine of 10 physicians reported d that prior authorizations (PAs) s s) have had a negative effect on clinicall outcomes their t ffor th i patients, tii t and d nearly l one of three (30%) reported that PAs have led to a serious adverse event for a patient in their care, according to a survey from the American Medical Association (AMA). Almost four of five respondents (79%) said patients abandon treatment due to struggles with their health insurers over treatment authorization. In the early days of the COVID-19 pandemic, such hassles eased because many payors temporarily relaxed their PA requirements “to reduce administrative burdens and support rapid patient access to needed drugs, tests and treatments,” said AMA President Susan R. Bailey, MD, in a statement accompanying the release of the survey. By the end of 2020, however, as COVID-19 cases surged, “the AMA found that most physicians were facing strict authorization hurdles that delayed patients’ access to needed care.” Payors and pharmacy benefit managers (PBMs) counter that they have, in fact, sought to alleviate burdens imposed by PA requirements during COVID-19, with adjustments made throughout the pandemic. “Early in the COVID-19 pandemic, Prime Therapeutics removed all medication refill restrictions for our approximately
ealth systems continue to make significant inroads into the specialty pharmacy market, with more than two-thirds of multihospital systems launching programs in a recent fiveyear period. But despite those gains, the sector still faces major roadblocks, including a lack of access to limited distribution drugs (LDDs) and payor contracts, according to ASHP’s inaugural National Survey of Health-System Specialty Pharmacy Practice. Still, many health systems have succeeded in overcoming those challenges,
Continued on page 16
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Specialty Pharmacy Continuum • July/August 2021
UP FRONT
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Volume 10 • Number 4 • July/August 2021
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Specialty Pharmacy Continuum • July/August 2021
OPERATIONS & MANAGEMENT
Medicare Part D Audits: Top 11 Areas for Scrutiny Medicare Part D pharmacy audits are on the rise, and pharmacists would be wise to adapt their business practices and know how to respond, an audit expert said at the virtual MHA 2021 Business Summit. “Prior to COVID-19, we’ve seen a nearly 80% increase in audits that pharmacies experience,” said Trenton Thiede, PharmD, MBA, the president of PAAS National, a pharmacy audit assistance company. These have primarily been from pharmacy benefit managers (PBMs) but also by plan sponsors and the Centers for Medicare & Medicaid Services, Dr. Thiede noted. The increase has occurred partly as a result of escalating health care costs and the opioid epidemic, as well as instances of fraud, waste and abuse, he said. There’s been a shift from on-site to more quick turnaround desk audits that try to validate quick outliers, such as high-dollar items or incorrect days’ supply. Nearly one-fourth of desk audits seen by his company now are for these prescription validation requests, he said. Over the past year, due to COVID-19, many PBMs have conducted virtual audits. These take elements from both on-site and desk audits, Dr. Thiede said, asking compliance questions, requiring photos of the pharmacy area and copies of licenses, as well as requesting copies of prescriptions and signature logs. Pharmacy owners who don’t perform well on audits face risking their reputation, license, fines and even imprisonment, he said. Financial recoveries are common, but his firm is seeing more and more network terminations due to poor compliance or bad actors. Overall, pharmacies need the following items to perform well on audits: prescriptions that are legal and valid per state and federal laws, proof of filling and billing accurately, proof of dispensing, proof of copay collection, and documents to prove enough inventory was purchased from an appropriate source. Common audit discrepancies can occur over items such as missing, invalid or altered prescriptions; unauthorized refills; refilling medication too soon;
incorrect dispense-as-written (DAW) codes; missing or invalid signature logs; and issues delivering medication greater than 10 days after the date of fill, Dr. Thiede said. Dr. Thiede presented the top 11 audit discrepancy areas noted by his firm, and advised how to prepare for them. Several relate to days’ supply, the estimated number of days the prescription will last excluding refills, based upon the prescribed quantity and directions: Days’ supply—insulin. Some prescriptions may require breaking open boxes of insulin pens to distribute to different patients, especially if a patient may need just one per month. But, FDA labeling updates from November 2019 and continued agency guidance from October 2020 (bit.ly/3BrZ0vz) compel health care professionals to dispense the pens to a single patient in the original sealed carton. Pharmacies should submit an accurate days’ supply where possible, or call a payor’s help desk to request an override. If you do break open boxes, employ safety measures such as tamper-indicator tape, label each pen for individual patient use and provide a copy of the patient instructions for use. In addition, wait until approximately 90% of the medication is used before refilling, and put the days’ supply into the patient’s instructions for use on the prescription label to alert the patient and internal pharmacy staff not to refill early. Days’ supply—topicals. This is a challenge because there may not be calculable instructions for use, and it’s important to submit an accurate days’ supply, if possible. Pharmacists could consider grams per application or maximum daily dose to determine how much is used each day. The fingertip unit (FTU) method that measures 1 FTU as equivalent to 0.5 g also can help calculate days’ supply. Days’ supply—inhalers. Submit D aan accurate days’ supply, if possible, and an do not refill these early. Pharmacists can ask for a days’ supply overcist ride, if needed, and add a note into ri tthe patient’s instructions for use on the prescription label (e.g., 60 days’ supply). If a patient requests a refill early, assess the circumstances and document everything before dispensing. b Days’ supply—eye drops. Again, submit an accurate days’ ssupply, if possible. Generally, pharmacists can estimate 20 drops/mL for
solution or 15 drops/mL for suspension, although some PBMs use their own estimates. Document any confounding factors that would cause patients to use more. DAW codes. Always start with a default of 0 (no product selection indicated) unless there is other supporting documentation to use a different code. If you use other codes, stick to 1 (substitution not allowed by prescriber), 2 (substitution allowed; patient requested product dispensed) or 9 (substitution allowed by prescriber but plan requests brand). Controlled substances. Have all necessary elements in place for federal law (e.g., patient address, physician address and DEA number), as well as whatever you need for state laws. For clinics that use buprenorphine for opioid addiction, PBMs will look to see that the X DEA number is listed on appropriate prescriptions. (This number, or “DATAwaived” registration, allows providers to avoid Drug Enforcement Administration registration requirements for narcotic treatment programs.) Electronic prescriptions. These have their own pitfalls, including incomplete fields for quantity, where it may say “1” but not spell out the package size, or doesn’t provide a unit of measure. It’s important for pharmacists to clarify that. Ensure that the DAW field and days’ supply are accurate, and that you don’t accept invalid prescriptions. Transfer prescriptions. Ensure that you meet all of the requirements necessary in your state, including the first and last dates of fill, the prescription number, the original and remaining refills, and the date of transfer. Using a dedicated transfer prescription pad with all required elements helps with compliance. Compounds. Ensure that your compounding log matches the claim that you submitted, and what you actually used to make the compound. The three items to watch are what you billed, what you filled and what your compounding logs showed. Over time, pharmacies may not update their compounding or a product National Drug Code number—that’s an easy recoupment for a PBM if you get audited. Also confirm that your dispensing software does not overbill a quantity, and do not overstate your level of effort for preparing compounds. Many PBMs prohibit billing for waste, so bill accurately for the quantity dispensed. Proof of dispensing and copay collection. These are two areas where any pharmacy can struggle. For proof of dispensing, track prescription numbers, dates of service, and signatures of the patients or their representatives, as well as the date received if
the medication is delivered. PBM contracts require copay collection with proof, with very few exceptions. Track the front and back of canceled checks, bank deposits or credit card merchant account reporting. Document your policies and procedures, and have timely invoicing and documented attempts at collection. It’s not uncommon to have some bad debt; some PBMs require tax return documents to show a patient is truly indigent and you’re consistently applying waivers for copay collection. Non–FDA-approved products of FDA-approved devices. Note that Medicare Part D drugs are those available only by prescription, approved by the FDA and used for a medically accepted indication. These include prescription drugs, biological products and insulin, and related supplies. It does not include dietary supplements or FDA-approved devices or off-label use. For compounded products, only costs associated with components of a Part D drug are allowable.
The ‘Big Hitters’ The above list comprises the “big hitters” that PBMs focus on during audits, commented Allison Fanning, an area vice president of PBM audits for Gallagher, a consulting company. “The majority of PBMs have daily review programs where they’ll look for outliers for insulins, topicals and inhalers, and immediately contact the pharmacies on these,” she said. Additional areas to watch besides days’ supply are excessive quantities and dispensing of short-term drugs, such as zolpidem (Ambien, Sanofi-Aventis), Ms. Fanning said. Some PBMs are cracking down on creams and other medications being prescribed to add to footbaths, which have no proof of efficacy, added Babette Edgar, PharmD, MBA, an area senior vice president for BluePeak Advisors. Moreover, the opioid epidemic has led to a higher focus on controlled substances, Dr. Edgar noted, adding, “Health plans have really pushed on the PBMs for better fraud, waste and abuse programs to help them manage and identify opioid abusers.” Pharmacies can prepare by keeping careful records or conducting internal audits to check for documentation in frequently audited areas, Ms. Fanning advised. “Many recoupments happen because the pharmacies don’t have the documentation readily available in time for the audit. This includes items such as missing prescription records, signature logs or copay records.” —Karen Blum The sources reported no relevant financial disclosures.
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Specialty Pharmacy Continuum • July/August 2021
OPERATIONS & MANAGEMENT At Blue Cross NC, medication optimization for chronic, challenging disorders such as hemophilia led to savings of roughly
$1.5 million Working With Health Plans to Improve Care Collaborations between health plan and health-system pharmacists can help organizations optimize medication use. At Blue Cross and Blue Shield of North Carolina (Blue Cross NC), for example, such partnerships have been integral to addressing the cost and quality of care, with each side bringing important assets to the table. “It really all comes down to the fact that all of us can work together to improve outcomes in our mutual patients,” said Brenden O’Hara, RPh, a clinical pharmacist, provider engagement initiatives at Blue Cross NC. Health-system pharmacists provide important disease state education to patients and often play a role in increasing the knowledge base of providers. They also have access to electronic health records, which gives them the opportunity to “get a holistic view of prescribed medications, diagnoses and other pertinent information needed to impact patient care and medication choices,” Mr. O’Hara said at the Academy of Managed Care Pharmacy (AMCP) 2021 Annual Meeting. “For instance, if we identify a member as having a hemoglobin A1c [HbA1c] over 8 or if they have not had a measurement in the last 12 months, we can share this information with the pharmacists and they can see when the last HbA1c test was if we have a missing value, what the value was and what medications have been prescribed,” he explained. “Then we can cross-check that information with what we show as being filled through their pharmacy benefit, and we can take measures to encourage utilization and adherence if that is suboptimal.” By monitoring outcomes such as medication adherence and blood pressure, health-system pharmacists can help spot patients who might benefit from clinical education, medication adjustment and adherence interventions as well as lifestyle modifications, all of which can improve quality of care and outcomes for members, Mr. O’Hara said. Plan pharmacists, for their part, “have a view across health systems based on claims data, so they can help identify patient groups requiring more attention,” he added.
Mr. O’Hara said Blue Cross NC pharmacists regularly share reports and insights about their members with health-system pharmacists, highlighting patients who account for the highest drug spending. Plan pharmacists also connect with specialty pharmacists to identify possible alternatives to the highest-cost treatments, particularly when they see patients receiving high doses of these agents. Mr. O’Hara said these higher doses place individuals at increased risk for adverse events and indicate that the drug may be ineffective. “Instead of escalating the dose— which comes with safety concerns and may not equate to improved outcomes— we might identify another medication that could be safer and lead to better outcomes at a lower dose,” he explained.
Dose Optimization Other types of medication interventions include optimizing the dose of a chronic medication to better control the disease and reduce the need for acute medications for conditions like hemophilia, he said, noting that these types of interventions have led to roughly $1.5 million in potential savings for four health-system clients over a one-year period, according to internal data. “Of the $1.5 million in potential savings, there was a single member with hemophilia that accounted for approximately $720,000 in savings,” Mr. O’Hara said, explaining that the member had a duplicate prescription for emicizumab-kxwh (Hemlibra, Genentech) that was being filled at two separate pharmacies. Blue Cross NC pharmacists also identified approximately $1.85 million in potential savings for four health-system partners over one year by switching
some patients’ medications to more cost-effective alternatives, he added. For example, they suggested transitioning from a prenatal vitamin that cost roughly $3,000 per 60-day fill to a similar product that cost $20 for 60 days. In another case, they found an opportunity to switch from branded bupropion, which cost an average $4,000 per fill, to generic bupropion XL, which costs roughly $20 per fill, Mr. O’Hara said. “Health-system pharmacists can use these savings—which are shared between the plan and health system—to justify additional pharmacy resources,” Mr. O’Hara noted.
Banner Health’s Approach Collaborating with health plan pharmacists has been a boon for Banner Health, based in Phoenix, said Candyce Collins, PharmD, an associate director of population health pharmacy at Banner Health’s Department of Pharmacy Services. Dr. Collins said her pharmacists work with plan pharmacists on a scheduled basis “to discuss population-specific opportunities to improve quality and control costs of care and develop annual priorities, like increasing clinically appropriate use of statin therapy to reduce the risks of heart attacks and stroke.” These initiatives are accomplished either by communicating directly with providers through electronic health record messaging and sharing recommendations on medication therapy or by calling a plan member directly to provide education, she said. “When we look at the Part D quality measure performance for statin use in persons with diabetes in our targeted populations, we are currently performing 1% to 4% points higher than last year, which tells us that more health plan members are taking appropriate statin therapy, and we have patient level data that show statins have been prescribed after our pharmacists have made recommendations regarding therapy,” Dr. Collins emphasized. Collaborating also helps align Banner Health’s formulary to ensure health plan members have access to the lowest-cost
medications covered by their pharmacy benefits, she said. “Health plan pharmacists also share best practices they have learned from other providers and other health systems, and they give us data we can use to take actions like implementing member and provider outreach initiatives,” said Dr. Collins, who was not involved in the AMCP presentation. Health plan pharmacists also regularly provide her team with quality measure performance updates, highlighting factors such as CMS Star Ratings looking at medication adherence, she said. That gives Banner Health pharmacists insight into the impact of their clinical interventions, Dr. Collins said. “We use those insights to understand what we’re doing right and how we can improve our initiatives and interventions,” she added.
Collaboration Not the Standard Despite the positive experiences her team has had collaborating with plan pharmacists, the opportunity to work together is not always there, Dr. Collins noted, saying that not all plans have the “near real-time pharmacy claims data that we need to collaborate, intervene and optimize medication therapy.” Scott Soefje, PharmD, MBA, the director of pharmacy cancer care and an assistant professor of pharmacy at Mayo Clinic in Rochester, Minn., said that while “I would love to work with plan pharmacists, we have not reached the point where we do so.” One barrier to that kind of collaboration both at Mayo Clinic and at other institutions is a persistent suspicion from both the plan and health-system sides that the other party is “out to gouge us,” lamented Dr. Soefje, who also was not involved in the AMCP presentation. “In reality, both sides are trying to provide the highest quality care at a reasonable price.” —David Wild The sources reported no relevant financial disclosures beyond their stated employment.
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Specialty Pharmacy Continuum • July/August 2021
OPERATIONS & MANAGEMENT
Cost, Response Rates a Mixed Bag in CAR T-Cell Rx Real-world total cost of care for patients undergoing chimeric antigen receptor (CAR) T-cell therapy for relapsed or refractory B-cell lymphoma can exceed $2 million, while about four of 10 patients do not experience a durable response, according to data presented at the American Society of Clinical Oncology’s 2021 virtual annual meeting. These therapies were approved for this indication in April 2017 (tisagenlecleucel [Kymriah, Novartis]) and October 2017 (axicabtagene ciloleucel [Yescarta, Kite/ Gilead]). Since then, there have been little real-world data on CAR T-cell episode total cost of care (TCC) and post-administration clinical events in commercially insured patients, noted the researchers, from Prime Therapeutics and Minnesota Oncology. To shed some light on this area, the team reviewed integrated pharmacy and medical claims for 74 adults who received either axicabtagene ciloleucel or tisagenlecleucel between January 2018 and June 2020 (abstract e19500). The median TCC for a CAR T-cell episode TCC was $610,999, with a median CAR T-cell drug cost of $411,278—significantly exceeding the $373,000 wholesale acquisition cost for CAR T-cell product to treat B-cell lymphoma (bit.ly/3hPPhrc), The TCC during the initial CAR T-cell therapy period ranged from $350,000 to more than $2 million, with 12% of episodes exceeding $1 million in total cost, reported Brett Sahli, PharmD, the senior director of value and outcomes at Prime. Of the 74 members treated with CAR T-cell therapy during the study period, 29 (39%) relapsed. Of these patients, 22 required subsequent chemotherapy, four required a bone marrow transplant and 13 died or were placed on hospice (these categories were not mutually exclusive), with a mean time to event of 228 days. Dr. Sahli predicted that his team’s findings will prompt discussions on CAR T-cell performance metrics, case rates and future spending, and also guide value-based arrangements for the treatment.
More Relapse Data Needed The findings regarding relapse are similar to what other institutions are seeing, and mirror updated findings of a clinical update of the pivotal JULIET trial of tisagenlecleucel presented at the 2020 annual meeting of the American Society of Hematology (Blood 2020;136[suppl 1]:48-49). Those results showed a relapse-free probability of 60.4% at 24 and 30 months among 61 patients with an initial response. “That’s what we are seeing in our patient population as well,” said Sarah Perreault, PharmD, a clinical pharmacy specialist for the cellular therapy program at Yale New Haven Smilow Cancer Hospital, in Connecticut. “We have
found similar complete remission rates with [axicabtagene ciloleucel] and [tisagenlecleucel].”
The reported cost of care for these commercially insured patients also mirrors what Yale has seen, Dr. Perreault said. “I know it’s on the pricier side for treatment, but in the end, you’re getting to a 60% cure rate, which is promising for people who have gone through two to four lines of therapy after a relapse. Is that worth over half a million dollars? If you’re curing people you would never cure before, then I would say yes.”
But Dr. Perreault noted that the mean follow-up in the study was 390 days. “That’s a decent time, but the end point for lymphoma is considered to be two years before you can say someone has had a complete remission. So, are they getting a complete remission or just one more year of disease-free life before a high relapse rate? It will take longer-term data to answer that question.” —Gina Shaw
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Specialty Pharmacy Continuum • July/August 2021
OPERATIONS & MANAGEMENT
Taking the Measure of a Leader ‘A leader is best when people barely know he exists, when his work is done, his aim fulfilled, they will say: we did it ourselves.’ —Lao Tzu
W
e all have experienced good leaders who we admire, as well as poor leaders who we don’t want to emulate. So, what are the desired characteristics that make a good leader, and the undesirable characteristics of poor leaders? What about me? Am I a good leader? How can I know? I’ve been asking myself that last question most of my adult life, because for most of that time, I have been an avid student of the leadership literature. In fact, I am still a learner and embrace that journey, because I believe we never fully “arrive”; there always are improvements to make in our leadership skills. The issue has been identifying the specific areas to improve. To that end, I’ve taken many assessment tools. The problem is they tend to measure personality inclinations and not true leadership skills. For leadership transformation to occur, I need to know where to focus. One of my more recent discoveries in that search is LionsLead, a product development company (www. lionslead.com) that creates assessments to enhance productivity and growth in all areas of individual, team and organizational development. Let’s explore some of the key LionsLead concepts.
Personality and Behavior Your personality, your hard wiring, is fixed from an early age and reveals tendencies toward certain competencies but is not necessarily an indicator of—nor should it be used as an excuse for—behavior. That’s why LionsLead assessments measure and modify behavior, not personality. By revealing insights about choices, actions and reactions, individuals and teams can drive toward improvements by making small changes in behaviors that can significantly affect outcomes. The assumption underlying that assessment approach is that the best leaders learn how to perfect their actions—“what they do”—rather than being defined by their personality—“what they are.” They are built and become influential, admired and successful by their choices, actions and reactions. Their behaviors and beliefs drive the outcomes.
The Assessment Model LionsLead, like most personality and/or leadership assessment methods, uses its own jargon and definitions, but I will try and drill down to a few that convey the basic approach. The assessments enable rich and meaningful conversations about topics not often measured or discussed by other methods. To that end, they include 192 attributes that lead the person taking the assessment through what is known as a 5T process of transformation: 1. Translate – mapping competencies, to attributes, to behaviors. 2. Truth-Telling – revealing accurate insights. 3. Trust – honoring confidence in all aspects of communication and development. 4. Testify – inspiring actions and achieving profound results. 5. Timely – discovering now what years of observation may never provide.
The Impact of Markers Of Excellence The LionsLead assessments drive progress toward the development of self, others, teams and, ultimately, the culture of companies and entire communities. They create: Purposeful behaviors. These are behaviors that are the measured best practices of results-oriented success. They’re also the behaviors that inspire right actions and choices. Principled leaders. These are leaders whose character matters most, and who embrace what the LionsLead developers have identified as seven character traits of top-tier leaders (more on that below). Authentic culture. Culture that is real and driven by guiding principles.
Benchmarking Most professionals and leaders wonder how they compare to the best leaders. The findings from a LionsLead assessment show how leaders compare to the benchmark of extraordinary leaders. These findings validate the strengths and weaknesses of assessed leaders. The interpretation of the findings should generate insightful and transformational conversations with a LionsLead advisor/coach. The tool is administered online and the results are reported online at the LionsLead administration website. There are a variety of LionsLead assessments that are geared toward different disciplines. My recommendation for pharmacy leaders is the LeadRight Assessment Instrument, which consists of 315 statements to
“Leadership in Action” is authored by Ernest R. Anderson Jr., MS, RPh, FASHP, FMSHP, of Brockton, Mass. Mr. Anderson welcomes your input on leadership issues, at ernestanderson1130@gmail.com.
Ernest R. Anderson Jr., MS, RPh, FASHP, FMSHP
answer, generally multiple choice. Administration of the online assessment takes 90 to 120 minutes. The LionsLead advisor schedules a 90-minute confidential debrief with the leader to review the findings and develop a plan based on the results. A follow-up assessment is administered to measure progress, usually six months to one year later.
LeadRight Report Overview Results of the LeadRight assessment comprise specific, applicable competencies, represented by multiple attributes from the focused instrument and measured by associated behaviors. The report consists of seven competencies and 27 attributes. The seven LeadRight assessment competencies are: 1. Self-Leadership 2. Leadership Essentials 3. Getting Things Done 4. Leading Others 5. Composure 6. Candidness 7. Personal Values Factor Another output of the assessment tool is the strengths that you would bring to a team. There are seven team roles that high-functioning teams must possess, as derived from the team role model of Meredith Belbin, PhD (bit.ly/39YSv6m). The team roles as defined by Belbin are: 1. Coordinator – creates the way team effort is applied. 2. Shaper – influences the way teams function. 3. Plant – advances new ideas and strategies. 4. Monitor – carefully evaluates ideas and suggestions. 5. Resource Investigator – explores and reports on ideas. 6. Team Worker – plays a supportive role filling gaps. 7. Complementor/Implementor – brings strong tactical focus and execution. The tool results identify a primary and secondary role and grades the participant’s ability in all roles as fair, good and strong. Ideally, your team will include enough well-matched members to fulfill all of the key roles. In some cases, an individual could fulfill multiple roles in their areas of strength. If all members of a team take the LeadRight tool, not only can one look at the best roles for team members to play, but one can look at the strengths and weaknesses of the entire team across the entire set of competencies. Based on this overview, hopefully you’ll appreciate that LionsLead can be a powerful leadership measurement tool. In fact, it’s validated by at least two statistical methods: the use of Cronbach scores, which measure an assessment’s internal consistency, and item response theory, which determines the relevant strengths of each assessment statement and helps to ensure accuracy. ■
Next column We will explore specifics of the LeadRight tool and how you might be able to use it to assess the competencies and attributes of your team members.
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An Infusion Center Push continued from page 1
care, Mr. Norris said. With many autoimmune biologic specialty drugs having a limited distribution, the logistics of getting this in the home can be so burdensome that “it makes sense” to administer them in AICs, he noted. “We are seeing payors push patients out of the hospital into more cost-effective sites of care,” Mr. Norris said. “We see this space continuing to expand, especially as the formulary of drugs gets larger.” The SOC optimization trend has been building for several years, said Logan Davis, PharmD, MBA, the director of franchise development for Vital Care Infusion Services, in Meridian, Miss. The COVID-19 pandemic further demonstrated to the industry that being dependent on hospital-based infusion centers isn’t ideal, he said. “There just isn’t enough capacity in the country at the moment” for standalone infusion centers, added Bryan Johnson, a co-founder and the CEO of WeInfuse, echoing the push by insurers to administer infused medications outside the hospital setting. “Wherever we can get a chair, an IV pole and a nurse, let’s try to make that site accessible to a patient, whether that’s in a home or through a management company. You’ll continue to see that—there’s just a lot of pressure coming down from the top.” Adding to the market opportunity for expansion is a robust pipeline of specialty infusion drugs, including aducanumab (Aduhelm, Biogen), approved in June for the treatment of Alzheimer’s disease (see page 20 for more coverage), and several others still in trials for Parkinson’s disease, the panelists said. The boundaries are blurring among specialties, medications and diseases, Mr. Johnson noted. For example, infliximab (Remicade, Janssen) is being used for both gastrointestinal and rheumatic conditions. Medical specialists accustomed to having few infusion medications available aren’t likely to have office-based infusion suites, he added, and as specialty infusion medications cross into more common diseases covering more people, the demand for services will continue to rise. Payors are reaching out to urgent care center chains, home infusion pharmacies and other regional service providers to open capacity to treat specialty medication patients in AICs, Mr. Norris said. In addition, some home infusion pharmacies are building infusion suites to capture the medical benefit market they could not traditionally access through the pharmacy benefit. Some home infusion pharmacies are adding infusion management services to their
list of offerings. The infusion space is experiencing significant investment from both private equity and industry, Mr. Johnson added.
Why Launch? There are numerous reasons to launch an infusion clinic, Dr. Davis said. Some of his franchisees wanted to be able to accept more referrals in their markets, access Medicare Part B benefits for referred patients, access drugs and better pricing limited to the infusion clinic class of trade, and access commercial payor contracts available to infusion clinics. But a sharp marketing and sales strategy is critical to success when starting out, the panelists said. “This is not a ‘build it and they will come’ space,” Mr. Norris said. “You can have the best nurses, the nicest building and the finest equipment, but you have to earn the trust in the community from referring providers.” This can involve the hard work of cold calling, creating medication-specific order forms to ease work for referring physicians and treating referrals as gold. Communicate back to referring physicians how the infusion went, he advised, even if it’s just a quick treatment note. When thinking about the physical space and build-out, most operators focus on factors such as infection control and meeting codes. But it’s equally important to consider patient amenities such as great parking, expanded hours, free Wi-Fi and coffee, Mr. Johnson said. “Think more Starbucks and less stainless steel,” he said. “These patients have chronic conditions they will visit you with every few weeks, maybe for the rest of their lives. They want to be comfortable. You want them to look forward to coming to your facility.” Mr. Johnson suggested these additional operational strategies: • Consult resources such as the National Infusion Center Association or the Infusion Nurses Society to note best practices and standards. • Hire a licensed professional (e.g., a physician, physician assistant or nurse practitioner) for clinical leadership. Their expertise will be needed in updating and maintaining protocols as well as dealing with any adverse events and communicating with other health care professionals. • Write and review emergency protocols, and post them clearly for your clinical team.
Not All Centers Created Equal Although the infusion itself is the same whether it’s administered in a hospital or another setting, AICs are
different in terms of their regulation, reimbursements and class of trade, commented David Franklin, the president of Advanced Care Consulting Services, in Ray, Mich. “There’s a really interesting new hybrid where independent infusion centers are partnering with physicians,” he said, “where physicians hold stock in the company, but they partner with independent infusion centers to operate the services through management
contracts. That’s what’s going on now more than anything.” Still, some of these independent infusion centers are facing reimbursement challenges because they may not be recognized by Medicare and Medicaid, Mr. Franklin noted. —Karen Blum The sources reported no relevant financial disclosures other than their stated employment.
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HSSPs Still Fighting continued from page 1
in part by leveraging the superior continuum of care they claim is a hallmark of their practice model. Whether it’s streamlining the prior authorization (PA) process, reaching for legal tools to break down payor walls, or speeding the pharmacy and therapeutics (P&T) specialty drug approval process, health systems have shown they are up to the challenge, as outlined in several presentations at the 2021 virtual ASHP Specialty Pharmacy Conference.
LDD Disconnect Only
37.7% of respondents reported gaining access to LDDs
82.1% cited “manufacturer refusal to engage” as major roadblock
71.7% said they were “frozen out or blocked by payors” LDD, limited distribution drugs
The conference kicked off with a presentation of the new survey, with several of the findings underscoring key practice issues and frustrations for health-system specialty pharmacies (HSSPs). The survey was sent to 230 contacts at 206 different HSSPs; 114 (53%) completed the survey. The survey included 99 questions over eight domains addressing demographics, workforce issues, operations and payor access, among others.
Most HSSPs dispense fewer than 45,000 specialty prescriptions per year and have an annual gross revenue of less than $100 million, the survey found. Most of these specialty pharmacies are relatively new, with 73.8% of survey respondents saying their organization has offered specialty pharmacy services for six years or less. “We also learned from this survey that HSSPs are primarily regional in their approach,” said Craig Pedersen, PhD, RPh, pharmacy manager at Virginia Mason Medical Center, in Washington state, who conducted survey development and analysis. “The majority have five state licenses or fewer; slightly over one-third only have one, although some have many— even up to 50 if they want to serve all 50 states. But most are like my health system: We are located in Washington and have licensees in Alaska, Arizona, California and Oregon. But we’re not reaching into the Midwest or the East because those aren’t patients our medical center serves.” Some of the survey’s other takeaways: Integration. The HSSP practice model is integrated into specialty clinics, with 64.9% of respondents reporting that they have HSSP pharmacists dedicated to specific clinics and involved in treatment decisions and drug therapy selection prior to prescriptions being written. “This finding is consistent across specialty pharmacies regardless of size,” said JoAnn Stubbings, BSPharm, the former associate director of specialty pharmacy at the University of Illinois at Chicago College of Pharmacy, who served on the advisory committee for the development of the survey. “This upstream involvement of HSSPs allows for appropriate drug selection before PA is submitted, as well as management of safety parameters, leading to faster medication access and better patient outcomes.” Pharmacy versus medical benefit. The HSSP business model is focused on self-administered (96.2%) and clinicadministered medications (80.2%) under the pharmacy benefit. Only a small number of HSSPs provide self-administered (31.1%) or clinic-administered medications (22.6%) under the medical benefit. “What I’ve seen in most health systems is that the medical benefit is typically managed by another area of the pharmacy enterprise, but that is changing,” said Ms. Stubbings, a member of the Specialty Pharmacy Continuum editorial advisory board. “There is significant overlap, and
we are starting to see HSSPs building infusion suites and enter home infusion, and this is becoming increasingly important. We hope to identify these trends in a future survey.” Access still a challenge. HSSPs have had only moderate success in gaining access to LDDs, with resistance from manufacturers and payors cited as major roadblocks (Figure). Inflammatory conditions No. 1. The most common therapeutic categories served were inflammatory conditions and hematology/oncology (both 92.4%), hepatology/hepatitis C (85.7%), neurology/multiple sclerosis (78.1%) and infectious disease/HIV (70.5%). More than half of all respondents indicated that their HSSPs also provided care in cardiology, endocrinology, cystic fibrosis, respiratory/pulmonary arterial hypertension and solid-organ transplant, and 90% said they offer PA support. 340B, shrinking reimbursements among other challenges. HSSPs see access to payor networks (82.9%), 340B Drug Pricing Program changes (42.9%) and shrinking reimbursement from payors (40%) as their top challenges, with new populations to serve and new therapeutic categories rated as leading opportunities for growth. “HSSPs are responding to these challenges by exploring new payment methods, new models such as value-based care, and further integration of services,” Ms. Stubbings said. “Overall, I am optimistic about [trends] that could positively impact our model. Some are legislative, some market-based, some based on our advocacy. Provider status is one change that is getting closer at the state and federal level, and could offer new opportunities for clinic-based pharmacists to be recognized and bill for their services. Some states are banning white bagging, and we are also seeing legislation proposed at federal level on DIR [direct and indirect remuneration] reform.”
Legal Tools Help Protect Specialty Pharmacies HSSPs have some legal tools they can use to address network access and other practice challenges cited in the ASHP survey, Jesse Dresser, Esq, an attorney with Frier Levitt LLC, noted during a conference session on legal conundrums in specialty pharmacy. Mr. Dresser advised that no matter the issue—compliance, network admission, site-of-care policies or reimbursement— it’s important to start with the patient and their type of plan. “That will dictate what laws and rules apply and what your rights and obligations are,” he said. As an example, he pointed to the three types of specialty pharmacy networks: 1. Closed or exclusive. “These are the ones where the sponsors are not
allowing anyone in except their own wholly owned specialty pharmacy,” Mr. Dresser said. “We typically see these arrangements in the employer-sponsored commercial market. Large plan sponsors with thousands or hundreds of thousands of employees, like PepsiCola, typically self-insure rather than spend an extra 10% to 15% on premiums on behalf of their employees, but they will still contract with an insurance company to administer their claims and PBMs to administer their pharmacy benefits. In this context, state laws don’t really apply and federal rules like those involving Medicare or Medicaid [also] don’t really apply.” Plans like these, he noted, are subject only to the Employee Retirement Income Security Act (ERISA), which is silent on what pharmacies must be in a network. 2. “Open,” but with heightened admission criteria. Such criteria include requirements for multiple forms of accreditation or licensure in all 50 states. 3. Truly open specialty pharmacy networks. “These are typically found in Medicare networks, where there is a robust federal Any Willing Provider law and a prohibition on payors from limiting who can be in the network,” Mr. Dresser said.
White and Brown Bagging Still a Concern As for specific legal strategies to deploy, that depends on the practice challenge, he noted. One of the most problematic issues is the growing trend among PBMs to move claims processing from the medication side to the pharmacy side, requiring more white and brown bagging—a hot topic throughout the conference. The practice has had a major affect on hospital infusion, Mr. Dresser said. “About this time last year, several large payors took virtually identical steps to require that in-office infused medications be filled at their wholly owned specialty pharmacies, and placing limitations, or removing the ability altogether, on providers’ ability to source and seek reimbursement for medications administered in their facilities.”
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There are laws being proposed in some states that would make mandatory white bagging illegal, but what can hospital and health-system specialty pharmacies do in the meantime? “Depending on the type of plan involved, you might be able to deploy Any Willing Provider” legislation, he said. “All 50 states and the District of Columbia, through Medicare Part D, are subject to the federal Any Willing Provider law, meaning that any willing pharmacy able to participate in a network’s terms and conditions has to be allowed in. The law is fairly robust, and its guidance requires that those terms and conditions have to be reasonable and relevant. It has been used successfully to protect not only network access, but fair and appropriate reimbursement for specialty pharmacies to participate in Medicare Part D programs.” There also are some state-based Any Willing Provider laws as well as state laws banning mandatory mailorder pharmacy. “About 33 states have some level of this kind of protection,” Mr. Dresser said. “I would encourage anyone facing any kind of exclusion to figure out what type of network the patients you are not being able to fill for are in, and then see if you can use some of these legal tools to your advantage.”
Audits, Other Challenges Mr. Dresser stressed that white bagging, although a big challenge, isn’t the only concern that HSSPs have to address with legal tools, citing the following: Fair pharmacy audit laws. “These laws apply at the state level, typically in the context of commercial insurance and not necessarily ERISA or Medicare,” Mr. Dresser said. “They provide time limits on PBM audits, as well as audit appeal procedures. They often limit the number of prescriptions that can be looked at in a given audit, and helpfully, prohibit recoupment for clerical errors or things that can be ‘cured.’” Prompt payment laws. “These include look-back periods limiting PBM audits,” Mr. Dresser explained. “Florida, for example, says you can’t go back more than 30 months in terms of a repayment demand. They also prohibit PBMs from unilaterally offsetting claims to recoup on audits, saying, ‘You owe us $100,000, and we’re going to recoup it immediately. You can appeal but we’re going to start now.’ If you’re facing an audit and potential recoupment, this is a good tool to have in your arsenal.” Mr. Dresser also spotlighted recent actions by PBMs in the 340B space. “They are trying to retain the spread between the costs of the 340B drug and the PBM reimbursement,” he said. “They send out notices to pharmacies requiring them to submit 340B claims with a
submission clarification code to signal to the PBM that it is a 340B claim.” This typically involves the use of a Submission Clarification Code of “20” in the NCPDP Field 420-DK. If a claim is 340B, the PBM then reimburses the pharmacy at a lower rate, for example, average wholesale price (AWP) - 30%, compared with a rate of AWP - 15% for non-340B claims. “Essentially, PBMs are looking to usurp that savings for themselves,” Mr. Dresser said. “This is not necessarily limited to Medicaid programs managed by PBM; it could include commercial plans and often does. They are also using third-party administrators to get access to this information. Some PBMs own their own third-party administrators, so they might have the ability to make the determination, even if the pharmacy didn’t submit the clarification code at the point of sale.” Mr. Dresser noted that there has been some success in pushing back against these efforts. “The tides have turned a bit in state legislation,” he said. “Some states, including most recently Ohio, have passed laws prohibiting PBMs from differentiating 340B and non-340B pricing. So, if you are facing mandates from a PBM that you use the clarification code, or otherwise encountering 340B-specific pricing, I encourage you to speak to counsel who can guide you on some of the recent tools that are available.”
Ascending the Specialty Summit
D
uring a town hall session at the 2021 virtual ASHP Specialty Pharmacy Conference, ASHP shared highlights from its February 2021 Specialty Pharmacy Future Directions Summit. One key accomplishment of the summit was the preparation of 71 consensus recommendations. The recommendations covered 13 practice domains, including specialty pharmacy practice model and performance; patient care services and access; workforce competency, credentials and culture; and safety, quality, outcomes and value, among others. More details on the recommendations will be published in a fall issue of the American Journal of Health-System Pharmacy. Udobi Campbell, PharmD, MBA, the regional executive director of pharmacy at UNC Health, in Chapel Hill, N.C., and the chair of the Summit Steering Committee, started the town hall with some observations on why the summit was such a timely and important initiative. She cited market forces as one powerful driver: Spending in the United States on specialty medications is projected to exceed 50% of overall drug expenditures in 2021, she noted. Vertical integration and the increasing adoption of limited drug distribution models for specialty drugs are other factors that are making it difficult for health systems to stay on the specialty pharmacy sidelines, she noted. “Probably just like me,” Dr. Campbell told attendees, “you have to make the case with your leadership every month that if we bury our heads in the sand, [these market forces] all threaten our very existence in the specialty pharmacy space.” Fortunately, many health-system pharmacies have responded proactively, she stressed. Indeed, 70% of multihospital health systems launched specialty pharmacy services between 2012 and 2017, she noted. Moreover, the prevalence of specialty pharmacy operations in hospitals grew from 7.8% in 2015 and 8.7% in 2016 to 19.9% in 2018 and 26.4% in 2019. —David Bronstein
Speeding Access To Medications As noted, timely access to specialty medications was a key concern cited by respondents to the ASHP practice survey. At Mass General Brigham, in Boston, the solution to this challenge was to employ integrated specialty pharmacists in its health-system clinic to mitigate treatment delays. In a retrospective chart review of 121 patients being prescribed specialty medications from an ambulatory autoimmune/allergy practice, the median time to dispense from prescription receipt was four days for the healthsystem specialty pharmacy and 13 days (P<0.001) for an outside specialty pharmacy. Using an integrated specialty pharmacy model versus a standard clinical workflow also yielded other benefits, including decreased time between PA requests and PA submission (one day vs. 12 days; P<0.001); and decreased time between the PA submission and recorded PA outcome (one day vs. 3.5 days; P=0.03). “Timely medication access is a primary barrier in the treatment of patients with autoimmune disease,” said lead author Soha Elshaboury, PharmD, a pharmacist clinical coordinator for the health system. “This is mainly due to
the cumbersome process around prior authorization and the resources it takes to coordinate this process.” For the study, Dr. Elshaboury and her colleagues completed a retrospective chart review for patients receiving prescriptions for self-administered specialty medications between Feb. 26 and Dec. 24, 2020. These were largely
needed for clarifications. Patients who received a refill after that date, in which they had a PA completed by clinic staff to start their therapy, were classified as standard of care. The investigators gathered data using electronic health records (EHRs), patient management software, adjudication software and records of phone calls
‘Timely medication access is a primary barrier in the treatment of patients with autoimmune disease. This is mainly due to the cumbersome process around prior authorization and the resources it takes to coordinate this process.’ —Soha Elshaboury, PharmD for injectable medications such as dupilumab (Dupixent, Sanofi/Regeneron), benralizumab (Fasenra, AstraZeneca) and mepolizumab (Nucala, GSK). Patients receiving a new prescription after Feb. 26 who required a PA to start therapy were categorized as the intervention group, where the specialty pharmacy staff processed the PA, reviewed therapy and consulted providers as
to outside pharmacies. The standard-of-care workflow started with a provider discussing therapy with a patient and asking them to complete the manufacturer’s hub form. The provider would then notify the medical assistants that therapy was to be initiated and would provide completed chart notes, the specialty medication prescription see SPEEDING ACCESS, page 12
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SPEEDING ACCESS continued from page 11
and completed hub form. The medical assistant would then request a benefits investigation via the hub by submitting the appropriate forms. After the benefits investigation, the clinic would submit the PA. If it was approved, they would send the prescription to the patient’s dispensing pharmacy or the provider would have to work on a PA. In contrast, the specialty pharmacy intervention workflow focused on a realtime benefits investigation after the provider entered the prescription and submitted the PA as needed in real time. If a PA was approved, the prescription would be reviewed clinically and sent to an outside specialty pharmacy to dispense
or to the integrated specialty pharmacy if they were a contracted pharmacy. If the PA was denied, a specialty pharmacy clinical pharmacist would work with the provider to either appeal the process or choose a different therapy. Overall, time to medication access was reduced in the intervention group compared with the standard of care when an outside specialty pharmacy was used for dispensing, with the benefit compounded when the HSSP was used for dispensing (seven vs. 37 days; P<0.001). Dr. Elshaboury and her colleagues attributed the reduction in time to medication access to the following features of the intervention workflow: • complete access to patients’ electronic medical records;
• utilization of software to manage PA requests; • prescription capture at the time of prescribing; • real-time benefits investigation; • on-site PA submission; • comprehensive clinical pharmacy review; and • on-site medication dispensing. The specialty pharmacy already had a standing relationship with the rheumatology, dermatology and inflammatory bowel disease clinics before the study. The autoimmune group was the last discipline being rolled out. “We have gotten excellent feedback from our physicians,” Dr. Elshaboury said. “Everybody had a sense that we had drastically improved this process, but we really wanted to put a number on what that impact was, and we picked patient medication access ultimately as the best marker of that impact, and decreasing the time to medication access.”
Kudos for Brigham’s Effort To Embed Pharmacists Embedding pharmacists directly in specialty clinics in this manner is what HSSPs should do, commented Josephine Hurtado, BSPharm, the director of specialty pharmacy for Texas Children’s Hospital, in Houston. Doing so at her
hospital has led to patients receiving new prescriptions for specialty medications in less than a day, as opposed to four days through outside specialty pharmacies, or about 9.5 days for new prescriptions needing a letter of necessity or PA. These pharmacists are trained in specific diseases, and being housed in a clinic, patients get to know them, noted Ms. Hurtado, who was not part of the ASHP conference. “When the physician writes the prescription, we’re right there able to do any prior approvals, or ask any questions if there’s an error,” she said. “We can literally get up, go down the hall and have a conversation with a physician. Then we have access to all the labwork, so we can provide letters of medical necessity, or if there are any lab values or clinic notes that need to be reviewed, we can do so. “I honestly believe that being part of a medical care team and being able to have that conversation where you know the physician by first name is very beneficial for us,” she added. “Otherwise, those outside pharmacies are calling and leaving a voice mail potentially and waiting for it to be returned.” —Karen Blum The sources reported no relevant financial disclosures.
Vanderbilt Software Tool Hastens Benefits Review For Specialty Medications
A
software tool developed by pharmacists at Vanderbilt University Medical Center, in Nashville, Tenn., has helped address a major practice hassle discussed during ASHP’s National Survey of HealthSystem Specialty Pharmacy Practice: navigating the coverage issues that crop up when inpatients need nonformulary specialty medications. “An increasing number of specialty medications have recently been approved by the FDA that may require inpatient administration or are given in combination with IV therapy, such as oral oncolytics and hepatitis C medications,” said Houston Wyatt, PharmD, CSP, the program director for complex projects at Vanderbilt’s specialty pharmacy. Managing these medications on the inpatient side presents several complex issues when it comes to billing and coordination that often are not considered, Dr. Wyatt noted. He cited, as an example, having to navigate limited distribution networks or Risk Evaluation and Mitigation Strategy programs that may exclude the inpatient hospital pharmacy. And then there is the challenge of managing inventory with expensive, infrequently used specialty medications. In addition, he said, part of the decision to initiate therapy depends on the ability of patients to access the medication for continued treatment when they are discharged. To try to overcome some of these challenges, in 2019, the P&T committee devised a plan to communicate updates on specialty medications needed for inpatients using a software tool developed at the medical center several years prior called REDCap (Research Electronic Data Capture). With Vanderbilt’s revised workflow, when nonformulary medications are ordered in the EHR, a REDCap link alerts the provider to enter a rationale for the medication’s use and to provide any relevant clinical information for the P&T committee. Then,
REDCap sends an email alert to the ordering provider, P&T committee, specialty pharmacy, inpatient central pharmacy and procurement team. While the request is being reviewed, the specialty pharmacy completes a benefits investigation to ensure the medication can be obtained for outpatient use at discharge. The pharmacy also coordinates with any external specialty pharmacies if medication must be dispensed after discharge, and completes its part of the REDCap survey to alert the P&T committee about the benefits investigation outcome. If the P&T committee approves the request, the central pharmacy is alerted to procure the medication for inpatient use and completes its part of the form showing product was obtained. At discharge, the specialty pharmacy provides a 30-day supply of medication or ensures the medication can be supplied to the patient through an external pharmacy. Each request is stored as a record in REDCap so the P&T committee can decide whether to add medication to the formulary based on volume.
The P&T committee receives one to two requests for nonformulary medications per week, generally for oral oncolytics such as venetoclax (Venclexta, AbbVie/ Genentech), midostaurin (Rydap, Novartis), dasatinib (Sprycel, Bristol Myers Squibb), or hepatitis C antiviral medications for patients receiving a liver transplant from a hepatitis C–positive donor, Dr. Wyatt said. “It’s definitely helped expedite the P&T decision process and medication procurement so patients aren’t starting IV therapy without oral treatment,” he said. The team also can see the status of all orders in real time on REDCap, and they are reimbursed for outpatient medications given at discharge. “Usually this process is turned around in 24 to 48 hours. Everyone involved has voiced how easy it’s been, and how transparent it is to help them get medication to the patient. And it also provides reporting capabilities.” —K.B. The sources reported no relevant financial disclosures.
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6 Strategies for Navigating Specialty Networks With specialty pharmacy now a significant driver of health care costs—spending on specialty drugs represented 49.6% of total drug expenditures in 2020, according to the latest industry figures—health-system practitioners and leaders face significant financial challenges, experts said during the 2021 virtual ASHP Specialty Pharmacy Conference. Hospitals and health systems have significantly expanded their specialty pharmacy presence over the past several years, said Matthew Rim, PharmD, the associate director of specialty pharmacy services at the University of Illinois at Chicago College of Pharmacy. In 2019, 89% of all hospitals with 600 or more beds had a specialty pharmacy, compared with just 44% in 2015; for hospitals with 400 to 599 beds, the figure jumped from 21% to 49%. Even among the smallest hospitals, there was significant growth: Only 6% of hospitals with less than 200 beds had a specialty pharmacy in 2015, compared with 18% in 2019. “We continue to struggle with many of the same challenges that we faced when I started working in specialty in 2013, including access to limited distribution drugs, channel management and administrative burden,” Dr. Rim said. “When I ask my colleagues what keeps them up at night, I hear about payor access restrictions and decreased reimbursement, the difficulty of entering and maintaining our place in PBM [pharmacy benefit manager] specialty pharmacy networks, and not being able to provide services to many of our patients due to white bagging and site-of-care [SOC] restrictions.” To navigate some of these networkrelated challenges, Michael Gannon, PharmD, an assistant director of specialty pharmacy at the University of Illinois at Chicago College of Pharmacy, shared some strategies around the contracting process. Collect employer information. Employers frequently carve out the pharmacy benefit from their primary medical provider, he noted. “A patient’s pharmacy benefits may be managed by a completely different company, which can create complications when doing benefit investigations and determining if you’re eligible to bill.” Ask for extensions. If you receive a notification that you are out of network for a particular patient’s coverage, ask for a month’s grace period. “This can allow you time to transition these patients to their preferred specialty pharmacy and working out strategies to apply to that network,” Dr. Gannon said. Negotiate rates. “For example, depending on your hospital system and how you view different business units, you could negotiate different
rates for other services, such as radiation therapy or diagnostics, to stay in or get into the pharmacy network,” Dr. Gannon said. “Reimbursement rates are subject to negotiation as well.” Negotiate exemptions. “If you have 100 patients within a certain network who are already with your specialty pharmacy and their care has been great, you could argue for an exemption so that you can still manage those patients and avoid gaps in care, and only new start patients would be subject to out-of-network management.”
Why White Bagging Persists Melissa Ortega, PharmD, the system executive director for ambulatory pharmacy services at the Massachusettsbased health system Wellforce, agreed that payor-mandated white bagging is a major point of frustration for some HSSPs. The practice is no longer an “exception to the rule” situation, but is becoming a standard of practice, she said during the ASHP specialty pharmacy conference. Dr. Ortega cited the following market dynamics driving white bagging, in which a third-party specialty pharmacy dispenses a drug and sends the drug directly to the hospital pharmacy or physician’s office for storage and administration by a clinician: Medical benefit changes. “Payors who tend to have more formalized relationships with their own specialty pharmacies are now shifting many infusion and injection-based therapies from the
of care for them. And what we see is not actually cost savings, but a shift of the cost burden [to] hospitals and providers.” She pointed to an uptick in the use of SOC programs over the past several years. “UnitedHealthcare’s landmark national policy change adopted in 2017 really set the tone for commercial payors,” she said. (That policy requires many expensive specialty biologics to be administered in a nonhospital setting.) “Recently, they just extended that policy to more than 70 medications, incorporating drugs usually administered in infusion clinics for the most complex and vulnerable patients. Blue Cross Blue Shield, Anthem, Aetna and Cigna also recently announced their site of care management policies.” Vertical integration. Major insurers such as Aetna, Anthem, Cigna and UnitedHealthcare now own PBMs, specialty pharmacies and provider services.
‘When I ask my colleagues what keeps them up at night, I hear about payor access restrictions … decreased reimbursement…. entering and maintaining our place in PBM specialty pharmacy networks, … and not being able to provide services to many of our patients due to white bagging and site-of-care restrictions.’ —Matthew Rim, PharmD Bridge gaps. If you receive notification that your pharmacy is being excluded from a current network, ask to sign a letter of intent to continue services to those patients while you reapply to the network. “This has been successful for us, buying us time to submit applications to stay in a network or be considered for a network,” Dr. Gannon said. Get organized. The network application process is daunting, but all the requirements are likely already managed and operationalized in your specialty pharmacy. “The biggest issue in the application process is organization. You want to be very precise about how you meet each of the network’s requirements, in order to minimize misinterpretation,” Dr. Gannon said. “One year, we applied to a network and were denied based on our application. The next year, we resubmitted, and all we changed was how we organized the application. None of the content changed, but this time we were allowed to move forward. “Each stage of the contracting process may present potential strategies to help manage potential risks,” Dr. Gannon concluded. “Engaging multiple subject matter experts across legal, finance, operations, managed care and decision support can help streamline executing third-party payor contracts.”
medical benefit to the pharmacy benefit,” she noted. “From the PBM perspective, they feel shifting everything toward the pharmacy benefit simplifies things, but while it shifts the payment toward their specialty pharmacy, it complicates drug flow and our ability to care for patients.” SOC management policies. “There has been an increase in program mandates versus a voluntary option for the patient and/or the physician,” Dr. Ortega said. “These programs focus on redirecting patients to the lowest cost locations, but they aren’t necessarily always clinically appropriate locations. They aren’t allowing the patient and physician to make a shared decision on the best site
“These companies now really have complete oversight of both the provider and pharmacy assets,” Dr. Ortega said. “For example, UnitedHealthcare, the parent company, allows Optum to manage their prescription pharmacy benefit, which tends to steer the referrals or restrict prescriptions to their own specialty pharmacy, Briova.” That process, she noted, “almost completely bypasses our ability as hospitals and health systems to provide safe and timely care.” —Gina Shaw The sources reported no relevant financial disclosures.
Defining Specialty Pharmacy
D
efining specialty pharmacy has been an elusive goal,, with several organizations taking a stab at it during their conferences and committee meetings. Now, ASHP has its own working definition, based partly on feedback gained from the group’s inaugural National Survey of Health-System Specialty Pharmacy Practice: “The health-system specialty pharmacy is an integrated, advanced practice model of care that incorporates specialty medication use management across the continuum of care.” JoAnn Stubbings, BSPharm, who served on the advisory committee for the development of the survey, added some further guidance: “We intend for you to use this definition in describing and justifying our model as leaders in this movement. We have a powerful story to tell, and we are in the position to define specialty pharmacy and not be defined by payors or accrediting organizations.”
—G.S.
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Medicare Pays Heavy Price for the Generics Gap Dispensing a brand-name drug when an equivalent generic is available can take a heavy financial toll on the Medicare Part D program as well as on patients’ pockets, according to researchers at Johns Hopkins University. In a study released earlier this year, the investigators zeroed in on 2.5 million Part D claims for branded drugs in 2017, that originated from either prescribers’ requests (1.4 million) or patient requests (1.1 million). They found that if available generics had been substi-
3 Solutions For Lowering Rx Costs
consumers and other more downstream product selection factors—all the other factors that account for selection of a brand product, especially when the drug is dispensed at the pharmacy counter.” The new study, Dr. Socal added, “really helped shine a light on how much
1. Ensure Medicare Part D plans cover all generic products at launch, particularly first generics.
2. Provide for placement of all generic products on tiers designated as generic and removed from brand tiers.
‘It was a big surprise when [HHS] released a report in 2018, showing that branded versions of multisource drugs were still being used so frequently that the additional cost to the Medicare care program was about $3 billion a year.’
3. Create a separate specialty tier to allow for differentiation among specialty brands and generics. Source: Association for Accessible Medicines.
—Mariana P. Socal, MD, PhD tuted for all those branded products, the Medicare program could have saved $1.67 billion and patients’ out-of-pocket expenditures could have been reduced by $270 million (JAMA Netw Open 2021;4[3]:e210483). Branded drugs requested by prescribers or patients typically carried the highest price tags, researchers found, with an average price 73.9% higher than the corresponding generics. “We had assumed that doctors were prescribing, and patients were taking, whatever drugs were most affordable,” said Mariana P. Socal, MD, PhD, an associate scientist at Johns Hopkins Bloomberg School of Public Health, in Baltimore, who led the study. “So, it was a big surprise when the Department of Health and Human Services released a report in 2018, showing that branded versions of multisource drugs were still being used so frequently that the additional cost to the Medicare program was about $3 billion a year” (bit.ly/3hVmPUN). “We tried to understand what was driving this branded drug utilization,” Dr. Socal told Specialty Pharmacy Continuum. In an early investigation, the Hopkins researchers focused on the influence that manufacturer rebates and other financial incentives had on formulary coverage by Medicare prescription plans and pharmacy benefit managers (PBMs) (JAMA Intern Med 2019;179[6]:832-833). “In that study, we did find that PBMs were sometimes favoring branded products,” Dr. Socal said. “But that wasn't accounting for everything we saw in that report. That's why we continued investigating the role of prescribers and
worse off not only the he payors are when brandded products are used ed instead of the generric, but also patients. s. We saw that when the he patient requests a brand and drug (dispense-as-written ritten code 2), the patient is paying on average almost 60% % more than for the generic.” For plans, the average program discount for a generic was about 73%. “So, plans were paying more and patients were paying more,” she said.
Prescriber Reluctance Dr. Socal noted that prescribers are often reluctant to substitute a generic when a patient's condition, such as epilepsy, is well controlled with a branded drug. “We’re not concerned about the utilization of branded drugs if patients are well managed on one, and their condition is so severe that nobody wants to change their regimen,” she said. However, branded requests weren't limited to drugs with narrow therapeutic indexes, she added. “Every single drug we investigated had prescriber and patient requests for brand dispensing.” Dr. Socal also said she wasn't concerned about the “vast majority” of cases where there was “very healthy competition in the market” because prices of branded and generic drugs were similar. What was concerning, she said, were brand drugs “costing so much more to patients” when there were less expensive alternatives, “and because of marketing or advertisements, patients are hesitant to take a generic and choose a branded drug.”
Dr. Socal suggested that improving the understanding of the generic and brandname drugs' relative costs and efficacy was one solution to the issue. “Prescribers want to do whatever is best for their patients,” she said. “Often a prescriber may choose one product versus another without knowing their patients are going to pay much more.” For patients, she said, the answer can be “understanding the role that directto-consumer advertising plays in helping them become familiar with a brand.” Discount coupons offered by manufacturers, she added, also can make a brand-name drug seem relatively inexpensive to a patient, “but the price to the system is much higher. “So, there are solutions in terms of raising awareness of physicians and patients and limiting direct-to-consumer advertising,” Dr. Socal said. “But there are also changes that should occur at the plan level to help patients make decisions based on how much one drug will cost as opposed to another. The Medicare program could increase that transparency, at least for some of the drugs we found.”
“About a third of all first generics are “A still not launched, and when they do launch, they are facing severe delays laun in fformulary coverage, in particular Medicare Part D plans,” said Craig by M Burton, the vice president for policy at Bur the Association for Accessible Medicines (AAM). cine He based the estimate on a study by H AAM that took “an extensive look” at first-generic launches and Medicare coverage rates from 2016 to 2019 (bit. ly/3BogVDk). Part of the reason for the delays, Mr. Burton said, “is directly tied to some of the unique design features of the Medicare program that reward plans for the use of higher-cost brand drugs, even when there is a generic.” Mr. Burton noted that while a lot of focus has been on rebates, another key issue has received less attention: the role that the Part D coverage gap plays. Under that program, he explained, a brandname drug provides 70% discount for patients entering the gap, while a generic, by law, cannot offer a discount. “The brand discount is treated as a patient's out-of-pocket spending,” Mr. Burton said, “and it moves the patient very quickly into the catastrophic phase of the benefit,” where patients' costs are dramatically lowered. “So, especially for newer, high-cost brands, a plan is financially encouraged to stick with the brand drug.” —Bruce Buckley
Marketing Delays Delays in marketing generic versions of high-priced branded drugs are another factor helping to drive up costs for Medicare plans and beneficiaries.
Dr. Socal reported that the Medicare Part D study was supported by grant money given to Johns Hopkins University by Arnold Ventures. Mr. Burton reported no relevant financial disclosures.
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The Generic Drugs Industry Responds to COVID-19 Add generic drug shortages to the long list of practice challenges that have emerged during the COVID-19 pandemic, according to several industry experts. From the beginning, the pandemic threatened to trigger a huge disruption in the generic drug industry’s worldwide operations. “There were very big concerns about how our industry would be impacted, particularly when certain countries were closed off to the rest of the world,” said Erik Komendant, the vice president for federal government affairs at the Association for Accessible Medicines (AAM), the organization that represents the generic industry. “The challenge initially was that everyone was shutting down,” Mr. Komendant said. “And when the world basically shut down air transportation, it required our members to reroute their supply chains and distribution.” Initial fears that the pandemic would stem the flow of critical generic medicines turned out to be overstated. “We didn’t see the type of impact that we saw with PPE [personal protective equipment], for example,” Mr. Komendant said. In fact, the association looked at generic order fulfillment rates during the pandemic, according to Craig Burton, the vice president for policy at AAM. Although he said there was a “small dip” in wholesaler supplies of generic drugs early in the crisis, it “quickly recovered, and now you have inventory and order fulfillment rates that are at or above historic norms.”
A Manufacturer’s Take For Dr. Reddy’s Laboratories, the closures meant coming up with novel strategies for COVID-19–instigated production and distribution vulnerabilities. Milan Kalawadia, MBA, the senior vice president and head of U.S. operations, said lockdowns and curfews in some Indian states threatened to curtail staff travel to and from the company’s multiple manufacturing facilities. “Our supply chain team jumped in and figured out solutions on all fronts,” Mr. Kalawadia said. “We worked with local governments to ensure our staff had the ability to get to work.” Factory hours were altered to ensure production lines were adequately staffed. “We typically run three eight-hour shifts a day, seven days a week,” he said. “We modified that to two 12-hour shifts on a seven-day period.” The change meant staffs could travel to and from their worksites outside of curfew hours. Mr. Kalawadia also said the team “looked at our systems to minimize some of the staffing needs, while also optimizing manufacturing output. We learned
that we could optimize our systems and processes, resulting in the same output with less staff. So, we became more efficient throughout COVID-19.” The supply chain team met the airline travel restriction challenge by securing
airfreight carrier space for the company’s products. “Our costs did increase,” Mr. Kalawadia said, “but it ensured having product here on the ground in the U.S. and that we were servicing our patients’ needs.” Mr. Kalawadia said the pandemic had a negative impact on injectable sales, prompted by the more than 20% decrease in hospital elective surgeries. “But from our supply perspective,” he
added, “we did ensure the supply of critical medicines.” The company also just launched a new e-commerce site called Dr. Reddy’s Direct, where hospital pharmacies and other customers will be able to purchase products outside of traditional channels. —Bruce Buckley The sources reported no relevant financial disclosures.
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Specialty Pharmacy Continuum • July/August 2021
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PA Hassles Persist continued from page 1
33 million members nationwide so they would have confidence of additional medicines on hand should their pharmacy run into supply issues,” said David Lassen, PharmD, the chief clinical officer at Prime. “We did not observe delays in members receiving needed medicines; in fact, we saw the opposite. There was an increase in claims use indicating members had ample supply of their medicines. As restrictions lifted across the country and clinic and hospital visits returned to normal, we returned to preCOVID practices and our claims patterns also returned to pre-COVID levels. Prime continuously reviews all utilization management practices to help ensure members have a positive overall experience while keeping costs manageable.”
A Full-Time Job The AMA survey found that medical practices complete an average of 40 PAs per physician, per week, requiring the equivalent of two business days (16
that they had had an interruption in their therapy, most commonly related to insurance issues,” she said. (The results were presented at the American Thoracic Society International Conference, in May 2019; poster A5095.) “Roughly 35% of them reported that it took a month or longer to get started on their medications due to insurance or pharmacy-related issues. I recently had a patient who went two months before getting on therapy because of PA and delivery delays. When you’re measuring life span in weeks or months without treatment, those weeks matter. Time is of the essence for these patients.” She agreed that PA requirements were not relaxed during the height of the pandemic. “No one was cutting any slack,” she said. “In some ways, it felt worse because we were having so many access issues with the restrictions on distribution of most PAH therapies. In fact, lately PAs and other formulary issues feel exacerbated. The nurses I work
‘I understand and appreciate the points of view of all the stakeholders: Payors want to ensure that we have responsible and appropriate medication utilization and drug spend, but this process shouldn’t be overly burdensome. And none of us want physician practices to have one or more full-time employees dedicated to just managing the PA process.’ —Sheila Arquette, RPh hours) of physician and staff time. To keep up with the administrative burden, two of five physicians (40%) employ staff members who work exclusively on tasks associated with PA. None of these findings were surprising to Rebekah Hanson Anguiano, PharmD, a clinical assistant professor of pharmacy practice and clinical pharmacist for ambulatory pharmacy services at the University of Illinois at Chicago College of Pharmacy, part of UI Health. “Our experience is very similar. I work primarily in specialty pharmacy, and it is usually a shocker if we prescribe a medication that does not require a PA. We assume it’s a mistake and take steps to double-check that the claim was processed correctly if that happens.” In a small survey of patients with pulmonary arterial hypertension (PAH), a rare, progressive disorder leading to right heart failure and ultimately death if untreated, Dr. Anguiano and her colleagues sought to characterize the patients’ burdens related to medication accessibility. “The survey was among only 26 patients, but 54% of them said
with are talking about this all the time, having multiple patients experiencing delays getting their treatments authorized or having to switch to new ones.”
Little Time for Patient Care One of the UI Health specialty clinics for PAH has at least one nurse whose time primarily is spent on PA-related activities, with very limited time for patient care. “We are trying to find some institution-wide solutions to the problem, such as piloting a central PA service,” Dr. Anguiano said. “That’s a common trend in health systems. The burden of PA resulting in interruptions to patient care has gotten so bad that it has to be a dedicated service.” The American Hospital Association (AHA) documented multiple examples of such cases in a 2020 white paper, including a 17-hospital system that spends $11 million annually complying with health plan PA requirements and a 355-bed psychiatric facility that has 24 full-time staff dealing with PAs. “As a former payor, I administered the PA program for my health plan,” said
Sheila Arquette, RPh, the president and CEO of the National Association of Specialty Pharmacy (NASP). (Prior to joining NASP, Ms. Arquette was the director of pharmacy services for the Western New York health plan Independent Health.) “We would look at the percentage of PAs approved for medications covered, and determine if those PAs were still required based on a host of criteria. I understand and appreciate the points of view of all the stakeholders: Payors want to ensure that we have responsible and appropriate medication utilization and drug spend, but this process shouldn’t be overly burdensome. And none of us want physician practices to have one or more full-time employees dedicated to just managing the PA process.”
PBM Acknowledges Problem Even some PBMs agree that the PA process must be reformed. “Frankly, I’m on the side of the doctors here. The PA process doesn’t work for anyone,” said Jason Borschow, the founder and CEO of Abarca, a PBM based in Aventura, Fla. “Our view on this is, ‘stop the madness.’
Some PAs are nonsensical, just administrative burdens with no clinical or financial benefit to anyone. I’d like to believe that is the minority. But if your PA is going to be approved 95% of the time, just get rid of the PA. And there’s no reason this process should take days or weeks. You should be able to get an answer in minutes.”
What Can Be Done? Standardization efforts and systems such as electronic PA (ePA)—an electronic process established in the National Council for Prescription Drug Programs (NCPDP) SCRIPT Standard that enables patient and drug-specific PA criteria and real-time approval—could streamline the process and ease burdens on patients and providers. Results from a pilot project led by America’s Health Insurance Plans (AHIP), released in March 2021, showed that AHIP’s Fast Prior Authorization Technology Highway (Fast PATH) ePA system reduced numbers of phone calls and faxes, made it easier to understand PA requirements, and, most importantly, reduced care delays. The study found that 71% of experienced providers who implemented ePA reported faster time to patient care; it also found ePA reduced the time between submitting a PA request and receiving a decision from the health plan by 69%. “The adoption of ePA is still quite a lot lower than we would expect it to be, however, given the benefits it offers,” said Mr. Borschow, who is a member of the Specialty Pharmacy Continuum editorial advisory board. “Most, if not all, leading
Specialty Pharmacy Continuum • July/August 2021
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One 17-hospital system spends
$11 million annually complying with health plan PA requirements, and a 355-bed psychiatric facility has
24 full-time staff dealing with PAs.
could lead to more consistency across the board and improve ePA adoption,” Ms. Arquette said.
‘Inform, Not Overrule’ Mr. Borschow said widespread ePA use would solve a significant portion of the PA problem. “Not all of it, but a good part of it. Prior authorization is an important tool, but you want to get the information to the provider about the benefit the patient is entitled to before they’ve gone through the decision-making process,” he said. “We want to inform that decision, not overrule it once it’s already made, and that means getting the information to the prescriber in real time.” He also reiterated that many PAs are unnecessary. “Do we have enough information already on the patient, where we should be able to intelligently auto-effectuate that PA before it even comes up without making the doctor answer a lot of additional questions?” he asked. “Abarca is researching how we can use artificial intelligence to use the long history of data we have on patients to predict the right answer for a PA. We believe AI
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‘I recently had a patient who went two months before getting on therapy because of PA and delivery delays. When you’re measuring life span in weeks or months without treatment, those weeks matter. Time is of the essence for these patients.’ —Rebekah Hanson Anguiano, PharmD EMRs [electronic medical records] have integrated ePAs that can connect to PBMs and health plans, but not all physicians and EMRs are as advanced in this regard. And there are cases where the PBM or health plan on the other side is unable or unwilling to implement ePA.” Standardization is also elusive. “In general, provider advocacy has long attempted to try to standardize PA processes, with little success on the national level and limited success on the state level,” Ms. Arquette said. “What’s difficult is that every payor and every PBM has different criteria, which makes it hard for providers to keep up.”
Medicare Eyes ePA Mandate Beginning in January 2022, all Medicare Part D plans will be required to have ePA capability for processing PA requests using a single standard, the NCPDP SCRIPT Standard version 2017071. (Medicare Advantage plans, however, are not included in the requirement, a point of contention for providers.) “With Medicare standardizing these criteria across Part D plans, that
can facilitate administrative simplification that allows us to reduce the burden on providers.”
Reforms Proposed The AMA, AHA and other stakeholders, including the American Pharmacists Association, have proposed a series of PA reform principles to “ensure that patients have timely access to treatment and reduce administrative costs to the health care system.” In addition to the use of standardized ePAs, they also called for detailed explanations for PA override denials, such as indication of any missing information; standardized review criteria across the industry; and public disclosure of patient-specific utilization management requirements, including PA, step therapy and formulary restrictions, with patient cost-sharing information, applied to individual drugs and medical services.
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Stakeholder transparency can help
Finding a Safe Harbor From Federal Scrutiny Ensuring the way that pharmaceutical manufacturers work with specialty pharmacies, advocacy groups and patients is transparent and fair can help keep companies away from federal scrutiny and expensive legal challenges, a panel of legal experts said during Informa Connect’s 2021 Compliance Congress for Specialty Products, held virtually. Manufacturers may choose to enter limited distribution network contracts with specialty pharmacies for particular products, knowing these pharmacies provide a high level of expertise and support to patients, according to Katherine Churchill, JD, a counsel of global trade controls and specialty pharma for Pfizer. These contracts can include enhanced services for specialty drugs, such as patient and prescriber outreach, Ms. Churchill noted. But the contracts may not be duplicative of core services already offered by the pharmacies, to avoid violating federal Anti-Kickback Statute (AKS) and applicable safe harbor regulations. “You have to make sure when interacting with specialty pharmacies, there’s no knowing or willful incentivization to purchase or to prescribe a particular therapy, because the Department of Health and Human Services wants to minimize fraudulent claims under federal health care programs,” Ms. Churchill said. “The same issues that give rise to an Anti-Kickback violation also give rise to a False Claims Act violation. The penalties can be quite severe, and with good reason. You have to be very careful that when a manufacturer is contracting with a specialty pharmacy to provide these services that we’re doing it the right way, and we’re not incentivizing prescriptions or doing anything that would change the independent clinical judgment of the specialty pharmacy.” Manufacturers can work within the AKS’s Personal Services safe harbor with specialty pharmacies to provide increased patient support, she said, provided that specific guidelines and criteria are satisfied to be compliant. This includes guidance on how an agreement is negotiated and what is memorialized in writing. Important elements include the fair market value for bona fide services that does not consider volume or incentivizing prescribing. It has to be for a commercially reasonable business purpose, and it has to be very clear what services will be provided and what that estimated total fee looks like. It can be tricky to stay within the safe harbor, Ms. Churchill added, “and when it goes wrong, it can go really wrong.” She cited, as an example, a 2015 case against Novartis, brought by the U.S. District
Attorney’s Office, Southern District of New York. Novartis was cited for AKS and False Claims Act violations for specialty drugs, including deferasirox (Exjade) and mycophenolic acid (Myfortic). The company was distributing the medications through a defined network of specialty pharmacies. Allegedly, when sales were less than projected, nurses within the pharmacies then called patients who had fallen off the drugs to incentivize them to restart their therapies, according to details of the case released by the district attorney’s office (bit.ly/3C9B7JC). The company then allegedly rewarded the pharmacy by channeling patients who were more likely to have higher medication adherence to this pharmacy, thereby incentivizing both prescribing behavior and increased volume, Ms. Churchill said. The final settlement was for $370 million, along with a $20 million forfeiture of proceeds from the scheme. Ms. Churchill emphasized the need for companies to be transparent. It has to be clearly disclosed that enhanced services are being funded by a manufacturer, and it’s important that services only be offered after a patient has received a prescription.
Core Versus Enhanced Services Manufacturers have taken different approaches to working with specialty pharmacies, said Rahul Khara, PharmD, JD, the chief compliance officer for Acceleron Pharma. Some think of specialty pharmacy core services as solely dispensing product, so enhanced services can be defined as anything above that. However, others note that specialty pharmacies offer a range of additional services across the board. In such cases, enhanced services would have to be defined as anything on top of those offerings. “Even among two to three pharmacies, there may be differences in the contracts on what is considered core versus enhanced services, and smaller manufacturers may have very little to no leverage to be able to edit the contracts,” Dr. Khara said. “Pharmacies are generally open to things like anti-switching [programs], but they’re going to be very squirrelly when you start to fiddle with services because that’s how they make a lot of their money.”
When building a distribution network, it’s important for manufacturers to set expectations for what they consider core services, Ms. Churchill added. If a benefits investigation is important to the company, and the specialty pharmacy requests extra fees for that, the manufacturer can tell the pharmacy they don’t meet the requirements for inclusion in the distribution network.
Travel Assistance Travel assistance to receive medication therapy is another tricky area of compliance for manufacturers, said panelist Lisa Shrayer, JD, the head therapeutics counsel, North America, for Vertex Pharmaceuticals. However, the Office of the Inspector General (OIG) made an exception in a 2020 opinion for one manufacturer for an immunotherapy used for acute lymphoblastic leukemia in patients up to age 25. The manufacturer proposed reimbursing patients for gas and tolls, or transportation for a patient and a caregiver to and from the closest center; reimbursing for modest lodging near the center during treatment and post-treatment monitoring; and out-of-pocket expenses of up to $50 per person per day to cover items such as meals. The program was designed for patients with financial need who live more than two hours from the nearest treatment center and no insurance for nonemergency medical travel. OIG decided not to impose sanctions on the manufacturer for AKS violations because the program provided greater access to care for lower-income and rural patients. The agency also allowed a limited number of physicians to prescribe the therapy, noting that the drug is a one-time potentially curative treatment and the financial assistance would not otherwise be available from another source.
Why Specialty Is Special When collaborating with manufacturers and payors, specialty pharmacies have several key areas of differentiation that can help all parties steer clear of regulatory trouble. These include skilled resource activity, payor utilization management diligence and funding assistance, commented Cheryl Allen, BSPharm, MBA,
the executive vice president of business development and industry relations for Asembia. “If the pharmacy is providing prior authorization and appeals support across all of its programs in a defined area, this payor utilization management support is a core service for that pharmacy,” Ms. Allen said. “In situations where a manufacturer expects a large percent of payor utilization management for a product, it is very important to understand how potential specialty pharmacy dispensing channel entities define prior authorization and appeals relative to core versus enhanced services.” Pharmacies that enter these enhanced services contracts generally have added expertise in a specific area, such as oncology, and additional certification from organizations such as URAC, Ms. Allen said. A pharmacy’s core services likely will be spelled out in policies and procedures that are required by accrediting bodies, she noted.
Product Preferencing Product preferencing programs also can be fraught, Ms. Allen noted. “Generally, it’s not pharmacists supporting one product over another for products in a competitive space,” she said. “More often, it’s the payor preferencing certain products for their covered patients.” Ultimately, all pharmacies are required by state law to appoint a pharmacist-in-charge who is responsible for ensuring adherence to legal requirements and that pharmacist in their professional judgment is acting in the patients’ best interest. Overall, specialty pharmacies entering manufacturer contracts need to ensure any fees received are in exchange for providing bona fide services that meet CMS’ four-pronged test, Ms. Allen said. They must be of fair market value, performed on behalf of the manufacturer and would otherwise be performed by a manufacturer. No fees may be passed through to clients or customers. —Karen Blum The sources reported no relevant financial disclosures other than their stated employment.
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Time to Heed OIG Guidance on Rx Discounts Pharmaceutical manufacturers looking to help patients gain access to expensive drug and medical treatments need to tread lightly and heed guidance from the Department of Health and Human Services’ Office of Inspector General (OIG), according to panelists speaking at Informa Connect’s 2021 Compliance Congress for Specialty Products, held virtually. Otherwise, they could be at risk for legal action. OIG for decades has provided guidance to providers and pharmacies about waiving patients’ cost-sharing obligations, and this provides a good foundation for the government’s view on copays and deductibles, said Matthew Wetzel, JD, the former associate general counsel and chief compliance officer for Grail Inc., during a session on best practices related to patient assistance programs (PAPs), copays and foundations. “OIG essentially says that waiving copays and cost sharing takes away a natural economic control on health care costs,” Mr. Wetzel said. “When patients have ‘skin in the game,’ they make more measured and reasonable approaches to their care, and they might not simply have a knee-jerk reaction to go to the most expensive treatment.” However, manufacturers are aware that waiving patient cost shares can provide more immediate access to therapies, as well as encourage adherence to medications, said Mr. Wetzel, now a partner in the Healthcare and Life Sciences division at Goodwin Procter LLP, in Washington, D.C. It’s fine to do so when following all applicable criteria and guidance from OIG, he noted. “It sounds pretty straightforward; you just have to follow the rules and you won’t find yourself at risk,” said Darren Jones, a principal and global life sciences advisory leader at Baker Tilly, in New York City. However, “we all very well know that the application of some of this is much more difficult.” There is a permitted place to support those who have a difficult time affording drugs, he said. But manufacturers must make sure the intent behind the programs being run “is crystal clear.” Corporate PAPs may provide free product for low-income, uninsured and commercially underinsured patients, but it must be based on established criteria, such as income level, Mr. Jones said. Medicare patients may be eligible only if the program operates totally outside the Medicare benefit. Medicaid patients may be eligible only if there is no Medicaid coverage for the drug. He also cautioned against providing free product to or for: • non-U.S. citizens or residents;
• those who do not satisfy economic thresholds; • Medicare patients who are within the Medicare benefit; • Medicaid patients who already have coverage for the drug; and • off-label uses. Manufacturers also may choose to make donations to charitable PAPs operated by 501(c)(3) organizations for individuals with particular medical conditions. Such programs must operate outside the control of the company, Mr. Jones noted. He added that a manufacturer’s committee making donation decisions should exclude representatives from sales and marketing. To operate within permitted rules, make sure there is no advertising or suggestions that someone reach out to a charitable foundation.
Moreover, do not provide websites for a charitable foundation without being asked, and do not cover product for off-label uses. In addition, the company may not influence the charity’s operations, including defining the disease categories that it will cover. “We have to make sure commercial representatives that are engaged to ensure patients have access to our products stay within a very defined set of parameters,” Mr. Jones said. It’s important that the workflows of the manufacturer and charity are not linked—a caution that is based on past enforcement actions. Indeed, a company’s influence on a charity’s operations has been “the longest-serving area of enforcement around patient support programs and independent charities, and it still remains an area of concern,” he said. Copay programs operated by manufacturers that provide commercially insured patients with assistance to reduce their out-of-pocket costs may not extend assistance to people covered by government insurance programs, Mr. Jones added. In addition, they must implement controls to verify a patient’s eligibility and cannot provide copay
4 Manufacturer/Payor Best Practices 1. PAPs, copay programs and foundations should maintain excellent records of any products provided to patients.
2. Manufacturer team members should not encourage individuals to stay on therapy by promising gifts or sharing information on overcoming side effects.
3. Manufacturers and third-party vendors should not provide certain information, including clinical justification on prior authorization forms or appeal-letter templates.
4. Manufacturers should avoid providing additional services, such as family and patient counseling and emotional support, or helping physician offices with data entry, filing and training.
support for off-label uses. Foundations operated by pharmaceutical companies must take care to be transparent and accountable regarding donations provided. They must ensure that drug copayments are not being disguised as routine charitable donations, and conduct due diligence to verify that foundations are operating separately from their donors. In the specialty disease space, single-donor foundations are prevalent, he noted, so it’s important that the supporting manufacturer not exert economic pressure on that foundation and be extremely hands-off. “It’s not just about any one individual donation or program; it’s more of a comprehensive look at all controls in place,” Mr. Wetzel said. “Are you following them, and do those controls balance out the fraud and abuse risk sufficiently to allow an industry donation in a way to help patients but keep the fraud risk minimal?”
Advice for Foundations OIG has laid out clear guidance under which they expect independent foundations to operate, commented Alan Klein, MBA, the chief development officer for the HealthWell Foundation, an independent, nonprofit charitable organization providing financial assistance to patients in more than 80 disease areas. The guidance states that foundations must maintain their independence from all donors, especially corporate donors, he said, and also limits to what extent foundations can communicate with supporters. Mr. Klein is the point person who communicates with corporate donors, and HealthWell only provides reporting information on its funds in aggregate to its donors. “We basically have compliance as the cornerstone of all activities, because we don’t want to do anything that would jeopardize any of the services we provide,” he said, adding that last year, the foundation helped more than 160,000 patients. HealthWell has multiple funds that help cover medications and treatments for conditions such as hepatitis C, acute myeloid leukemia and cystic fibrosis, Mr. Klein said. Pharmaceutical manufacturers tend to support funds for disease states for which they have stakeholders, he added, but once they distribute those funds, “it’s like throwing money over a fence. We are left to dole it out compliantly on a first come, first served basis; they have no say-so.” —Karen Blum The speakers reported no relevant financial disclosures other than their stated employment.
20
Specialty Pharmacy Continuum • July/August 2021
POLICY
Debate continues over efficacy, value of aducanumab
Some Stakeholders Put the Breaks on AD Drug Nearly two months after the FDA’s controversial decision to approve aducanumab (Aduhelm, Biogen) for the treatment of Alzheimer’s disease, payors are still determining how they will cover the drug, while hospitals and health systems grapple with decisions about which patients, if any, should be offered the amyloid plaque–targeting agent. Meanwhile, two congressional committees—the House Committee on Oversight and Reform and the Committee on Energy and Commerce—have launched an investigation into the approval process and pricing of the drug, which at its highest dose costs over $4,300 per infusion, or about $56,000 annually. The FDA is also taking action: The agency recently called for an Office of Inspector General investigation into its own process, and has narrowed the indication for the medication to address concerns over the broad scope of the original labeling. On July 12, the Centers for Medicare & Medicaid Services (CMS) joined the fray when it announced that it was opening a
“Our goal at the end of this process is to provide the American public with clear, evidence-based decisions,” said Tamara Syrek Jensen, the director of the coverage and analysis group at CMS’s Center for Clinical Standards and Quality. In an earnings call on July 22, Biogen executives reported that some Medicare Advantage plans already have approved prior authorizations for aducanumab. “For the MACs, due to the miscellaneous coding, it does take them a little bit of time to process the
Only about
35% of the estimated
900 clinical sites with the capacity to deliver aducanumab have completed a pharmacy and therapeutics review for the drug or won’t require one.
national coverage determination (NCD) analysis to assess whether Medicare will establish a national Medicare coverage policy, not just for aducanumab but also for “any future monoclonal antibodies that target amyloid for the treatment of Alzheimer’s disease.” Two public “listening sessions” were held on July 22 and 27, and CMS said a proposed decision was expected within six months and a final within nine months. In the meantime, coverage determinations for aducanumab will continue to be made at the local level by the Medicare Administrative Contractors (MACs) in 12 jurisdictions across the country, according to CMS. (Any NCD, if adopted, would override regional MAC decisions.)
[aducanumab] claims, but we are also aware that MACs have received claims already,” said Alisha Alaimo, the president of Biogen’s U.S. organization. Several commercial plans announced that they will not cover aducanumab, including Blue Cross Blue Shield affiliates in Florida, Kansas, New York, Michigan, North Carolina and Pennsylvania. Policies posted online indicate that these payors are denying coverage because they consider the drug “investigational” or “experimental” or because “a clinical benefit has not been established.” Prime Therapeutics, a pharmacy benefit manager owned by 19 Blue Cross Blue Shield plans, said the decision about aducanumab coverage will fall to those individual plans. “Given that Aduhelm
is an IV infusion, Prime has deemed it out of scope for pharmacy benefit coverage and will not cover it on formulary,” April Kunze, PharmD, the senior director for clinical formulary development & trend management strategy, told Specialty Pharmacy Continuum. “Since it is a medically administered drug, the decision to cover will fall to individual Blues Plans. Those decisions will be reflected in the plans’ medical policies.” On July 23, Point32Health (a merger of Tufts Health Plan and Harvard Pilgrim Health Care) announced it would not cover aducanumab. Point32Health’s Claire Levesque, MD, also spoke during one of the Medicare NCD listening sessions, urging CMS to view aducanumab as an investigational drug and not provide unilateral coverage. She noted that she had lost her own husband to Alzheimer’s disease just before the COVID-19 pandemic. “This is a terrible disease, and I want a cure for my family, for other families and for Point32Health,” said Dr. Levesque, formerly an assistant professor of neurology at Boston University Medical Center. “But [aducanumab] has unproven benefits and high risk. We don’t have enough information from Biogen about what happened to the individuals in the trials who experienced [amyloidrelated imaging abnormalities]. Even very small areas of bleeding or swelling can lead to real declines in function for people. We also don’t know how long these side effects persisted. A better understanding is critical.” Some commercial payors have announced coverage for aducanumab with required prior authorization, including Highmark Blue Cross Blue Shield and Blue Cross Blue Shield of Michigan, while other plans are biding their time. In its own July 15 earnings call, representatives of UnitedHealth Group said the company was looking to Medicare for guidance. “There’s still a lot of information that we need to be able to make really clear decisions, and I think we’re not the only ones in that regard looking for greater clarity in this arena,” CEO Andrew Witty said.
Health Systems Push Back At least three major hospital systems—Cleveland Clinic in Ohio, Mount Sinai Health System in New York, and Providence Health in Washington state—announced they will not provide aducanumab, although affiliated physicians could, at least in theory, prescribe it for their patients to receive it elsewhere. The Neurology Center, a leading independent neurology group with seven locations in Washington, D.C.,
and Maryland, also announced that it would not be providing aducanumab “at this time.” In the July 22 earnings call, Biogen reported that only about 35% of the estimated 900 clinical sites identified as having the capacity to deliver aducanumab (including amyloid testing, MRI monitoring and neurological diagnosis) had completed a pharmacy and therapeutics review or won’t require one. Washington University in St. Louis still plans to offer the drug to appropriate patients “once coverage is established,” said Erik Musiek, MD, PhD, a professor of neurology at Washington University School of Medicine in St. Louis. “No one here has administered the drug or has tried to convince insurance to pay for it yet.” Biogen has pushed back against the aducanumab outcry. The company presented four posters at the Alzheimer’s Association annual conference, held virtually and in Denver from July 26-30, tying the drug to several markers of improvement. And on July 22, Biogen posted an open letter defending the FDA’s decision-making process. “We recognize that Aduhelm’s dataset was complex and its journey to this point did not follow a conventional path. But the road to innovation is rarely straightforward, and Aduhelm is not an exception. We stand behind the clinical evidence provided by the studies and the data-driven scientific approach taken.”
A Narrowed Indication As for the new, narrowed scope of the aducanumab label, it now states that “treatment with Aduhelm should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. There are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied.” Gordon Smith, MD, a neurologist at Virginia Commonwealth University (VCU), in Richmond, welcomed the updated label. However, “it doesn’t really change how we are viewing this drug,” he told Specialty Pharmacy Continuum. VCU is reviewing how and to whom it will provide aducanumab. “I don’t know what the outcome of our review process will be,” Dr. Smith said. “But I can say that treating moderate to advanced Alzheimer’s patients with aducanumab was never on the table.” —Gina Shaw Dr. Musiek has research funding from the NIH. Drs. Kunze and Smith reported no relevant financial disclosures beyond stated employment.
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22
Specialty Pharmacy Continuum •July/August 2021
CLINICAL
Gene Therapy Series, Part 1:
The New Frontier of Hemophilia Treatment ADRIENNE BRENNAN, PHARMD, CSP Clinical Program Manager AllianceRx Walgreens Prime Pittsburgh, Pennsylvania
H
emophilia is a genetic bleeding disorder caused by absent or defective clotting factor, leading to spontaneous or excessive bleeding
after injury.1 Hemophilia is an X chromosome–linked
disorder expressed in males, although females are carriers and can have the disease in rare cases.1 It is estimated that between 20,000 and 33,000 people in the United States have hemophilia.1-3 Although most people with hemophilia inherit it from a family member, approximately one-third of cases are caused by a spontaneous genetic mutation in those with no family history.1 There are 2 main types of hemophilia: hemophilia A, a deficiency of factor VIII, and hemophilia B, a deficiency of factor IX; hemophilia A is approximately 4 times more common than hemophilia B.2,3 Signs and symptoms are the same regardless of which clotting factor a person is missing and include bleeding in joints or muscles, bruising, and frequent nosebleeds.1 The severity of disease depends on the amount of clotting factor circulating in the body, with cases classified as mild, moderate, or severe.1-3
Current Treatments Since the mid-1990s, the mainstay of hemophilia treatment has been IV infusions of recombinant clotting factor replacement, administered prophylactically or as needed when a bleed occurs.4-5 The earlier versions of clotting factor products require infusion 2 to 3 times per week, but more recent advancements have brought to market products with longer half-lives that extend the time between infusions as well as a bispecific antibody product that mimics the function of factor VIII rather than directly replacing it.5 Up to 20% of
patients will develop antibodies to factor replacement products, known as inhibitors.1,6 These patients must be treated with higher and more frequent doses of a traditional factor replacement product, a bypassing agent, or a bispecific antibody product.6 A summary of current treatment options is provided in Table 1. Regardless of the factor product selected, the annual cost of treatment for hemophilia can be as much as $1 million per patient.7
Future Treatment Option: Gene Therapy Although clotting factor replacement therapy has been very effective for the management of hemophilia, gene therapy offers exciting potential to cure this lifelong disease. The underlying nature of hemophilia makes it a perfect candidate for gene therapy. First, it is caused by a known single gene mutation. This is preferable to diseases caused by an array of genetic mutations, which currently cannot be addressed by gene therapy. Second, curing the disease requires the addition of a gene, as opposed to the more complicated process of silencing or turning off a gene. Third, even small increases in protein production (in this case, clotting factor) resulting from gene therapy can have a significant effect on the course of the disease. It is for
Gene and Cell Therapy Defined Gene and cell therapy (GCT) is an exciting new area of medicine offering the potential to treat, or even cure, diseases that previously may have had limited or no treatment options. These therapies treat or cure disease by introducing cells or DNA to address the underlying cause of a disease. Cell therapy involves removal of cells from either the patient (autologous) or a donor (allogeneic), modification of those cells outside the body, and reinfusion of the modified cells back into the patient to treat the disease.1 The type of cell used depends on the specific treatment. Gene therapy is the delivery of genetic material via a vector, usually viral in origin, to replace, deactivate, or repair faulty DNA that is causing a disease.1 Use of a viral vector allows the new genetic material to be inserted directly into a patient’s own DNA, thereby enabling proper protein production to lessen the effects of or completely cure a disease. This is the part 1 of a series describing the potential of gene therapy for various disease states. 1. American Society of Gene and Cell Therapy. Gene and cell therapy FAQs. Accessed July 28, 2021. https://www.asgct.org/education/more-resources/gene-and-cell-therapy-faqs
these reasons that the hemophilia gene therapy pipeline is relatively crowded compared with that for other diseases.7 As of July 2021, there are 3 gene therapies for hemophilia A and 2 for hemophilia B in latestage development.8 These products are summarized in Table 2. Gene therapies for hemophilia are intended to be one-time IV infusions. All the products in late-stage development use a recombinant adeno-associated viral (AAV) vector to deliver a functional copy of the F8 or F9 gene to a patient’s liver cells, which is where natural production of factors VIII and IX occur. After successful uptake of the new functioning gene, a patient should begin to produce levels of factor VIII or IX that are sufficient to prevent bleeding episodes and potentially eliminate the need for exogenous factor replacement. The various pipeline products are differentiated by the proprietary technology each company uses to engineer viral vectors and gene cassettes for ideal uptake and gene expression.9,10 Data from phase 3 trials are limited, but those that have been published suggest that these therapies are capable of increasing
natural factor production in patients for at least several years after administration, resulting in significantly reduced annualized bleed rates and a decrease in or total elimination of factor infusions.10 It is widely expected that hemophilia gene therapies will enter the US market starting in 2022. There was hope that valoctocogene roxaparvovec (Roctavian, BioMarin) would gain FDA approval in August 2020; however, the FDA issued a Complete Response Letter requesting that BioMarin conduct additional clinical trials with 2 more years of data.11
Special Considerations for Hemophilia Gene Therapy Once gene therapy for hemophilia is available to patients in the United States, it will be important to determine the ideal candidates for treatment. Clinical trials so far have been limited to patients who have moderate or severe disease and no inhibitors or autoantibodies. This could significantly limit the pool of eligible patients, with exclusion criteria related to inhibitors alone making up to 30% of hemophilia A patients and 3% of hemophilia B patients ineligible for gene therapy.10 Even
23
Specialty Pharmacy Continuum • July/August 2021
CLINICAL
Table 1. Approved Therapies for Hemophilia A and B Medication
Table 2. Phase 3 Gene Therapies For Hemophilia A and B
Brand Name, Company
Standard-acting factor VIII Antihemophilic factor/von Willebrand factor complex (human)
Alphanate, Grifols
Product
Delivery Method
Indication
Giroctocogene fitelparvovec (Pfizer)
AAV vector
Hemophilia A
SPK-8011 (Spark Therapeutics/Roche)
AAV vector with B-domain deleted human factor VIII gene
Valoctocogene roxaparvovec (Roctavian, BioMarin)
AAV VIII vector
Etranacogene dezaparvovec (uniQure/CSL Behring)
AAV5 with FIX-Padua mutant
Fidanacogene elaparvovec (Spark Therapeutics/Pfizer)
AAV vector
Humate-P, CSL Behring Antihemophilic factor (human)
Hemofil M, Baxter Koate, Kedrion
Antihemophilic factor (recombinant)
Advate, Takeda Kogenate FS, Bayer Kovaltry, Bayer Novoeight, Novo Nordisk Nuwiq, Octapharma
Hemophilia B
AAV, adenovirus-associated vector
Recombinate, Baxalta Xyntha, Pfizer Von Willebrand factor/coagulation factor Wilate, Octapharma VIII complex (human) Long-acting factor VIII Antihemophilic factor (recombinant) PEGylated
Adynovate, Takeda
Antihemophilic factor (recombinant), single chain
Afstyla, CSL Behring
Antihemophilic factor (recombinant), Fc fusion protein
Eloctate, Bioverativ
Antihemophilic factor (recombinant), glycopegylated-exei
Esperoct, Novo Nordisk
Antihemophilic factor (recombinant) PEGylated-aucl
Jivi, Bayer
Standard-acting factor IX Coagulation factor IX (human)
Alphanine, Grifols Mononine, CSL Behring
Factor IX complex
Profilnine SD
Long-acting factor IX Coagulation factor IX (recombinant), Fc fusion protein
Alprolix, Sanofi Genzyme
Coagulation factor IX (recombinant), glycoPEGylated
Rebinyn, Novo Nordisk
Coagulation factor IX (recombinant)
Benefix, Pfizer Idelvion, CSL Behring Ixinity, Medexus Rixubis, Takeda
Bispecific antibody Emicizumab-kxwh
Hemlibra, Genentech
Bypassing agents Anti-inhibitor coagulant complex
Feiba VH, Takeda
Coagulation factor VIIa (recombinant)
Novoseven RT, Novo Nordisk
if a patient’s disease characteristics seem amenable to gene therapy, it is difficult to predict to what extent they will respond and produce factor even after receiving a functional F8 or F9 gene.9 Risks, side effects, and long-term implications of therapy will need
to be considered by each patient offered gene therapy for hemophilia. There have been rare cases of immune reactions after administration of AAV-vector gene therapy; such reactions may need to be medically managed.9 Although data have shown these gene therapies
to be effective for a number of years, experience and data to predict whether they will remain effective for a patient’s entire life are lacking.9 It also is unclear whether a patient could be treated with an alternative therapy if gene therapy is ineffective. Cost will be an additional important consideration. Before the FDA’s Complete Response Letter, a perpatient price of $2 million to $3 million was being considered for valoctocogene roxaparvovec, which would have made it one of the most expensive drugs in the world.7 Although a $2 million price tag seems astronomical, if a gene therapy could eliminate the need for routine factor replacement, it may actually be more cost-effective given the high cost of factor products. In fact, an analysis by the Institute for Clinical and Economic Review determined that even at a price of $2.5 million, valoctocogene roxaparvovec would be cost saving compared with standard factor VIII replacement.12
Role of the Specialty Pharmacist Today, specialty pharmacists are important members of hemophilia patient care teams. At AllianceRx Walgreens Prime, our specialty pharmacists help ensure patients always have on hand an adequate supply of factor product and any supplies necessary to administer therapy. Our specialty pharmacists educate patients about proper medication administration and storage and are well versed in techniques to manage bleeding. As treatment models for hemophilia patients shift away from routine factor replacement and toward gene therapy, specialty pharmacists will remain a vital resource, providing disease management and education as well as long-term outcomes monitoring.
References 1.
CDC. What is hemophilia? Accessed June 16, 2021. https://www.cdc.gov/ncbddd/ hemophilia/facts.html
2. National Hemophilia Foundation. Hemophilia A. Accessed June 16, 2021. https://www.hemophilia.org/bleedingdisorders-a-z/types/hemophilia-a 3. National Hemophilia Foundation. Hemophilia B. Accessed June 16, 2021. https://www.hemophilia.org/bleedingdisorders-a-z/types/hemophilia-b 4. National Hemophilia Foundation. History. Accessed June 16, 2021. https://www. hemophilia.org/bleeding-disorders-a-z/ overview/history 5. CDC. Treatment of hemophilia. Accessed June 16, 2021. https://www.cdc.gov/ ncbddd/hemophilia/treatment.html 6. CDC. Inhibitors and hemophilia. Accessed June 16, 2021. https://www.cdc.gov/ ncbddd/hemophilia/inhibitors.html 7. Fidler B. Gene therapy for hemophilia: so close, yet so far away. Biopharma Dive. January 28, 2021. Accessed June 16, 2021. https://www.biopharmadive.com/news/ hemophilia-gene-therapy-biomarinuniqure-pfizer-roche/594168/ 8. Informa. Biomedtracker. Hemophilia A indication profile. Accessed March 24, 2021. https://www.biomedtracker.com/ indicationreport.cfm?indid=511 9. Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for hemophilia. Blood. 2019;133(5):407-414. 10. ASH Clinical News. Gene therapy for hemophilia: progress and setbacks. November 1, 2020. Accessed June 16, 2021. https://www.ashclinicalnews.org/spotlight/ feature-articles/gene-therapy-hemophiliaprogress-setbacks/ 11. BioMarin. BioMarin receives complete response letter (CRL) from FDA for valoctocogene roxaparvovec gene therapy for severe hemophilia A. Published August 19, 2020. Accessed June 16, 2021. https:// investors.biomarin.com/2020-08-19BioMarin-Receives-Complete-ResponseLetter-CRL-from-FDA-for-ValoctocogeneRoxaparvovec-Gene-Therapy-for-SevereHemophilia-A 12. Institute for Clinical and Economic Review. Hemophilia A. An assessment of valoctogene roxaparvovec emicizumab for the treatment of hemophilia A. Accessed June 16, 2021. https://icer.org/assessment/ hemophilia-a-2020/
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