Specialty Pharmacy Continuum - May/ June 2022

Serving managed care, health-system and specialty decision makers Volume 11 • Number 3 • May/June 2022 • specialtypharmacycontinuum.com

POLICY

6 payor tactics to control drug spending ..................... 3

Payors Pushing Back On Digital Therapeutics

4 value-based contracting strategies .....4

Ohio Insurers Give Provider Status a Boost

By Karen Blum

Chicago—Since 2019, when Ohio legislators passed a bill allowing health plans to recognize pharmacists as care providers, several plans have established programs to recognize pharmacists in this expanded role and demonstrate their value.

Several forces converged to influence the state legislature to acknowledge pharmacists as healthcare providers who could be reimbursed for patient sservices, said Stuart Beatty, PharmD, BCACP, FAPhA, the director of strategy B aand practice transformation at the Ohio Pharmacists Association (OPA), during P tthe AMCP 2022 annual meeting.

OPERATIONS & MGMT Are health systems transitioning to at-home oncology infusion model? ................ 12 Cooking up a digestible drug budget ................................. 13 Doug Long’s annual industry outlook.............. 14

Continued on page 5

By David Bronstein

CLINICAL A peek into NASH Rx pipeline pricing ......... 18 The specialty pharmacist’s role in cGVHD............................. 22

Las Vegas—A software program that helps clini-cians diagnose autism as early as 18 months of age, versus the 3 to 4 years required by conventional workups. ups.

An interactive game for children with attentionondeficit/hyperactivity disorder (ADHD) that can bee a powerful adjunct to drug therapy. A phone-based d app that helps patients with opioid use disorder (OUD) comply mply with their addiction medication.

All of these interventions, part of a burgeoning treatment class known as prescription digital therapeutics (PDTs), are approved by the FDA. They also have been a boon during COVID-19, because many of them can be used remotely and fill critical provider gaps caused by the pandemic.

However, PDTs face a major hurdle: Less than half of payors say they are willing to cover them, according to a new survey by Managed Markets Insight & Technology (MMIT) and reported at AXS22/Asembia Specialty Pharmacy Summit 2022.

Respondents cited several reasons for the resistance, according to Jayne Hornung, MMIT’s chief medical officer, who presented the survey results. One insurer expressed an unwillingness to “pay for convenience” during a focus group discussion on PDTs that was part of the survey. Another payor stated that many of the PDTs it assessed “lack clinical value,” which led it to decide “not to go any further” in reviewing the merits of PDTs, Ms. Hornung noted.

Such attitudes are at odds with the clinical data underpinning the current crop of Continued on page 20

Green containers:

Cold Chain Update See page 17

Pharmacists Can Help Drive C Biosimilars Growth

By Dave Doolittle B

Las Vegas—As medication experts and some of the most accessible members of the healthcare team, pharmacists are well positioned to support the continued adoption of biosimilars.

However, several clinical, operational and financial challenges—as well as misperceptions—need to be overcome for biosimilars to reach their full potential, Sonia Oskouei, PharmD, vice president, Biosimilars, for Cardinal Health, said during a presentation at the AXS22/Asembia Specialty Pharmacy Summit 2022.

Continued on page 10

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Specialty Pharmacy Continuum • May/June 2022

POLICY

6 Payor Tactics to Control Drug Spending

By Karen Blum

The growth of high-cost specialty drugs is causing payors to scramble to manage spending on these products, speakers said during the 2022 Managed Health Care Associates Inc. (MHA) Business Summit in Las Vegas. The payors discussed six trends affecting management of specialty and infusion drugs.

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Specialty products are outpacing traditional medications. Specialty drug spending in 2020 was $265.3 billion, or 49.6% of total pharmacy expenditures, said Kimberly Grant, PharmD, a clinical pharmacist with IPD Analytics, citing published data (Am J Health Syst Pharm 2021;78[14]:1294-1308), and is on target to exceed that this year. Some 65% of new drug approvals in the next three years are estimated to fall into the rare disease or oncology categories, Dr. Grant said.

The increase in specialty products is a challenge for payors, Dr. Grant said. Not only are more drugs being approved; many of them have expanding indications. “When you look into the pipeline, manufacturers are really going after multiple additional indications over the next three to five years,” she said.

One example is efgartigimod alfafcab (Vyvgart, Argenx), approved by the FDA in December 2021 for adults with generalized myasthenia gravis.

“It’s a small patient population right now,” Dr. Grant said, “but when you look at Argenx’s goals, they’re looking to be active in about 15 disease states by 2025.” A payor might just focus on a myasthenia gravis policy now but needs to keep these drugs on the radar for additional indications, she said.

She added that products for rare and ultrarare diseases are expected to account for more than 20% of all prescription drug sales by 2024. One-time cell and gene therapies also are increasing.

Currently, payors consider these prodroducts individually, Dr. Grant said, d, but aan estimated 25 to 30 million Ame Americans have a rare disease, increasing the chances that payors will have multiple affected beneficiaries. Some drugs, such as onasemnogene abeparvovec-xioi (Zolgensma, Novartis) for spinal muscular atrophy, were approved “with a pretty broad label,” she said. In response, payors are trying to manage

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use of the drug based on how it was studied in clinical trials, “which is a lot stricter than the FDA-approved labeling,” Dr. Grant said. “Currently, about 57% of coverage decisions for cell and gene therapies are more restrictive than the drug’s FDA-approved label.”

Payors also are struggling with how to define value for one-time, potentially curative therapies, without long-term outcomes data, she noted.

Creative contracting and payment models are gaining traction. Health plans are engaging in various creative contracts and payment models with manufacturers to contain costs, Dr. Grant said. These include payment plans tied to achieving certain outcomes; mortgage models in which payors make a 20% down payment on a medication, followed by monthly sums; and subscription models in which payors pay a flat monthly fee. There are limited publicly available data on these arrangements, she said.

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3 Biosimilars Strategies te wit w with h pay p payors yors to accept ! Negotiate the e same reimbursement eimbu ment rate as their their preferred prefe d pharmacy. pharmac with m manufacturers anufa anu @ Work to gain n acces access to limited distribution drugs. distribut proficient in reim# BBecome bursement, inventory management and prior authorization/ appeals, and integrate specialty pharmacy–delivered drugs into inventory and electronic medical records systems.

Source: Kimberly Grant, PharmD.

One area of interest is mobility clauses, Dr. Grant said. If a payor is covering an expensive therapy for a beneficiary, and

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that person moves to another plan, will the payment plan follow the patient? “I suspect we’re going to be hearing more about the execution of these over the next few years,” she said.

Biosimilars are predicted to slash drug expenditures.

Biosimilars will likely have the greatest impact on future drug spending, with estimated savings of $38 billion from 2021 to 2025, Dr. Grant said. Some issues remain, including a need for more patient and provider education about these products, and “skinny labels,” where a biosimilar is approved for one of several of a reference product’s indications.

Payor strategies regarding biosimilars vary, she said, with some plans nervous about losing rebates on the reference products still preferring the brand names, others allowing a few preferred biosimilars or an “all-in” attitude. “With biosimilar all-in, you’re losing rebates on the reference products, so you’re going to see a lot of aggressive management strategies in terms of step therapies or prior authorizations just to move patients on to the biosimilars,” she said. Incentivized switch programs may offer beneficiaries a one-time payment to move to a biosimilar or preferred biologic. (For more biosimilars trends, see page 1, as well as box on this page.)

Pressure is building to shift to the medical benefit. Plans didn’t historically manage drugs on the medical benefit as strictly as the pharmacy benefit, but now there is increasing economic pressure to do so, Dr. Grant said. Payor strategies here include aggressive site-of-care optimization strategies directing patients to the most cost-effective location to receive medications, and requiring billing through specialty pharmacies, known as bagging strategies, where health systems and physician practices must accept bagged medications from pharmacies to administer to patients. The best option is gold bagging, in which a specialty pharmacy dispenses prescriptions to its own clinics for administration, she said. Some states and professional groups, such as the American Hospital Association, have banned or oppose bagging for its potential disruptions in care.

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Competition is building among patient assistance and access programs. Manufacturer-sponsored financial assistance programs designed to offset out-of-pocket costs for patients have been offered in multiple formats, such as a coupon/copay cards, free trials, patient assistance programs or bridge/quickstart programs, said Julia Mahler, PharmD, a clinical pharmacist with IPD Analytics.

Biosimilars also have patient access programs, Dr. Mahler said, with new programs tending to mirror those of competitor medications. “This is important because if I’m taking adalimumab [Humira, AbbVie] and paying $5 a month, and I’m being switched to a lower-cost biosimilar, I don’t want to be paying more than $5 a month for a brand,” she said. “These patient access programs have to be as robust for biosimilars as they are for the reference brand product.”

Copay accumulators still in play.

Payors continue to use copay accumulators and maximizer programs to shift costs to manufacturer financial assistance programs, she said. The accumulator adjustments are a strategy to stop copay assistance from being applied to patient deductibles and outof-pocket spending, until a coupon’s value is exhausted. The maximizers take the maximum value of financial assistance programs and split them evenly throughout a coverage year.

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Challenges Ahead The presentation was beneficial to MHA members in attendance, commented Stephen Moll, MBA, the director of sales, Alternate Site Care Division, at MHA, and moderator of the session.

“The biggest takeaway was solidification on the direction of the biosimilar market, and that having a very large impact on drugs in the pipeline, as well as the challenges that we’ll still face with acceptance from doctors using biosimilars to the payor side,” Mr. Moll said. “I hope the presentation gave them enough information to take back and implement into their business of pharmacy.”

Dr. Grant has received an unrestricted educational grant from Bayer HealthCare Pharmaceuticals Inc. Dr. Mahler and Mr. Moll reported no relevant financial disclosures.

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Specialty Pharmacy Continuum • May/June 2022

POLICY

4 Value-Based Contracting Strategies

By Karen Blum

Chicago—Value-based contracts (VBCs) provide an opportunity for manufacturers to evaluate outcomes not studied in clinical trials, while offering payors attractive pricing.

Speakers at a panel discussion at the AMCP 2022 annual meeting provided insight into these benefits, along with an assessment of the VBC marketplace and advice on how payors can pursue such contracts.

“I see this as an organized scramble right now,” Paul Jeffrey, PharmD, the principal at Paul Jeffrey Consulting and retired senior director of pharmacy for MassHealth, the Massachusetts Medicaid program, said during the discussion.

Rapid Growth VBCs can be traced to the 1930s, with the start of managed payment plans, Dr. Jeffrey noted. At that time, he said, it wasn’t about value as much as a way for people to purchase healthcare.

Over the past 30 years, payors have shifted from paying for service to quality and then to value, he said. The term “value-based care” was introduced in the early part of this century, he said, and has progressed continually with the formation of accountable care organizations. “Value-based contracts” as a term began entering the literature and lexicon starting around 2010.

The number of publicly disclosed VBCs increased from two in 2009 to 19 in 2018, representing eight therapeutic areas, said Mahsa Salsabili, PharmD, PhD, a pharmacoeconomics specialist at University of Massachusetts Chan Medical School, in Shrewsbury.

Cardiology and neurology have been the top therapeutic areas covered by VBCs, followed by endocrinology, she said.

4 Negotiating Stages MassHealth’s team goes through four stages during direct negotiations with manufacturers, said Neha Kashalikar, PharmD, a clinical pharmacist with the Office of Clinical Affairs, the MassHealth clinical decision support unit staffed by UMass Chan Medical School. The multidisciplinary team includes clinical and operational pharmacists who lead negotiations, evaluate offers and implement new contracts. They are backed up by senior leadership, pharmacoeconomics specialists, data analysts, and legal and information technology supports.

Team members meet with manufacturers frequently, often several times a week to discuss pipeline products, or proposed value-based or supplemental rebate agreements.

Once a contracting proposal has been submitted, the team conducts a thorough review, including clinical evaluation of current evidence-based medicine as well as a review of any market trends or pricing, coupled with utilization data. The team then compiles a fiscal analysis to determine savings that may come from the contract. When appropriate, the team consults other stakeholders, such as physicians, to ensure alignment around the proposed contract terms.

Once the team and other stakeholders agree on the proposed contract terms, MassHealth enters into negotiations with the manufacturer to ensure the contract provides the greatest value to the state program.

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‘All of these [VBC models] are undergoing evaluation. This market is anything but settled.’

—Paul Jeffrey, PharmD

Several types of contracts exist today, Dr. Jeffrey said. There are warranties, in which payors get their money back if a product doesn’t work.

Some contracts allow payors to spread out payments for drugs. Subscription models allow payors to pay one price to treat as many beneficiaries as needed. And hybrid models combine elements of these different plans.

“All of these are undergoing evaluation,” Dr. Jeffrey said. “This market is anything but settled.”

MassHealth has reached agreements for supplemental rebates with

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Then, they work to put the agreement in place. This includes implementing any criteria changes to the MassHealth drug list, communicating to providers about upcoming changes and coordinating with managed care organization plans to ensure alignment in formulary decisions. Most contracts signed by the program are for one year, Dr. Kashalikar said.

MassHealth takes a proactive approach to identifying high-priority drugs for contracting on the basis of

17 manufacturers for

45 drugs, for a nearly

$201 million annual value.

how they may provide value to the program, she said. Her team runs a focused report each year to identify drugs that are high cost, that have a high averageper-member per-year cost, that have low rebates and those with increasing utilization among their beneficiaries.

Once high-priority drugs are identified, a pharmacoeconomics specialist helps the team further refine its priority based on market landscape, utilization data, international pricing and other characteristics, to determine which drugs may be most successful for negotiation.

It’s important to be wary of scenarios where contract terms may potentially put a plan at odds with best practice, Dr. Kashalikar cautioned.

To date, MassHealth has reached agreements for supplemental rebates with 17 manufacturers for 45 drugs, for a nearly $201 million annual value, Dr. Kashalikar said. The program has eight value-based agreements in place, one for a novel digital therapeutic product, and continues to have ongoing negotiations with manufacturers for many medications.

VBCs Versus Supplemental Rebate Agreements One of the first questions the team members ask themselves is whether a particular drug is better suited for a VBC or a supplemental rebate agreement, in which a manufacturer may provide an enhanced rebate (on top of the federal rebate) for preferred status for its product compared with clinical competitors, Dr. Kashalikar said. Drugs well suited for VBCs include those that may have been approved based on biomarkers, those with limited real-world evidence

and those in which there is a question surrounding safety and efficacy. It allows the manufacturer to stand behind the expected outcomes of a drug.

On the flip side, those more suited to supplemental rebate agreements include drugs with established safety and efficacy data; those with a high monitoring or administration cost; and with patient-reported outcomes.

High-cost drugs, pipeline products and those with increasing utilization fall in the middle and could be suitable for either arrangement, she said.

It’s important for payors and manufacturers to work together to identify the best endpoints for value-based contracts. Those end points should be clinically relevant, tracked as part of routine patient care and able to be reported easily by providers or office staff. Contracts must be structured in a manner that is operationalizable with an objective end point that can be tracked either on a prior authorization form or through pharmacy or medical claims data.

Next Steps Future trends to look for include continued state legislation in this area; publications of evaluations on the utility of value-based agreements; the evolution of a standardized approach to contracts; the solidification of best practices; and the application of VBCs to digital therapeutics, Dr. Jeffrey said.

“I don’t think that value-based contracting is going to go away,” he said. “I think it’s going to continue to escalate and become more prevalent.”

The speakers reported no relevant financial disclosures.

r su

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Specialty Pharmacy Continuum • May/June 2022

POLICY

Provider Status Boost continued from page 1

Legislation had been introduced to include pharmacists as providers in the state. Meanwhile, healthcare reform was trending toward value-based care, which led to pharmacists being more involved in patient care activities nationwide. Finally, there was much scrutiny of pharmacy benefit manager (PBM) practices in the state, particularly within the Medicaid program. This issue was covered widely in state media, “allowing legislators and the public to become aware of … the pharmacy model, [and] also the potential for the pharmacist to be an untapped resource for access to a potentially lower-cost healthcare model,” said Dr. Beatty, who is also an associate professor of clinical pharmacy at The Ohio State University College of Pharmacy, in Columbus, during a panel discussion.

Pharmacist provider status, as covered under Ohio Senate Bill 265, was signed into law in January 2019, Dr. Beatty said. It permitted insurers to cover pharmacist services and allowed programs to start without any type of mandate. From there, the OPA began working with the state’s five Medicaid managed care organizations on plans to accept pharmacists as providers. Once pharmacists sign up for a Medicaid ID number and enroll with Medicaid plans, they can begin billing as a provider if all rules are met.

Focusing on Rural Areas CareSource, a health plan based in Dayton, worked with one pharmacy in the rural area of Camden and another in an underserved community near downtown Dayton, said Nicholas Trego, PharmD, RPh, the company’s associate vice president of the Ohio Pharmacy Market. They focused on four areas: smoking cessation, diabetes care, asthma control and naloxone therapy for patients taking opioids.

OPA worked with pharmacists and the CareSource team to assist with implementation of the services.

Documentation and billing templates were created, and the OPA team helped guide pharmacists through visits, monitoring of patients and implementation of the new program. The CareSource team also attended calls to ensure appropriate patient selection, make sure pharmacists knew about other resources available from the health plan and ascertain appropriate data collection.

Looking at the effectiveness of pharmacists’ interventions, 80% of patients had improvements in asthma control test scores; 75% experienced a reduction in blood glucose; 50% reduced their tobacco use; 89% started on tobacco

cessation medications; and three patients were counseled and given naloxone. There were anecdotes of success as well, Dr. Trego said, such as a pharmacist detecting a foot ulcer in one patient with diabetes, and obtaining a nebulizer within two days for another patient with asthma who had been going to a hospital emergency department (ED) weekly for breathing treatments. Along the way, pharmacists asked for the ability to order laboratory tests to help with work on hemoglobin A1c (HbA1c) levels for people with diabetes.

“That’s a really big gap pharmacists are able to fill in for health plans,” Dr. Trego said. “A1c testing is very important to have. It’s associated with the National Committee for Quality Assurance’s HEDIS (healthcare effectiveness data and information set) metrics, so that is another really big opportunity for this to expand in the future.”

Pharmacists Pitch In During COVID-19 In what Dr. Trego said was perhaps his favorite story about the effectiveness of the program, he cited the experience of two physicians at the medical practice near the Camden pharmacy who came down with COVID-19 at the same time and had to close their office for two weeks. CareSource allowed pharmacists to see patients for any disease state during that period to avoid having to shuttle patients 45 minutes each way from Camden to Dayton to be seen by another physician. “How much cooler of a story can you have than pharmacists stepping in and taking care of the healthcare for the town?” Dr. Trego noted.

When the Ohio legislation passed, Buckeye Health Plan, part of Centene Corporation, was starting to hear enthusiasm from hospital systems and others wanting to hire pharmacists but looking for programs to show outcomes and return on investment to leadership, said Meera Patel-Zook, PharmD, the vice president of pharmacy operations for the health plan. In some cases, healthcare entities had pharmacists on staff, but not an established way to get reimbursed for their time.

To start, the plan focused on working with two federally qualified health centers and one hospital system before expanding to other pharmacies. They worked through a collaborative practice agreement to focus on HEDIS measures and holistic disease state management for conditions such as diabetes, cardiovascular disease, asthma, behavioral health and other chronic conditions, with the goals of reducing healthcare spending, improving member

experience and enhancing provider partnerships. Monthly dashboards were provided to pharmacists to give them information about which patients had potential gaps in care, such as uncontrolled HbA1c levels or elevated blood pressure. The teams also partnered with OPA for discussions on new workflow and billing practices. “Eventually, we wanted to be able to show that this model that we’re creating can decrease inpatient visits, decrease ED visits,” Dr. Patel-Zook said. “Post-utilization from this should be favorable.”

So far, the program has about six months of claims data, but the three locations are showing that with pharmacist intervention, members have been 18% more likely to get routine vaccinations, 17% more likely to have blood pressure under control and 32% more likely to get a COVID-19 vaccine than in other practices without pharmacist involvement, she said.

Anecdotes also have been favorable, Dr. Patel-Zook noted. In one case, a pharmacist had a 45-minute conversation with a member whose HbA1c level was more than 13%. While they talked, the pharmacist observed the member was becoming hypoglycemic, so she gave her some food. They also discovered the person was living on her son’s couch in an unsafe area, and had anxiety as a result. The pharmacist linked the patient to the health plan’s social determinants of health care coordination team, which connected the member to stable housing.

Since then, the member’s HbA1c level has fallen to 6% in less than six months.

The person now is on antianxiety medication, is adherent to diabetes medication and has had her artwork featured in local newspapers.

Molina Healthcare Takes Different Approach Molina Healthcare of Ohio used a different strategy, allowing pharmacists at 10 independent pharmacies in the state to treat patients for conditions as they saw a need, said Kimberly Broyles-Kpogli, PharmD, MBA, the company’s director of pharmacy. “We didn’t want them to just think about diabetes, hypertension or respiratory concerns,” she said. “We knew that they would because a lot of our members have those conditions, but we didn’t want the pharmacists to feel they were in a box. We wanted them to provide care to patients where it was needed because these pharmacists likely knew our patients better than we did at the health plan.”

Pharmacists provided various interventions and services such as administering vaccinations, counseling for medications and their side effects, and offering smoking cessation and diabetes education. One pharmacy chose to

With pharmacist intervention, members have been

18%

more likely

to get routine vaccinations,

17%

more likely

to have blood pressure under control and

32%

more likely

to get a COVID-19 vaccine than in other practices without pharmacist involvement.

pursue behavioral health and developed a pharmacist intervention for depression and anxiety conditions.

In this program, pharmacists administered standardized Patient Health Questionnaire-9 (PHQ9) or Generalized Anxiety Disorder (GAD7) tests to patients who regularly filled antidepressant or antianxiety medications, to assess their mental states. The site thought this was especially important given the effect of the COVID-19 pandemic on mental health and access to primary care services. After obtaining baseline scores, pharmacists worked to educate members about the medications, ensured adherence, provided counseling to optimize therapy effectiveness and made recommendations to providers if needed. Over a sixmonth period, pharmacists met with 43 patients. About 73% of patients had stable or improved PHQ9/GAD7 scores after pharmacist follow-up.

Pharmacists participating in the programs were invited to join group phone calls with OPA for troubleshooting and sharing advice. Overall, pharmacists have loved their experiences, Dr. Beatty said. “The programs recognizing pharmacists as providers are creating an incentive in the healthcare model that is fully taking advantage of the training of pharmacists. Our pharmacists are saying, ‘This is the reason I went to school.

I want to get back to actually taking care of these patients that I’ve seen sometimes for 10 to 15 years and help them be healthier.’ We look forward to the continuation of this program in Ohio and other states that allow pharmacists to add to the high level of care that should be expected by patients.”

The speakers reported no relevant financial disclosures.

For patients with cGVHD ­æÐÌ ɖǠǡ řЭīĮ ­åĴÐī å­ðăķīÐ ďå ­Ĵ ăЭĮĴ ǡ Ĩīðďī ăðĊÐĮ ďå ĮřĮĴÐĉðÆ ĴìÐī­Ĩřș ìÐăĨ ĴìÐĉǠȭǢ

ROCK ON

INDICATION REZUROCK™ (belumosudil) is indicated for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (chronic GVHD) after failure of at least two prior lines of systemic therapy.

IMPORTANT SAFETY INFORMATION Warnings and Precautions • Embryo-Fetal Toxicity: ­ĮÐÌ ďĊ ť ĊÌðĊæĮ ðĊ ­Ċðĉ­ăĮ ­ĊÌ ðĴĮ ĉÐÆì­ĊðĮĉ ďå ­ÆĴðďĊș t'¢ tZ N Æ­Ċ Æ­ķĮÐ åÐĴ­ă ì­īĉ œìÐĊ administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with REZUROCK and for at least one week after the last dose Adverse Reactions • }ìÐ ĉďĮĴ ÆďĉĉďĊ ȧɖ ǡǟɦȨ ­ÌŒÐīĮÐ īЭÆĴðďĊĮș ðĊÆăķÌðĊæ ă­Åďī­Ĵďīř ­ÅĊďīĉ­ăðĴðÐĮș œÐīÐ ðĊåÐÆĴðďĊĮș ­ĮĴìÐĊð­ș Ċ­ķĮЭș Ìð­īīìЭș ÌřĮĨĊЭș Æďķæìș ÐÌÐĉ­ș ìÐĉďīīì­æÐș ­ÅÌďĉðĊ­ă Ĩ­ðĊș ĉķĮÆķăďĮāÐăÐĴ­ă Ĩ­ðĊș ìЭ̭ÆìÐș ĨìďĮĨì­ĴÐ ÌÐÆīЭĮÐÌș æ­ĉĉ­ æăķĴ­ĉřă Ĵī­ĊĮåÐī­ĮÐ ðĊÆīЭĮÐÌș ăřĉĨìďÆřĴÐĮ ÌÐÆīЭĮÐÌș ­ĊÌ ìřĨÐīĴÐĊĮðďĊ • qÐīĉ­ĊÐĊĴ ÌðĮÆďĊĴðĊķ­ĴðďĊ ďå t'¢ tZ N ÌķÐ Ĵď ­ÌŒÐīĮÐ īЭÆĴðďĊĮ ďÆÆķīīÐÌ ðĊ Ǡǧɦ ďå Ĩ­ĴðÐĊĴĮȘ }ìÐ ­ÌŒÐīĮÐ īЭÆĴðďĊĮ œìðÆì īÐĮķăĴÐÌ ðĊ ĨÐīĉ­ĊÐĊĴ ÌðĮÆďĊĴðĊķ­ĴðďĊ ďå t'¢ tZ N ðĊ ɔ Ǣɦ ďå Ĩ­ĴðÐĊĴĮ ðĊÆăķÌÐÌ Ċ­ķĮЭ ȧǣɦȨȘ ÌŒÐīĮÐ īЭÆĴðďĊĮ ăЭÌðĊæ Ĵď ÌďĮÐ ðĊĴÐīīķĨĴðďĊ ďÆÆķīīÐÌ ðĊ ǡǨɦ ďå Ĩ­ĴðÐĊĴĮȘ }ìÐ ­ÌŒÐīĮÐ īЭÆĴðďĊĮ ăЭÌðĊæ Ĵď ÌďĮÐ ðĊĴÐīīķĨĴðďĊ ðĊ ɖ ǡɦ œÐīÐ ðĊåÐÆĴðďĊĮ ȧǠǠɦȨș Ìð­īīìЭ ȧǣɦȨș ­ĊÌ ­ĮĴìÐĊð­ș ÌřĮĨĊЭș ìÐĉďīīì­æÐș ìřĨďĴÐĊĮðďĊș ăðŒÐī åķĊÆĴðďĊ ĴÐĮĴ ­ÅĊďīĉ­ăș Ċ­ķĮЭș ĨřīÐŘð­ș ÐÌÐĉ­ș ­ĊÌ īÐĊ­ă å­ðăķīÐ œðĴì ȧǡɦ ЭÆìȨ • TďĊðĴďī ĴďĴ­ă ÅðăðīķÅðĊș ­ĮĨ­īĴ­ĴÐ ­ĉðĊďĴī­ĊĮåÐī­ĮÐ ȧ w}Ȩș ­ĊÌ ­ă­ĊðĊÐ ­ĉðĊďĴī­ĊĮåÐī­ĮÐ ȧ O}Ȩ ­Ĵ ăЭĮĴ ĉďĊĴìăř Drug Interactions • Strong CYP3A Inducers: Coadministration of REZUROCK with strong CYP3A inducers decreases belumosudil ÐŘĨďĮķīÐș œìðÆì ĉ­ř īÐÌķÆÐ ĴìÐ ÐŨ Æ­Æř ďå t'¢ tZ NȘ AĊÆīЭĮÐ ĴìÐ ÌďĮ­æÐ ďå t'¢ tZ N Ĵď ǡǟǟ ĉæ ĴœðÆÐ Ì­ðăř when coadministered with strong CYP3A inducers • Proton Pump Inhibitors: Coadministration of REZUROCK with proton pump inhibitors decreases belumosudil ÐŘĨďĮķīÐș œìðÆì ĉ­ř īÐÌķÆÐ ĴìÐ ÐŨ Æ­Æř ďå t'¢ tZ NȘ AĊÆīЭĮÐ ĴìÐ ÌďĮ­æÐ ďå t'¢ tZ N Ĵď ǡǟǟ ĉæ ĴœðÆÐ Ì­ðăř when coadministered with proton pump inhibitors

ORRa

75% (95% CI, 63-85; P<.0001) 1,4

REZUROCK achieved clinically and statistically ĮðæĊðť Æ­ĊĴ ÐŨ Æ­Æř with the 200-mg once-daily dose in a real-world demographic of patients with cGVHD.1

• ZĊÆÐȭÌ­ðăř ďī­ă ĉÐÌðÆ­ĴðďĊ Ĵì­Ĵ Ĵ­īæÐĴĮ ÅďĴì ðĊŦ ­ĉĉ­ĴðďĊ ­ĊÌ ť ÅīďĮðĮ Ĵìīďķæì ĮÐăÐÆĴðŒÐ tZ Nǡ ðĊìðÅðĴðďĊ1-3 • CR was observed in all organsș ðĊÆăķÌðĊæ ĴìďĮÐ œðĴì ť ÅīďĴðÆ ĉ­ĊðåÐĮĴ­ĴðďĊĮ

5

• There was no death or new systemic therapy initiation in 62% (95% CI, 46-74) of the responder population at 12 months1 • Clinically meaningful improvements in QOL, with CS and CNI dose reductions and discontinuations ­ÆìðÐŒÐÌ ðĊ ÅďĴì īÐĮĨďĊÌÐīĮ and nonresponders5 • Well tolerated1

ASSIST

™

Enroll your patients with cGVHD in Kadmon ASSIST so our ĮĨÐÆð­ăðĮĴĮ Æ­Ċ ť ĊÌ ĴìÐ ÅÐĮĴ Ĩīďæī­ĉ Ĵď ť Ĵ řďķī Ĩ­ĴðÐĊĴĮȸ ÆďŒÐī­æÐȘ Kadmon ASSIST ðĮ ­Œ­ðă­ÅăÐ Monday through Friday, 8ŬƢ-8ƲƢ ET, Åř Æ­ăăðĊæ 1-844-KADMON1 (523-6661).

VISIT REZUROCKhcp.com TO LEARN MORE cGVHD, chronic graft-versus-host disease; CNI, calcineurin inhibitor; CR, complete response; CS, corticosteroid; FDA, US Food and Drug Administration; NIH, National Institutes of Health; ORR, overall response rate; PR, partial response; QOL, quality of life; ROCK2, rho-associated coiled-coil–containing protein kinase-2.

Proportion of patients who achieved CR or PR according to the 2014 NIH cGVHD Consensus Criteria in the 200-mg once-daily arm.1

a

IMPORTANT SAFETY INFORMATION (cont) ĮÐ ðĊ wĨÐÆðť Æ qďĨķă­ĴðďĊĮ • Pregnancy: ­ĮÐÌ ďĊ ť ĊÌðĊæĮ åīďĉ ­Ċðĉ­ă ĮĴķÌðÐĮ ­ĊÌ ĴìÐ ĉÐÆì­ĊðĮĉ ďå ­ÆĴðďĊș t'¢ tZ N Æ­Ċ Æ­ķĮÐ åÐĴ­ă ì­īĉ œìÐĊ administered to pregnant women. There are no available human data on REZUROCK use in pregnant women to evaluate for a drug-associated risk. Advise pregnant women and females of reproductive potential of the potential risk to the fetus • Lactation: There are no data available on the presence of belumosudil or its metabolites in human milk or the effects on ĴìÐ ÅīЭĮĴåÐÌ ÆìðăÌș ďī ĉðăā ĨīďÌķÆĴðďĊȘ ÐÆ­ķĮÐ ďå ĴìÐ ĨďĴÐĊĴð­ă åďī ĮÐīðďķĮ ­ÌŒÐīĮÐ īЭÆĴðďĊĮ åīďĉ ÅÐăķĉďĮķÌðă ðĊ ĴìÐ ÅīЭĮĴåÐÌ ÆìðăÌș ­ÌŒðĮÐ ă­ÆĴ­ĴðĊæ œďĉÐĊ ĊďĴ Ĵď ÅīЭĮĴåÐÐÌ ÌķīðĊæ ĴīЭĴĉÐĊĴ œðĴì t'¢ tZ N ­ĊÌ åďī ­Ĵ ăЭĮĴ ďĊÐ œÐÐā after the last dose • Pediatric Use: The safety and effectiveness of REZUROCK have been established in pediatric patients 12 years and older.

The safety and effectiveness of REZUROCK in pediatric patients less than 12 years old have not been established • Geriatric Use: Zå ĴìÐ Ǡǧǥ Ĩ­ĴðÐĊĴĮ œðĴì ÆìīďĊðÆ : ># ðĊ ÆăðĊðÆ­ă ĮĴķÌðÐĮ ďå t'¢ tZ Nș ǡǥɦ œÐīÐ ǥǤ řЭīĮ ­ĊÌ ďăÌÐīȘ Uď clinically meaningful differences in safety or effectiveness of REZUROCK were observed in comparison to younger patients • Renal and Hepatic Impairment: Treatment with REZUROCK has not been studied in patients with pre-existing severe īÐĊ­ă ďī ìÐĨ­ĴðÆ ðĉĨ­ðīĉÐĊĴȘ 9ďī Ĩ­ĴðÐĊĴĮ œðĴì ĨīÐȭÐŘðĮĴðĊæ ĮÐŒÐīÐ īÐĊ­ă ďī ìÐĨ­ĴðÆ ðĉĨ­ðīĉÐĊĴș ÆďĊĮðÌÐī ĴìÐ īðĮāĮ ­ĊÌ ĨďĴÐĊĴð­ă ÅÐĊÐť ĴĮ ÅÐåďīÐ ðĊðĴð­ĴðĊæ ĴīЭĴĉÐĊĴ œðĴì t'¢ tZ N You are encouraged to report side effects of prescription drugs to the FDA. Visit www.FDA.gov/medwatch or call Ǡȭǧǟǟȭ9# ȭǠǟǧǧȘ ďķ ĉ­ř ­ăĮď ÆďĊĴ­ÆĴ N­ÌĉďĊ qì­īĉ­ÆÐķĴðÆ­ăĮș OO ș ­Ĵ ǠȭǧǦǦȭǢǦǦȭǦǧǥǡ Ĵď īÐĨďīĴ ĮðÌÐ ÐååÐÆĴĮȘ References: 1. REZUROCK. Package insert. Kadmon Pharmaceuticals, LLC; 2021. 2. Zanin-Zhorov A, Weiss JM, Nyuydzefe MS, et al. Selective oral ROCK2 inhibitor down-regulates IL-21 and IL-17 secretion in human T cells via STAT3-dependent mechanism. Proc Natl Acad Sci USA. 2014;111(47):16814-16819. doi:10.1073/pnas.1414189111 3. Flynn R, Paz K, Du J, et al. Targeted rho associated kinase 2 inhibition suppresses murine and human chronic GVHD through a Stat3-dependent mechanism.

Blood. 2016;127(17):2144-2154. doi:10.1182/blood-2015-10-678706 4. #­Ĵ­ ďĊ ť ăÐȘ N­ÌĉďĊ qì­īĉ­ÆÐķĴðÆ­ăĮș OO ț ǡǟǡǠȘ 5. Cutler CS, Lee SJ, Arai S, et al. Belumosudil for chronic graft-versus-host disease (cGVHD) after 2 or more prior lines of therapy: the ROCKstar study. Blood. 2021;blood.2021012021. doi:10.1182/blood.2021012021

Please see Brief Summary of full Prescribing Information on adjacent pages.

© 2021 Kadmon Pharmaceuticals, LLC. All Rights Reserved.

KAD25000281 11/21

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REZUROCK™ (belumosudil) tablets, for oral use Initial U.S. Approval: 2021 BRIEF SUMMARY: Please see package insert for full prescribing information.

1 INDICATIONS AND USAGE REZUROCK is indicated for the treatment of adult and pediatric patients 12 years and older with chronic graft-versushost disease (chronic GVHD) after failure of at least two prior lines of systemic therapy.

4 CONTRAINDICATIONS None.

5 WARNINGS AND PRECAUTIONS 5.1 Embryo-Fetal Toxicity Based on findings in animals and its mechanism of action, REZUROCK can cause fetal harm when administered to a pregnant woman. In animal reproduction studies, administration of belumosudil to pregnant rats and rabbits during the period organogenesis caused adverse developmental outcomes including embryo-fetal mortality and malformations at maternal exposures (AUC) less than those in patients at the recommended dose.

Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with REZUROCK and for at least one week after the last dose [see Use in Specific Populations (8.1, 8.3), Nonclinical Toxicology (13.1) in the full prescribing information].

6 ADVERSE REACTIONS 6.1 Clinical Trial Experience Because clinical trials are conducted under widely variable conditions, adverse reaction rates observed in clinical trials of a drug cannot be directly compared with rates of clinical trials of another drug and may not reflect the rates observed in practice.

Chronic Graft versus Host Disease In two clinical trials (Study KD025-213 and Study KD025-208), 83 adult patients with chronic GVHD were treated with REZUROCK 200 mg once daily [see Clinical Studies (14.1) in the full prescribing information]. The median duration of treatment was 9.2 months (range 0.5 to 44.7 months).

Fatal adverse reaction was reported in one patient with severe nausea, vomiting, diarrhea and multi-organ failure.

Permanent discontinuation of REZUROCK due to adverse reactions occurred in 18% of patients. The adverse reactions which resulted in permanent discontinuation of REZUROCK in > 3% of patients included nausea (4%).

Adverse reactions leading to dose interruption occurred in 29% of patients. The adverse reactions leading to dose interruption in ≥ 2% were infections (11%), diarrhea (4%), and asthenia, dyspnea, hemorrhage, hypotension, liver function test abnormal, nausea, pyrexia, edema, and renal failure with (2% each).

The most common (≥ 20%) adverse reactions, including laboratory abnormalities, were infections, asthenia, nausea, diarrhea, dyspnea, cough, edema, hemorrhage, abdominal pain, musculoskeletal pain, headache, phosphate decreased, gamma glutamyl transferase increased, lymphocytes decreased, and hypertension.

Table 2 summarizes the nonlaboratory adverse reactions.

Table 2: Nonlaboratory Adverse Reactions in ≥ 10% Patients with Chronic GVHD Treated with REZUROCK REZUROCK 200 mg once daily (N=83) Adverse Reaction

Table 3 summarizes the laboratory abnormalities in REZUROCK. Table 3: Selected Laboratory Abnormalities in Patients with Chronic GVHD Treated with REZUROCK REZUROCK 200 mg once daily

Parameter

Grade 0-1 Baseline

Grade 2-4 Max Post

Grade 3-4 Max Post

(N)

(%)

(%)

Chemistry Phosphate Decreased

76

28

7

Gamma Glutamyl Transferase Increased

47

21

11

Calcium Decreased

82

12

1

Alkaline Phosphatase Increased

80

9

0

Potassium Increased

82

7

1

Alanine Aminotransferase Increased

83

7

2

Creatinine Increased

83

4

0

Lymphocytes Decreased

62

29

13

Hemoglobin Decreased

79

11

1

Platelets Decreased

82

10

5

Neutrophil Count Decreased

83

8

4

Hematology

7 DRUG INTERACTIONS 7.1 Effect of Other Drugs on REZUROCK Strong CYP3A Inducers Coadministration of REZUROCK with strong CYP3A inducers decreases belumosudil exposure [see Clinical Pharmacology (12.3) in the full prescribing information], which may reduce the efficacy of REZUROCK. Increase the dosage of REZUROCK when coadministered with strong CYP3A inducers [see Dosage and Administration (2.3) in the full prescribing information].

All Grades (%)

Grades 3-4 (%)

Infection (pathogen not specified)a

53

16

Viral infectionb

19

4

Bacterial infectionc

16

4

Astheniad

46

4

Edemae

27

1

Pyrexia Gastrointestinal

18

1

Nauseaf

42

4

Diarrhea

35

5

Abdominal paing

22

1

Dysphagia

16

0

8 USE IN SPECIFIC POPULATIONS 8.1 Pregnancy Risk Summary Based on findings from animal studies and the mechanism of action [see Clinical Pharmacology (12.1) in the full prescribing information], REZUROCK can cause fetal harm when administered to pregnant women. There are no available human data on REZUROCK use in pregnant women to evaluate for a drug-associated risk. In animal reproduction studies, administration of belumosudil to pregnant rats and rabbits during the period of organogenesis resulted in adverse developmental outcomes, including alterations to growth, embryo-fetal mortality, and embryo-fetal malformations at maternal exposures (AUC) approximately ≥ 3- (rat) and ≥ 0.07 (rabbit) times the human exposure (AUC) at the recommended dose (see Animal Data). Advise pregnant women and females of reproductive potential of the potential risk to the fetus.

Dyspneah

33

5

In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.

Coughi

30

0

Nasal congestion

12

0

Hemorrhagej

23

5

Hypertension

21

7

Musculoskeletal paink

22

4

Muscle spasm

17

0

Arthralgia

15

2

21

0

Decreased appetite

17

1

Skin and subcutaneous Rashm

12

0

Pruritusn

11

0

Infections and infestations

General disorders and administration site conditions

Respiratory, thoracic and mediastinal

Vascular

Musculoskeletal and connective tissue

Nervous system Headachel Metabolism and nutrition

a

includes edema peripheral, generalized edema, face edema, localized edema, edema. includes nausea, vomiting. g includes abdominal pain, abdominal pain upper, abdominal pain lower. h includes dyspnea, dyspnea exertional, apnea, orthopnea, sleep apnea syndrome. i includes cough, productive cough. j includes contusion, hematoma, epistaxis, increased tendency to bruise, conjunctival hemorrhage, hematochezia, mouth hemorrhage, catheter site hemorrhage, hematuria, hemothorax, purpura. k includes pain in extremity, back pain, flank pain, limb discomfort, musculoskeletal chest pain, neck pain, musculoskeletal pain. l includes headache, migraine. m includes rash, rash maculo-papular, rash erythematous, rash generalized, dermatitis exfoliative. n includes pruritus, pruritus generalized. f

infection with an unspecified pathogen includes acute sinusitis, device related infection, ear infection, folliculitis, gastroenteritis, gastrointestinal infection, hordeolum, infectious colitis, lung infection, skin infection, tooth infection, urinary tract infection, wound infection, upper respiratory tract infection, pneumonia, conjunctivitis, sinusitis, respiratory tract infection, bronchitis, sepsis, septic shock. b includes influenza, rhinovirus infection, gastroenteritis viral, viral upper respiratory tract infection, bronchitis viral, Epstein-Barr viremia, Epstein-Barr virus infection, parainfluenzae virus infection, Varicella zoster virus infection, viral infection. c includes cellulitis, Helicobacter infection, Staphylococcal bacteremia, catheter site cellulitis, Clostridium difficile colitis, Escherichia urinary tract infection, gastroenteritis Escherichia coli, Pseudomonas infection, urinary tract infection bacterial. d includes fatigue, asthenia, malaise.

Proton Pump Inhibitors Coadministration of REZUROCK with proton pump inhibitors decreases belumosudil exposure [see Clinical Pharmacology (12.3) in the full prescribing information], which may reduce the efficacy of REZUROCK. Increase the dosage of REZUROCK when coadministered with proton pump inhibitors [see Dosage and Administration (2.3) in the full prescribing information].

Data Animal Data Embryo-fetal development studies were conducted in rats with administration of belumosudil to pregnant animals during the period of organogenesis at oral doses of 25, 50, 150, and 300 mg/kg/day in a pilot study and doses of 15, 50, and 150 mg/kg/day in a pivotal study. In the pilot study, maternal toxicity and embryofetal developmental effects were observed. Maternal toxicity (reduced body weight gain) occurred at 150 and 300 mg/kg/day doses. Increased post-implantation loss occurred at 50 and 300 mg/kg/day. Fetal-malformations were observed at ≥ 50 mg/kg/day and included absence of anus and tail, omphalocele, and dome shaped head.

The exposure (AUC) at 50 mg/kg/day in rats is approximately 3 times the human exposure at the recommended dose of 200 mg.

In an embryo-fetal developmental study in rabbits, pregnant animals administered oral doses of belumosudil at 50, 125, and 225 mg/kg/day during the period of organogenesis resulted in maternal toxicity and embryo-fetal developmental effects. Maternal toxicity (body weight loss and mortality) was observed at doses ≥ 125 mg/kg/day.

Embryo-fetal effects were observed at doses ≥ 50 mg/kg/day and included spontaneous abortion, increased post-implantation loss, decreased percentage of live fetuses, malformations, and decreased fetal body weight.

Malformations included those in the tail (short), ribs (branched, fused or deformed), sternebrae (fused), and neural arches (fused, misaligned, and deformed). The exposure (AUC) at 50 mg/kg/day in rabbits is approximately 0.07 times the human exposure at the recommended dose of 200 mg.

8.2 Lactation Risk Summary There are no data available on the presence of belumosudil or its metabolites in human milk or the effects on the breastfed child, or milk production. Because of the potential for serious adverse reactions from belumosudil in the breastfed child, advise lactating women not to breastfeed during treatment with REZUROCK and for at least one week after the last dose.

8.3 Females and Males of Reproductive Potential REZUROCK can cause fetal harm when administered to a pregnant woman [see Use in Specific Populations (8.1)].

Pregnancy Testing Verify the pregnancy status of females of reproductive potential prior to initiating treatment with REZUROCK.

Contraception Females Advise females of reproductive potential to use effective contraception during treatment with REZUROCK and for at least one week after the last dose of REZUROCK. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be informed of the potential hazard to a fetus.

Males Advise males with female partners of reproductive potential to use effective contraception during treatment with REZUROCK and for at least one week after the last dose of REZUROCK. Infertility Females Based on findings from rats, REZUROCK may impair female fertility. The effect on fertility is reversible [see Nonclinical Toxicology (13.1) in the full prescribing information].

Males Based on findings from rats and dogs, REZUROCK may impair male fertility. The effects on fertility are reversible [see Nonclinical Toxicology (13.1) in the full prescribing information].

8.4 Pediatric Use The safety and effectiveness of REZUROCK have been established in pediatric patients 12 years and older.

Use of REZUROCK in this age group is supported by evidence from adequate and well-controlled studies of REZUROCK in adults with additional population pharmacokinetic data demonstrating that age and body weight had no clinically meaningful effect on the pharmacokinetics of drug substance, that the exposure of drug substance is expected to be similar between adults and pediatric patients age 12 years and older, and that the course of disease is sufficiently similar in adult and pediatric patients to allow extrapolation of data in adults to pediatric patients.

The safety and effectiveness of REZUROCK in pediatric patients less than 12 years old have not been established.

8.5 Geriatric Use Of the 186 patients with chronic GVHD in clinical studies of REZUROCK, 26% were 65 years and older. No clinically meaningful differences in safety or effectiveness of REZUROCK were observed in comparison to younger patients.

Active ingredient made in India.

Distributed and marketed by: Kadmon Pharmaceuticals, LLC Warrendale, PA 15086 1-877-377-7862 REZUROCK™ is a trademark of Kadmon Pharmaceuticals, LLC. © 2021 Kadmon Pharmaceuticals, LLC, Warrendale, PA 15086. All rights reserved.

KAD25000266 10/21

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Specialty Pharmacy Continuum • May/June 2022

OPERATIONS & MANAGEMENT

Expanding Biosimilars continued from page 1

“Although there are still barriers to adoption across the board, momentum around biosimilars continues to accelerate, which can bring patients and the overall healthcare system expanded benefits through broader access to affordable lifesaving medications,” Dr. Oskouei said.

The U.S. biosimilars market has grown significantly since the FDA approved the

first biosimilar, filgrastim-sndz (Zarxio, Sandoz), in 2015. Since then, the FDA has approved 35 biosimilars, 21 of which are commercially available, Dr. Oskouei said. The vast majority of those available (17) are provider-administered oncology therapies such as bevacizumabawwb (Mvasi, Amgen), rituximab-pvvr (Ruxience, Pfizer) and trastuzumab-dkst (Ogivri, Mylan-Biocon).

The U.S. biosimilars market will continue to grow and evolve over the coming years as more types of therapies become available and patient access increases, Dr. Oskouei said. Specifically, the approval last year of insulin glargineyfgn (Semglee, Viatris/Biocon)—the first interchangeable biosimilar that may be substituted for the reference product at the pharmacy without the intervention of the prescriber—means the U.S. market is entering a new phase of biosimilar activity, she said.

“The majority of the activity has been

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in the medical benefit space with healthcare provider–administered products,” she told Specialty Pharmacy Continuum.

“The next wave will be focused on more products that enter the pharmacy benefit reimbursement model, so more selfinjectables and dispensing at retail, specialty sites of care and mail order. It’s a very exciting time.”

For example, seven adalimumab (Humira, AbbVie) biosimilars will become available in 2023, and another five are pending FDA approval. In addition, the number of biologics facing biosimilars competition will more than double by 2026, affecting mostly immunology and ophthalmology agents such as aflibercept (Eylea, Regeneron), etanercept (Enbrel, Amgen) and ustekinumab (Stelara, Janssen).

The Promise of Biosimilars According to a 2021 report from the Association for Accessible Medicines, the aggregate savings from biosimilars could exceed $133 billion by 2025, a considerable increase from the $8 billion in savings attributed to biosimilars in 2020.

“That is the promise of biosimilars: They bring competition to some of those costly biologic treatment options, thereby lowering healthcare costs and enhancing patient accessibility and affordability to these treatments,” Dr. Oskouei said during her presentation.

However, perceptions of biosimilars vary among specialties, with more hesitation among prescribers in new therapeutic areas such as diabetes, ophthalmology and rheumatology, according to a survey of 115 retail pharmacists and more than 600 physicians conducted by Cardinal Health in 2021.

Much of that hesitation is driven by a lack of understanding of biosimilars’ efficacy and when to substitute a biosimilar for a reference product, the survey showed. For example, 42% of rheumatology prescribers and 32% of ophthalmology prescribers said they are likely to prescribe a biosimilar to new patients.

However, only 11% and 32%, respectively, said they would switch a patient who is having success on a reference product.

Meanwhile, 67% of oncology prescribers said they would most likely subscribe a biosimilar to both new and existing patients, according to the survey data (Figure).

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Additional Resources FDA educational resources for providers and patients bit.ly/3yFYoDy

Cardinal Health biosimilars resources bit.ly/3yFUMkQ

www.fungitell.com • 888.395.2221 MKT#20-043

11

Specialty Pharmacy Continuum • May/June 2022

OPERATIONS & MANAGEMENT The aggregate savings from biosimilars could exceed

$133 billion by 2025—a considerable increase from the

$8 billion in savings attributed to biosimilars in 2020.

Source: Association for Accessible Medicines.

This role will continue to grow as more biosimilars become available in more therapeutic areas and sites of care expand, including retail and specialty pharmacies and mail-order services, Dr. Oskouei said.

Pharmacists also are well positioned to help navigate the financial, clinical and operational considerations that will be necessary in successfully managing the growth of biosimilars, she said.

Cost will be a significant factor in the success of biosimilars, many of which will be outpatient administered or dispensed, Dr. Oskouei said.

Cash-paying, underinsured or uninsured patients will benefit from these low-cost alternatives, so “understanding patient access and reimbursement will be even more critical when these products come to market,” she said.

From a clinical standpoint, pharmacists can help drive patient and clinical understanding and acceptance through counseling and education, she said.

“Having proactive multi-stakeholder educational efforts are key. And this is truly multi-stakeholder from physicians, pharmacists, nurses and managed care stakeholders to really ensure that

“What is interesting to note is that the top concern is efficacy with biosimilars, which highlights the need to strengthen clinical confidence in these products with education efforts,” said Dr. Oskouei, who added that 71% of survey respondents said biosimilar education materials would be helpful in conversations with patients.

In addition, lack of payor adoption is also driving hesitancy regarding biosimilars, the survey showed. Among diabetes prescribers—which includes endocrinologists and primary care physicians—72% said they would utilize or prescribe biosimilars for existing patients whom payors have mandated a biosimilar (Figure).

“So, you can see as we get to that pharmacy benefit, self-injectable product–type, even the prescribers have a greater awareness that a lot of utilization is going to be dictated by managed care,” Dr. Oskouei said.

Despite this hesitation, pharmacists already support the growth of biosimilars through activities such as patient and clinical education, pharmacovigilance services, patient counseling, and cost-effectiveness analyses, she said.

100

80

67%

60

42%

42%

38% 32%

20

30%

35%

27%

25% 20%

19% 11%

5%

5% N/A

0

New patients

N/A

7% 2%

Existing I am not likely Existing patients Existing patients to prescribe a having limited patients for having success success on a whom payors biosimilar on a reference reference have mandated for any patient product at this time product a biosimilarb

Figure. For which patients are you most likely to prescribe a biosimilar?a a b

Increased Empowerment “Although there are barriers to adoption across the board, momentum around biosimilars continues to accelerate, which can bring patients and the overall healthcare system expanded benefits through broader access to affordable, lifesaving medications,” Dr. Oskouei said.

And as biosimilars continue to grow—including an increase in retail interchangeable biosimilars—pharmacists will be called upon to help foster familiarity, confidence and acceptance among patients and providers.

“Pharmacists will be further empowered to support biosimilar adoption efforts and experiences,” Dr. Oskouei said. “Because of the expansion of pharmacists’ presence in these sites of care, pharmacists can continue to reach more patients.”

The sources reported no relevant financial disclosures.

T

$1

So

72%

40

“By having a successful biosimilar market and lowering these healthcare costs, what we’re essentially doing is bringing the healthcare dollar to invest in the next generation of innovative treatments to improve even more patient outcomes and lives,” she said.

$

Oncology prescribers n=373 Rheumatology prescribers n= 102 Ophthalmology prescribers n=102 Diabetes prescribers n=54

67%

there’s awareness, comfort and confidence in these agents,” she said.

Pharmacists also will be instrumental in operationalizing interchangeable biosimilars, but they must work within the confines of each patient’s insurance requirements.

For example, if a patient gets a prescription for a brand-name biologic but their insurance plan prefers an interchangeable biosimilar, pharmacists will be in a position to automatically substitute to the biosimilar without obtaining a new prescription from the provider, Dr. Oskouei said. “So, there are some efficiencies there,” she said.

However, each state has different rules and regulations regarding interchangeability. “Some have different requirements when it comes to documentation, how long to keep records, who you communicate to and what time frame,” Dr. Oskouei said.

“Understanding those state laws … is going to be important as these products continue to come to market.”

Pharmacists also should become active in supporting policies and policy reforms that can incentivize biosimilars and create competition among manufacturers, which will help further drive innovation, she said.

Respondents asked to check all that apply.

This answer choice was not included in oncology and ophthalmology surveys.

Source: 2022 Cardinal Health Biosimilars Report: the U.S. journey and path ahead. Cardinal Health.

Published Feb 2022. www.cardinalhealth.com/en/product-solutions/pharmaceutical-products/biosimilars/ biosimilars-report.html

12

Specialty Pharmacy Continuum • May/June 2022

OPERATIONS & MANAGEMENT

How an SP’s Distribution Expertise Benefits Payors Ana M. Cavanaugh, RPh, MBA Senior Director, National Payer Sales and Account Management, Pharmacy Services AllianceRx Walgreens Prime

L

imited distribution drugs (LDDs) are high-touch medications to which only a handful of specialty pharmacies have access. Because these medications usually have specific requirements, pharmaceutical manufacturers may choose to limit distribution of the drug to only a few specialty pharmacies. Pharmaceutical manufacturers choose a specialty pharmacy as a trusted partner because of its ability to support patients throughout their treatment journey.

What Payors Want Payors benefit from having one partner for all their specialty pharmacy needs. For payors, a specialty pharmacy’s broad access to LDDs means they will likely have the following: • End-to-end solutions. LDD access allows a specialty pharmacy to meet most of the specialty needs of its clients. For example, if the pharmacy does not have access to a product, it can work directly with the manufacturer to attempt to gain access on behalf of the patient.

• Rapid access to medications. Because there is no delay or expense related to third-party

suppliers, payors can quickly access hard-tofind medications. Being part of an exclusive or narrow distribution network can allow pharmacies to have expedient contract additions to its payors. In addition, specialty pharmacies that have deep relationships with pharmaceutical manufacturers can begin discussions early about the specialty drug pipeline and how quickly patients can access drugs once available on the market.

• Greater patient satisfaction. Central and local pharmacies operating under singular clinical programs allow for a consistent patient experience aimed at driving adherence and improving overall treatment experience.

• Reduced costs. When a specialty pharmacy manages the full drug therapy needs of patients, its pharmacists and nurses can support possible medication issues, such as side effects, that could lead to nonadherence. In turn, those patients who remain on their drug regimen provide a healthier membership to the payor, potentially reducing overall costs.

• Actionable insights. Specialty pharmacies can provide their payors regular reports outlining utilization and spending of all medications, including LDDs. Many specialty pharmacies offer real-time access to critical information through

proprietary client data and insights tools. These tools can often visually transform a payor’s current business data into interactive insights, enabling the payor to best evaluate its overall payor strategy and respond to patient needs and market trends.

Ana Cavanaugh, RPh, MBA, is the senior director of National Payer Sales and Account Management, Pharmacy Services, at AllianceRx Walgreens Prime.

More on the Web For strategies on how specialty pharmacies can help payors meet their goals, see expanded version at www.specialtypharmacycontinuum.com.

Oncology Infusion Moving From Hospital to Home

By Gina Shaw

Boston—“If your institution, department or program does not have a home infusion setup for your oncology patients, you need to start working on it.”

That was the message from Laure DuBois, PharmD, BCOP, pharmacy clinical coordinator at the University of Kansas Medical Center, in Kansas City, discussing the increasing trend toward siteof-care management at a session at the 2022 annual meeting of the Hematology/ Oncology Pharmacy Association.

“At our institution, we first observed this with Aetna wanting to transfer patients on PD-L1 [programmed death ligand-1] inhibitors to home infusion by self-injection or the physician’s office,” Dr. DuBois said. “These policies are increasingly in place for many supportive care medications, as well as some targeted therapies.”

Dr. DuBois said their hospital leaders were convinced to start a home infusion program when they were shown that they could keep and treat patients rather than send them to nationwide home infusion companies. “You can start by targeting easier supportive agents, like denosumab [Prolia, Amgen], IVIG [IV immune globulin], antibiotics and leuprolide, and then consider moving to treatments like rituximab [Rituxan, Genentech/Biogen], eculizumab [Soliris, Alexion] and pembrolizumab [Keytruda, Merck].”

A crucial step is integrating a new home infusion program into the electronic health record (EHR), she said.

“When we first started this, we had problems. Epic is our EHR and does not have a good platform for home infusion, so we started with a separate electronic ordering system. The doctors then couldn’t tell if the orders they had sent to home infusion were being fulfilled, so I hit the brakes and built home infusion as its own department in our EHR. The doctors are now able to use these order sets, make supportive care plans and treatment plans for home infusion, and then release the prescription to the home infusion pharmacy, after which they can see everything that happens.”

Dr. DuBois and her team included all supportive care medications that could be added to the plan as prescriptions, with all prescribing information built out, and then created advanced order groups in the EHR. “The physician could then select what medications they were prescribing a patient for home infusion and add them into the plan,” she said.

“We also set up take-home prescriptions for an emergency kit to be kept at home, including all possible medications

needed for line care and reactions. They were filled and left at the patient’s house during the first infusion.”

Dr. DuBois offered a few recommendations from experience: Reach out to the patient. The home infusion department should call the patient the day after a home-based therapy is supposed to be administered. “If the patient says they haven’t yet administered their pegfilgrastim, I’ll sit on the phone with them until they have,” she said.

Decouple treatments from physician visits by 24 to 48 hours. “Initially, we tried to set up the first home infusion of pembrolizumab in the afternoon following a morning physician visit,” she said. “But then, if the physician decided to hold therapy after the visit, we had already sent the drug to the house. Now we allow at least 48 hours from physician visit to infusion to allow for dose changes and held therapy.”

Be ready for weight-based dosing. This may require patients to record their weight at home as a second check, Dr. DuBois noted. “With a drug like trastuzumab [Herceptin, Roche], for example, we are comfortable with the patient doing a weight check at home, and the pharmacist asks them for their recorded weight before filling the prescription,” she said.

Prioritize patient comfort. This is particularly important with more active

therapies such as pembrolizumab and trastuzumab, Dr. DuBois stressed, adding that it is also important to help patients feel comfortable with the home infusion process. “We have established a warm handoff process from the hospital-based infusion nurse to the home nurse during the hospital visit, where the home nurse meets the patient and explains what the home infusion process will look like,” she said. “Because safety checks in the hospital setting aren’t always done in the patient’s room, they may not have seen them before. So, when the home nurse calls the hospital pharmacist to do that check, the patient may be concerned that it’s because something is wrong or they aren’t sure what they’re doing. We now make sure to educate the patient that these are the same safety checks we always do.”

Fight for exceptions to site-of-care restrictions. “With some of our new IVIG patients, we have gotten a site-ofcare denial for hospital infusion right away,” Dr. DuBois said. “We obviously don’t like to have the first dose in the home due to the possibility of unexpected reactions, so we will fight for a one-time exception to make sure they will tolerate the drug before sending it to the home.”

Dr. DuBois reported no relevant financial disclosures.

13

Specialty Pharmacy Continuum • May/June 2022

OPERATIONS & MANAGEMENT

Bon appétit!

Cooking Up an SP Drug Forecasting Tool

By Marie Rosenthal

Chicago—In 2019, specialty medications accounted for 65% of all new drug approvals, rising to 74% by 2021. With about 55% of these often expensive specialty medications coming under the pharmacy benefit, anticipating their economic impact is crucial—a process that should start even before the drug is approved, experts noted.

But how do third-party payors and pharmacy directors, for that matter, budget for the unknown? They develop a forecasting model, according to Brian MacDonald, PharmD, RPh, the director of specialty clinical strategy at Magellan Rx Management, in Middleton, R.I. He offered some tips for doing that at AMCP 2022 annual meeting.

“The growing, evolving specialty pipeline presents an opportunity for payors to assess how they anticipate the potential financial impacts brought on by these new-to market drugs,” Dr. MacDonald said.

He compared the process to cooking. First, one needs to

plan the menu, then gather the ingredients, cook the meal, and plate or present the food.

The steps in budget modeling are determining the objective of the budget (planning the menu); doing the research to find the components needed to create the model (gather ingredients); building the model when all the research is done (cooking the ingredients); and presenting the forecast (plating or presentation of the meal).

“You have to know what you are cooking, and that’s determining the objective of the budget model that we’re going to try to build. We’re going to gather our ingredients,

4 Forecasting Tips

B

rian MacDonald, PharmD, RPh, the director of specialty clinical strategy at Magellan Rx Management, in Middleton, R.I., offered additional tips for forecasting a budget.

Seek out solid data. Specifically, look to market forecasts, potential sales data or sales projections, as well as the price of any competitors that might be on the market.

Think about duration of treatment and safety. Will the specialty drug be used for one treatment, a short course or for long-term maintenance therapy? Determine the adverse event profile of the product and how that will affect clinical and financial outcomes.

Think about adherence. “The yearly cost of a drug assumes that the patient will get all of their doses, get all their refills, get all their injections, whatever it might be,” Dr. MacDonald said. “Obviously we all know this rarely happens.

Adherence is something that we have continued to chase as clinicians and managed care experts for years and years, [and will continue to do so for years] to come. So, it’s important to account for potential missed doses or lapses in adherence when we’re estimating the cost per patient per year,” he said.

Keep in mind the “side dishes.” These additional considerations include drug administration fees, laboratory fees and other costs that will be required as patients begin using the new medication.

1 2 3

4

look at the data, the values and the components that will make up our model.

We’re going to cook up those ingredients and build out a model. And then the model will be presented and then adjusted for specific scenarios,” Dr. MacDonald explained.

While one might think it’s just a matter of looking at utilization and cost, there actually are many factors or components that will go into a forecasting model, he said. When looking at utilization, disease or condition prevalence and uptake rate need to be considered.

Many sources of information on prevalence exist, such as the literature, government agencies, state associations, patient advocacy groups, and even the pharmaceutical companies themselves, Dr. MacDonald noted. When researching the prevalence rate, remember that U.S. prevalence, rather than the global one, is the most important rate for the forecasting model.

“Using comprehensive and accurate data sources to understand the drug development pipeline and projected timelines for new drug releases is an important part of accurately forecasting medication expenditures,” said James A. Jorgenson, RPh, MS, FASHP, the CEO of Visante, who was asked to comment on Dr. MacDonald’s model.

“Monitoring patent expirations, generic and biosimilar competition releases along with regularly reviewing and modeling these sources are also key components of effective drug budgeting.”

Given the significant impact of drugs on health system expenses along with revenues and margins, “an accurate budget model is a vital component of a successful health system financial system,” Mr. Jorgenson added.

Rx Uptake Can Take Time One might think that patients and prescribers would immediately want the new medication, but uptake tends to be low at first and then increase in subsequent years. If there are other medications on the market that are less expensive or have other desirable features—for instance, an oral instead of injectable formulation—uptake might be slower for a new medication.

If the new medication is the only one for a particular disease state or has fewer adverse events or less frequent dosing, it might be more desirable, and therefore uptake might be faster, Dr. MacDonald explained. Those considerations will help anticipate the cost of treatment.

One needs to also think about demographics. Who will use the drug? Will

it be used mainly by a Medicare population, children, or a racial or ethnic minority group? These are all important considerations.

“The demographic information of the patients in the clinical trials and interpreting some of these statistics can be tricky,” he warned. Sometimes it is difficult to extrapolate study data to various populations.

“You have to be discerning in terms of your evaluation of the clinical data that are out there,” Dr. MacDonald said, adding that it is also important to consider how social determinants of health could affect clinical trial enrollment, which “could really impact how the future [economic] models turn out.”

The cost of treatment may be one of the hardest ingredients to get. “This is an important factor and a crucial component of the model,” he said. “So, tying back to what we we’re just talking about regarding prevalence, sales estimates can give us an idea of how much a treatment may cost per patient. If we have a good feeling about how many patients may be treated, we want to know what an annual cost of treatment might be for each patient.

“Having put all this together, we can say the meal is ready to serve and we’re all ready to be expert modelers,” Dr. MacDonald quipped.

He admitted it takes some practice, but insisted that modeling is something that many people can do. However, there also are several companies and services that specialize in modeling and projections for specialty medications.

Although this is a budget “recipe” that pharmacy directors could follow, too, it is not a process that is only done once a year. Pharmacy directors need to always have their eyes on the bottom line, Mr. Jorgenson warned.

“An effective budgeting model for medications within a health system should be a continuous process and not just a one-time event each year at budget time,” he explained.

“Understanding potential shifts in patient populations and demographics, strategic organizational initiatives that may emphasize different disease states, provider recruitment plans, shifts in payors or payor policies, manufacturer price changes, the impact of new therapy on total cost of care and the net cost of the drugs are a few of the elements that should be considered well in advance of the release of a new drug to the market.”

The sources reported no relevant financial disclosures.

14

Specialty Pharmacy Continuum • May/June 2022

OPERATIONS & MANAGEMENT

Healthcare services also recovering

Drug Launches Persist Amid Pandemic

By Karen Blum

Chicago—Healthcare offices readjusted to a new normal in 2021 during the peak of the COVID-19 pandemic, but new drug launches remained strong, according to a keynote address at the AMCP 2022 annual meeting. Specialty drugs could take the majority of the market this year.

Almost 1 billion diagnostic medical visits did not happen in 2020, said Douglas Long, MBA, the vice president of industry relations for IQVIA, a pharmaceutical information company, although visits have been steadily increasing since then. Looking at cancer alone, in the peak pandemic period of April 2020, there were 87% fewer mammograms, 90% fewer colonoscopies and 83% fewer Pap

testing, are less likely to prescribe new medications, Mr. Long said.

IQVIA gave the healthcare ecosystem’s overall response to COVID-19 an “A” grade, but Mr. Long said he would boost that to an A-plus: “The research and development coming out with vaccines in record time is totally remarkable,” he said.

“Then [the focus shifted to] repurposing old treatments to see if they would work.”

2021, increasing year-over-year growth by over 399%.

In 2021, antidiabetics were the top therapy class when including multiple classes of trade, with $69 billion in sales and a year-over-year growth of 16%.

The top three products overall were the monoclonal antibody adalimumab (Humira, AbbVie), with $27.5 billion in sales and 14.7% year-over-year growth; the anticoagulant apixaban (Eliquis, Bristol Myers Squibb), with $15.8 billion in sales and 23.2% year-over-year growth; and the antidiabetic medication dulaglutide (Trulicity, Lilly), with $12.2 billion in sales and 40.2% year-over-year growth. (For 2020 data, see Table.)

Drilling down to the retail market, the top three products were apixaban, with $12 billion in sales and 23% year-overyear growth; dulaglutide, with $10 billion in sales and nearly 41% year-over-year growth; and the antidiabetic medication semaglutide (Ozempic, Novo Nordisk), with $7.3 billion in sales and year-overyear growth of nearly 67%.

Protected brand-name pharmaceutical

list prices increased 4.4% in 2020, while net prices decreased by nearly 3%, Mr. Long said—the fourth year at or below the Consumer Price Index.

Although there was a –0.7% change in prices for prescription drugs in October 2021 compared with the prior year, the costs rose for physician services, hospital care and other personal healthcare.

“The perception is that healthcare is very expensive, which it is, but it’s not due to drug costs,” he said. “What’s driving it is hospital services, which have inflated by more than 200% since the year 2000.”

Generic sales continued to decline— by 5% as of January 2022—because of price deflation, Mr. Long noted.

Generics constituted 85% of prescriptions in 2021 but accounted for only 9.5% of dollars spent.

The market also is in “the sweet spot” for biosimilars, he said, with projected savings resulting from biosimilars estimated to reach $104 billion over the next five years. This is largely due to the patent expiration on adalimumab,

Table. Top 10 Performers in 2020

smears, as medical offices were closed.

That started to recover through the first quarter of 2021 but not to the level expected, he said: Overall numbers were still down by 14% to 25%. Specialties with the biggest gaps in care included endocrinology, rheumatology, primary care and oncology, Mr. Long said.

Susan Cantrell, RPh, the CEO of AMCP, saw similar trends emerge in the group’s own research. In May 2020, AMCP collaborated with the Alliance of Community Health Plans to conduct a consumer survey about healthcare perceptions and concerns. “We saw from that early data that there was going to be a long-term issue where people were delaying visits, surgeries and procedures,” she said. “And we started to really see that come to reality.”

The ups and downs of telehealth was another key trend cited by Mr. Long.

Throughout 2021, he noted, telehealth visits dropped by an average 0.4% per week, while institutional office visits grew by 1% to 1.5% per week. Prescription volume could pick up as this continues, as physicians seeing patients over telehealth, without access to laboratory

Manufacturing also continued, he said, even by generic manufacturers in China, despite COVID-19 pressures. Another supply squeeze occurred when plants in India shut down due to labor shortages.

Marketplace Trends Specialty pharmaceutical growth is outpacing traditional growth, and now has about a 49% share of total, nondiscounted spending, Mr. Long said. The year 2022 could be when specialty pharmaceuticals will achieve a greater share of the market than traditional products, he said: “The reason it hasn’t changed yet is because COVID-19 vaccines were considered traditional products.” (At a subsequent presentation at AXS22/ Asembia Specialty Pharmacy Summit 2022, Mr. Long noted that specialty has indeed passed traditional pharmaceuticals in market share.)

Immunology products and anticoagulants are showing the greatest one- and five-year growth patterns in the United States, followed by antidiabetic and oncologic drugs. Nervous system disorder products have exhibited the greatest specialty growth in retail in

Product

Company

Indication

Launch

Year 1 sales, $

Year 1 NBRx

Veklury (remdesivir)

Gilead

COVID-19

October

4.0B

NAa

Tepezzab Horizon (teprotumumab-trbw)

Thyroid eye February disease

556.9 million

NA

Ubrelvy (ubrogepant)

Allergan

Migraine

January

501.5 million

191,400

Nurtec (Rimegepant)

Biohaven

Migraine

March

406.6 million

180,700

Kesimptab (ofatumumab)

Novartis

Multiple sclerosis

September 280.3 million

5,100

Ruxience (rituximab-pvvr)

Pfizer

NHL, CLL, MPA

February

279.6 million

NA

Trodelvy (sacituzumab govitecan-hziy)

Immunomedics

Breast cancer

April

204.1 million

NA

Breztri Aerosphere Astra (budesonide, Zeneca glycopyrrolate and formoterol fumarate)

COPD

September 180.2 million

111,900

Zirabev (bevacizumab-bvzr)

Pfizer

MCRC, NSCLC, rGBM, mRCC, CC

January

89.0 million

NA

Phesgob (pertuzumab, trastuzumab and hyaluronidase-zzxf)

Genentech

Breast cancer

July

82.4 million

NA

CC, cervical cancer; CLL, chronic lymphocytic leukemia; COPD, chronic obstructive pulmonary disease; MCRC, metastatic colorectal cancer; MPAS, microscopic polyangiitis; mRCC, metastatic renal cell carninoma; NA, not available; NBRx, new prescriptions; NHL, non-Hodgkin lymphoma; NSCLC, non-squamous non-small cell lung cancer; rGBM, recurrent glioblastoma. a

Not available or likely understated due to non-retail and non-mail channel distribution.

b

Likely understated due to product reporting blocks.

Source: Douglas Long, MBA, of IQVIA at the AMCP 2022 annual meeting.

15

Specialty Pharmacy Continuum • May/June 2022

OPERATIONS & MANAGEMENT

What’s In, What’s Out And What’s Next in the Healthcare Marketplace What’s In • • • • •

COVID-19 testing and vaccines Biosimilars Specialty innovation Generic price deflation Net brand-name drug prices declining

What’s Out

companies because they require fewer people and smaller populations.”

A ‘Quite Remarkable’ Response Learning about how the country managed COVID-19 and trends in drug spending were important to Helen Lee, PharmD, MBA, the senior director of pharmacy services for Alameda Alliance for Health, in California, who was asked to comment on the presentation. “It was quite remarkable how pharmacists were able to contribute to move up the vaccination rates when the entire country

was kind of struggling, so that was really a great achievement,” she said.

Dr. Lee said she also was impressed by the data showing that the use of telehealth has been shrinking by an average 0.4% per week while office visits increased by 1% to 1.5% per week in 2021. “People clearly prefer face-to-face office visits,” she said. “We really want to empower our patients to proactively look out for their health—and we are here to help with that.”

Beyond healthcare appointments, the pandemic affected mental health

and substance use disorders, AMCP’s Ms. Cantrell said, pointing to data from Mr. Long indicating a 16% increase in prescription opioid deaths since the start of the pandemic, following 28 months of decline. “The societal and overall health impacts will just be tremendous,” she said, “and I think that’s something that we’ll be grappling with for a long time.”

The sources reported no relevant financial disclosures other than their stated employment.

• Double-digit brand-name price increases • Traditional brand innovation

What’s Next • Biosimilars for adalimumab (Humira, AbbVie) and ustekinumab (Stelara, Janssen) in 2023 • New Alzheimer’s drugs • COVID-19 booster uncertainty

Free CE/CME now available!

1.0 AMA PRA Category 1 Credit™ 1.0 AANP credit 1.0 ACPE credit 1.0 ANCC credit

followed by ustekinumab (Stelara, Janssen). Several biosimilars for adalimumab are expected to launch in 2023.

New Product Launches Pharmaceutical companies continued to bring new products to market despite uncertainty introduced by the pandemic, Mr. Long said. There were 74 launches in 2021, of which 35% were for oncology products, 15% for infectious diseases and 12% for central nervous system disorders. The biggest of these was for semaglutide (Wegovy, Novo Nordisk), a weight loss drug, which achieved $602.5 million in sales, followed by sotorasib (Lumakras, Amgen), for non-small cell lung cancer, which achieved $63.7 million in sales.

“The biggest challenge is that the launch trajectory of these products is not anywhere near what it was before, because for a long period of time, you couldn’t see doctors face to face; you couldn’t sample them,” he said. “A lot of this had to do with digital launches. If you had a product that required a lot of titration or multiple doctor visits, that was extremely difficult.”

Two of the top three potential drug launches for 2022 are Alzheimer’s drugs—LY3002813 (donanemab, Lilly) and RG1450, RO4909832 (gantenerumab, Genentech)—Mr. Long said, citing a top 10 list from Fierce Pharma. No. 2 on the list is the diabetes drug LY3298176 (tirzepatide, Lilly). Rounding out the list are medications for psoriasis, chronic kidney disease, asthma and other conditions.

“What’s interesting is that it’s a mix of big and small companies,” he said.

“It used to be that Big Pharma was the commercial engine of launches. Now some of these can be done by smaller

Safe Opioid Prescribing A Patient-Centered Approach to the FDA Blueprint A 3-Part Series RELEASE DATE: OCTOBER 27, 2021

EXPIRATION DATE: DECEMBER 31, 2022

This activity is jointly provided by Global Education Group and Applied Clinical Education.

CHAIR Charles E. Argoff, MD

FACULTY These activities are supported by an independent educational grant from the Opioid Analgesic REMS Program Companies.

Please see https://opioidanalgesicrems.com/Resources/Docs/List_of_RPC_Companies.pdf for a listing of REMS Program Companies.

This activity is intended to be fully compliant with the Opioid Analgesic REMS education requirements issued by the U.S. Food and Drug Administration (FDA).

Yvonne M. D’Arcy, CRNP, CNS Marc R. Gerber, MD Courtney M. Kominek, PharmD, BCPS, CPE Bill H. McCarberg, MD, FABM

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16

Specialty Pharmacy Continuum • May/June 2022

OPERATIONS & MANAGEMENT

Tips for Preventing Patient Data Breaches

By Dave Doolittle

Las Vegas—As pharmacists’ role in healthcare continues to expand, access to patient data is more important than ever. But using and accessing data—via patient portals, electronic health records, texts and emails—puts pharmacies at greater risk for cyberthreats, data breaches and leaks.

“It’s not just about good patient care; it’s about protecting data,” said Shawn Griffin, MD, during the AXS22/Asembia Specialty Pharmacy Summit 2022. “And your biggest risk is probably not a cyberattack. It’s more cyber-sloppiness in your organization because in most networks, the biggest risk is the person at the keyboard.”

Before becoming the president and CEO of URAC, Dr. Griffin served as the vice president for clinical performance improvement and applied analytics at Premier Inc., and as chief quality and informatics officer for several major healthcare institutions.

Under coordinated care models, pharmacy staff have access to and are sharing a wide variety of patient data, from identifying information such as name and date of birth to protected health information such as diagnoses, medical history, comorbidities, clinical assessments, lab results and clinical outcomes.

Any of this information can be compromised, stolen or held hostage, which can lead to hefty fines, lawsuits and a damaged reputation, according to Dr. Griffin, who outlined how pharmacies can best protect patients and their practices.

“We’ve gotten past the point where our technical support can be a 13-year-old kid in your family,” Dr. Griffin said. “This is getting big, and this is getting serious.

Medical providers have been facing this

now for decades, and they’re not doing a great job with it, so welcome to the party.”

“If your data’s on a server in your pharmacy, how do you keep people away from taking the server and running out the back door?” Dr. Griffin asked. “We’ve all seen the stories of hospital systems with big IT departments picking up a virus and getting all of their systems locked up, and they can’t get to their data. Are you prepared for that?

How are you preventing that?”

According to Dr. Griffin, pharmacies can protect data by: • keeping data on a secure backup system; • storing data off-site; • limiting staff access to data exclusively to those who need it; • ensuring clear roles for anyone who has access to data; • testing interconnected systems regularly; and • encrypting data.

Pharmacies also should conduct regular risk assessments as well as test their business continuity plans, Dr. Griffin said.

“The time to test your business continuity plan is not when you’re having a business continuity issue,” he said. “[That] plan is your data parachute. You really want to develop it when you’re not in a rush, when everything’s not on fire, and when regulators aren’t knocking on your door because of a data breach.”

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Pharmacies also need to not only protect themselves from ransomware and malware attacks, but they need to have plans in place in case they do fall victim to one of these attacks, Dr. Griffin said.

“Do you have a ransomware policy?

If your stuff all got locked up and they wanted $5,000 in Bitcoin, would you do it? Should you do it? Do you know how to get Bitcoin?” he said.

Data-sharing and interoperability not only improve patient care and outcomes, but they are a requirement under the 21st Century Cures Act. That makes protecting patient data among pharmacists’ biggest priorities, said Dr. Griffin, who emphasized the importance of understanding where weaknesses lie.

“Some of you will crash and burn— and by the way, that’s part of the learning curve is crashing and burning,” Dr. Griffin said. “You want to do it when you’re sitting in the sandbox, not when you’re out on the battlefield. Make small mistakes early, work out the bugs and move on. Failure is not fine. Serious legal implications and monetary fines are possible for big failures. So, get to work on it. Really look at your workload. Look at your tools. Look at your connections. Look at the data that you have coming in.”

The sources reported no relevant financial relationships.

URAC-HOSP Partnership Focuses on Specialty Pharmacy, Telehealth Accreditation LAS VEGAS—A new partnership between Health System Owned Specialty Pharmacy Alliance (HOSP) and the accreditor URAC will make specialty pharmacy and telehealth accreditations more affordable for HOSP members while fostering collaboration between the organizations.

The partnership, announced during the AXS22/Asembia Specialty Pharmacy Summit 2022, will broaden the scope of benefits for HOSP members and allow the two organizations to work together on accreditation and certification measures, HOSP Board Chairman Gary Kerr, PharmD, told Specialty Pharmacy Continuum.

“We aim to educate our members and provide them with the tools they need as well as find new ways to work with partners like URAC in the integrated specialty pharmacy ecosystem,” Dr. Kerr said. “Education and collaboration around accreditation also support the HOSP mission to share common experiences and knowledge with industry partners, so we can grow and find timely solutions to our challenges together.”

As telehealth use increases in the wake of the COVID-19 pandemic, telehealth accreditation continues to evolve as well.

“This increase in utilization applies to how pharmacies manage patients and medication adherence during the pandemic,” Jeffrey Carr, URAC’s vice president, business development, said in an email. “URAC does not want price to be a barrier to improving quality. Our partnership allows HOSP members a more affordable option to pursue new pharmacy and telehealth accreditations.”

HOSP is a nonprofit coalition of major health systems focused on integrated specialty pharmacy best practices and advocacy.

“We select partners who align with our commitment and efforts to strengthen patient care through the integrated specialty pharmacy model,” Dr. Kerr said. “Our partners understand the value and importance of collaboration in the advancements in research, advocacy and education that are required to advance the quality of care to patients.”

—Dave Doolittle

17

Specialty Pharmacy Continuum • May/June 2022

OPERATIONS & MANAGEMENT

Cornstarch Container Eases Environmental Concerns

By Karen Blum

A move from plastic foam to cornstarch-based coolers to ship refrigerated medications cost the University of Kentucky (UK) Specialty Pharmacy a little bit more in dollars but is reaping rewards in greener operations and improved patient satisfaction, pharmacists there have found.

“We were shipping out hundreds of Styrofoam coolers all across the state of Kentucky every day,” said Brooke Rauch, PharmD, the associate director of operations for the UK Specialty Pharmacy and Infusion Services team, in Lexington.

“We were starting to get concerned about our carbon footprint and there not being a way to appropriately dispose of those coolers. So, we decided to investigate some greener options.”

One of the first products the team heard about was cornstarch coolers manufactured by Green Cell Foam in Michigan. The containers are made from 100% non-GMO, U.S.-grown corn.

The material is certified compostable in backyard and industrial facilities and can be dissolved in a sink for safe, easy disposal, according to the company website.

The team tested the packaging slowly over a 10-month period to ensure it would work well in different types of weather, Dr. Rauch said. In December 2019, they made the switch, using the new coolers plus frozen gel packs that sweat less, so they don’t affect the box, which decomposes when exposed to water.

A year later, the team compiled statistics on cost, plastic foam avoidance and patient satisfaction. Anonymous surveys were sent to every patient receiving a cooler package in December 2020, asking about their patient satisfaction with the coolers, the importance of environmentally friendly packaging and how patients disposed of their coolers. Patients could complete the survey online using a QR code or by prepaid mail.

The pharmacy avoided 494 cubic yards of plastic foam use, but the annual price increased about 9%, according to a poster presented at the ASHP 2021 Midyear Clinical Meeting and Exhibition, held virtually. “It ended up being slightly more expensive for us in the long run,” Dr. Rauch said. “But overall, we decided that the change wass worth the investment to just be more re environmentally conscious in our day-today work.”

Approximately 1077 patient surveys were received.

Aboutt 90.7% of patients said id being environmentally ally friendly was important tant

to them, and 87.8% were satisfied with the cornstarch-based coolers. About half said they used alternate disposal options besides the trash.

“Changing from Styrofoam-based coolers to cornstarch-based was never a cost initiative; rather, it was an opportunity to improve patient satisfaction while implementing more environmentally friendly packouts,” said lead study author Sara Jo Santangelo, a PharmD candidate at the university.

The packaging may not work as well in areas of the country with high amounts of humidity, Dr. Rauch cautioned. The team plans to provide additional education to patients and employees on the environmentally friendly benefits of the packaging, and to explore alternative packaging options for specialty medications that do not require refrigeration during transit, Ms. Santangelo said.

Sign up @ SpecialtyPharmacyContinuum.com/ Registration Get the latest news FREE from the most widely read specialty pharmacy publication in the United States, including multimedia and web-only content, delivered directly to your inbox!

Inova Health’s Experience Inova Health System, in Falls Church, Va., also decided to use the Green Cell Foam packaging when launching its specialty pharmacy in 2019. It’s now using these packages for about 300 patients per month. Inova previously had not sent out any temperature-sensitive packing and shipping, said Debra Brooks, BS Pharm, the health system’s director of retail and specialty pharmacy.

“We knew we were just starting our specialty program and would need to choose a temperature-sensitive product for shipping,” Ms. Brooks said. “Inova thought about the long-term environmental impact of some other types of packaging and chose to go with Green Cell Foam because as we grow our business, we are committed to reducing our carbon footprint, reducing waste and saving resources.”

Patients “have been very supportive of this ggreen p product,” she added. “They the fact that it is easappreciate th ily dissolv dissolved in water or they use it in their plant can us beds.” bed The sources reported no relevant financial disclosures.

A don’t-miss resource for health systems interested in specialty pharmaceuticals and services.

Brought to you by the same team who publish

18

Specialty Pharmacy Continuum • May/June 2022

CLINICAL

Predicting the Cost of NASH Therapies desaturase-1 (SCD1) inhibitors; pegylated fibroblast growth factor-21 analogs; apoptosis signal–regulating kinase-1 (ASK1) inhibitors; and galectin-3 inhibitors (Nat Rev Gastroenterol Hepatol 2021;18:373-392).

Some drugs that have failed as monotherapy for NASH are still being studied as part of dual or triple therapies, Dr. Lugo noted. For example, selonsertib (Gilead), an ASK1 inhibitor, and elafibranor, a PPAR agonist, are being continued in trials as part of combination therapies.

Phase 2 trials are being conducted in many additional agents, Dr. Lugo said, including the first potential triplecombination therapy of semaglutide (Ozempic, Novo Nordisk), a subcutaneous GLP-1 receptor agonist, with the FXR agonist cilofexor and the ACC inhibitor firsocostat (Gilead) (box).

Belapectin (Galectin): An IV galectin-3 inhibitor

with NAFLD (61,332 individuals) and NASH (13,645 individuals) diagnoses.

They also reviewed usage of medications for diabetes such as dapagliflozin (Farxiga, AstraZeneca), obeticholic acid (Ocaliva, Intercept), odevixibat (Bylvay, Albireo), pioglitazone and semaglutide.

Rates were fairly low, ranging from 0% to 1.3% of beneficiaries with NAFLD and 0% to 2.8% of those with NASH. Next, Dr. Palmrose’s team incorporated cost estimates from the Institute for Clinical and Economic Review (ICER) on the long-term cost-effectiveness of obeticholic acid for NASH. The estimated cost is $219.96 per dose for federal agencies, or $80,340 per year.

The published ICER model posted prices for a quality-adjusted life-year (QALY) threshold of $50,000, $100,000 or $150,000 per year, treating an estimated 3 million patients over five years, and with a budget impact threshold of $819 million per year for new drugs used. In this model, ICER estimated that only 4.2% of patients could be treated before reaching the QALY threshold of $50,000 if the annual per-patient cost was $11,800. At an annual per-patient cost of $16,000, only 3% of beneficiaries could be treated before reaching the QALY threshold of $150,000.

Payors can use data points such as these as starting points to estimate budget impact and try to determine the level at which it would be difficult for the health system to absorb without displacing other needed services, Dr. Palmrose said. Building on the ICER analyses, TRICARE estimated it could treat 10.8% of beneficiaries receiving dapagliflozin at a yearly cost per patient of $4,752 before reaching their impact threshold, or 100% of beneficiaries receiving pioglitazone at a yearly cost per patient of $84 before reaching their impact threshold, he said, citing some examples.

Payors can evaluate their potential patient populations for NASH medications based on risk factors among their beneficiaries such as age, obesity, diabetes and dyslipidemia, Dr. Lugo added, and be prepared to expect “a significant cost to the health system.” Work with pharmacoeconomics specialists to put together models, evaluate the potential budget impact, develop any prior authorization criteria focusing on criteria in the clinical trials and “come up with a good definition of response to ensure you’re getting the most bang for your buck out of these products,” she advised.

Cenicriviroc (Allergan): An oral CCR2/5 dual antagonist being studied in combination with the FXR agonist tropifexor (Novartis) in a phase 2b study

Dr. Lugo reported no relevant financial disclosures. Dr. Palmrose reported holding stock in Walgreens Boots Alliance.

By Karen Blum

Chicago—The race continues for pharmaceutical companies to gain FDA approval for a drug for nonalcoholic steatohepatitis (NASH), speakers said during the AMCP 2022 annual meeting.

Payors should plan ahead to estimate the effects on their budgets.

“There’s so many people with their hands in the pot trying to get a product approved,” said Amy Lugo, PharmD, BCPS, a clinical pharmacy specialist and formulary manager for the Defense Health Agency Pharmacy Operations Division. “It’s important to stay vigilant, focusing on ClinicalTrials.gov, with how many different sponsors are in the game.”

As of January 2022, there were 86 drug-related trials for NASH in phases 2 and 3 from 62 sponsors, Dr. Lugo said, as well as 11 phase 4 studies of currently marketed products. An estimated 22% of the gastroenterology drug pipeline is for liver disease therapies, she said, citing April 2019 data from IQVIA. “Eventually, something is going to get FDA approved, and likely it’s going to have an orphan designation, which oftentimes allows for the manufacturer to charge a higher price,” she added.

NASH is a subset of nonalcoholic fatty liver disease (NAFLD), a condition of excess fat buildup in the liver, Dr. Lugo said. It is marked by inflammation, liver fibrosis and sometimes cirrhosis. About one-fourth of U.S. adults, 75% of obese individuals and 10% of children aged 2 to 19 years have NAFLD (Hepatology 2016;64[1]:73-84). This represents the potential patient pool that could progress to NASH, she said. An estimated 5% to

6.5% of U.S. adults have NASH, and 20% of those patients will develop cirrhosis.

Risk factors include age (40-60 years), obesity, diabetes, high cholesterol and ethnicity, with Hispanics having the highest prevalence (Cochrane Database Syst Rev 2017;3[3]:CD011640), Dr. Lugo said. “Given the high prevalence of NASH risk factors in the U.S. population, one could assume the patient pool for a NASH drug to be tremendous,” she said.

With no currently approved therapies, the mainstays of treatment include lifestyle modifications such as dietary changes and exercise, weight loss and control of diabetes, high cholesterol, and cardiovascular health.

The three main targets among NASH drugs in development are to treat fibrosis, the metabolic pathway or inflammation, Dr. Lugo said. A variety of agents are being studied, she said, including farnesoid X receptor (FXR) agonists; peroxisome proliferator–activated receptor (PPAR) agonists; glucagon-like peptide-1 (GLP-1) receptor agonists and sodium– glucose cotransporter-2 (SGLT2) inhibitors; ileal bile acid transporter (IBAT) inhibitors; acetyl coenzyme A carboxylase (ACC) inhibitors; dual C-C chemokine receptor types 2 and 5 (CCR2/5) antagonists; thyroid hormone receptorbeta (THR-beta) agonists; stearoyl-CoA

Phase 3 Trials to Watch Odevixibat: An oral IBAT inhibitor approved for progressive familial intrahepatic cholestasis, a rare progressive liver disease Obeticholic acid: An oral FXR agonist approved for the treatment of primary biliary cholangitis Dapagliflozin: An oral SGLT2 inhibitor approved for the treatment of type 2 diabetes, heart failure and chronic kidney disease Arachidyl amido cholanoic acid (Aramchol, Galmed): An oral SCD1 inhibitor MGL-3196 (Resmetirom, Madrigal): An oral THR-beta agonist Selonsertib: An oral ASK1 inhibitor being studied in combination with the FXR agonist cilofexor and the ACC inhibitor firsocostat Oltipraz (PharmaKing): An oral liver X receptoralpha inhibitor

Data for Payors It’s difficult to estimate what the budget impact will be of any approved drugs, said Gregory Palmrose, PharmD, PhD, BCACP, the chief of enterprise integrated analytic solutions for the Defense Health Agency Pharmacy Operations Division. But there are some limited data available payors can use to conduct forecasts.

TRICARE, the military’s health plan, has some 9.6 million beneficiaries, Dr. Palmrose said, 71% of whom used the pharmacy benefit in fiscal year 2021. To estimate the impact of NASH agents once approved, his team looked at their demographics of beneficiaries

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20

Specialty Pharmacy Continuum • May/June 2022

CLINICAL

Payor PDT Pushback

10 PDTs have been cleared by the FDA for use in the management of behavioral health disorders.

continued from page 1

PDTs on the market, she stressed. The ADHD therapeutic, EndeavorRx (Akili Interactive), is a case in point. The immersive video game was approved June 15, 2020, as an add-on therapy for children 8 to 12 years of age with ADHD. It uses a selective stimulus management engine to target areas of the brain that play a key role in attention function, Ms. Hornung explained.

In randomized controlled clinical trials involving more than 600 children with ADHD, EndeavorRx improved key measures of objective attention. “Not all of the results were statistically significant, but there was always a response across the five studies included in the analysis,” Ms. Hornung said. “The common side effects were no different than your typical pharmaceuticals on the market, such as dizziness, frustration, etc. The point is there are peer-reviewed clinical trials demonstrating efficacy, and yet payors are still holding back on reimbursing for EndeavorRx, as well as other PDTs.”

That reluctance would be easier to understand if PDTs were expensive, but that’s not the case with EndeavorRx, Ms. Hornung said. “The cost for Endeavor is $150 a month for patients with no insurance. If covered, it would be less than $100 a month, and additional patient assistance is also available. Yet, Anthem says it’s not considered medically necessary; Aetna says it’s experimental and investigational; and UHC [United Healthcare] says it’s unproven and not medically necessary.”

If cost isn’t the obstacle, then why the reluctance to pay for PDTs? “I keep coming back to the approval process, based on what we saw in the payor survey and anecdotally,” Ms. Hornung said. “Yes, these software programs and games are approved by the FDA, but it’s not via the agency’s usual process for reviewing and approving drugs.”

Instead, she noted, the review process for some PDTs is coordinated by the FDA’s Digital Health Center of Excellence, “which is not as familiar to payors as the advisory committees that review conventional therapeutics,” Ms. Hornung said. “So, the payors feel held back because they don’t believe that there’s a formal standard regulatory process for reviewing those studies.”

Ms. Hornung added a personal account of just how much of a missed opportunity this payor resistance to covering The Canvas Dx system includes a questionnaire completed by a video analyst who reviews two videos of the child with autism spectrum disorder recorded by the parent/caregiver.

The Promise and Pitfalls Of PDTs in Mental Health

P

rescription digital therapeutics (PDTs) hold value for patients whose conditions cannot effectively be treated by medications alone—particularly mental health disorders, according to Michael Angelini, PharmD, BCPP, a professor of pharmacy practice at Massachusetts College of Pharmacy and Health Sciences, in Boston.

Indeed, only 65% of patients respond to antidepressants, dropout rates of participants in schizophrenia trials can be as high as 66%, and drugs such as lithium can cause permanent renal impairment in up to 20% of patients treated chronically, he noted during a session on PDTs at the AMCP 2022 annual meeting.

PDTs, in contrast, offer several advantages: There are no drug interactions or pharmacologic intolerabilities, and they could increase access to care and adherence to treatment plans, Dr. Angelini said.

However, there also are several limitations, including patients who stop using the product because they get bored or don’t understand how it functions. These tools can produce vast amounts of data that need interpretation. Moreover, they are not necessarily appropriate for acute crises, such as psychotic episodes.

—Karen Blum

Approximately

137 PDTs are in the pipeline,

68% of which are intended for psychiatric and neurologic conditions.

The market is projected to grow by

23% between 2021 and 2028.

Source: Magellan Rx Management.

EndeavorRx (Akili Interactive) uses a selective stimulus management engine to target areas of the brain in children affected by attention-deficit/hyperactivity disorder.

EndeavorRx represents.

“I have a stepson who has ADHD,” she said. “He has run through the gamut of pharmaceuticals that are available for him to use. He’s been on combination therapies, cognitive behavioral therapy, and I’m not sure we ever hit on the ideal intervention.

So, something like EndeavorRx offers hope that there is another product in our arsenal for patients with ADHD like my stepson to try. We just have to get the payors on board for this type of technology.”

Unfortunately, “if you look at the national analytics database on EndeavorRx coverage, it shows exactly what we’ve been talking about,” Ms. Hornung noted. “Only 25% of payors have coverage for it, or coverage with a prior authorization.”

Other PDTs Facing Opposition The PDT for autism spectrum disorder (ASD), Canvas Dx (Cognoa), is another lost opportunity to improve patient care, Ms. Hornung noted. The FDA approved the device on June 2, 2021. Also known as the Cognoa ASD Diagnosis Aid, the technology uses machine learning-based software to help healthcare providers diagnose ASD in children 18 months through 5 years of age who exhibit symptoms of the disorder.

Ms. Hornung cited a pivotal clinical trial in 425 patients that compared the assessments made by Canvas Dx with those made by a panel of clinical experts who used the current standard ASD diagnostic process. The study showed Canvas Dx provided a “Positive for ASD” or “Negative for ASD” result to aid in making a diagnosis in 32% of patients. For trial participants with a positive or negative ASD result, Canvas Dx results matched the panel’s conclusions for 81% of patients who tested positive for ASD by the software and 98% of patients who tested negative for ASD by the software.

In addition, Canvas Dx made an accurate ASD determination in 98.4% of patients with the condition and 78.9% of patients without it, she noted.

“Can you imagine what we can do if more payors acknowledged these data and covered the device?” Ms. Hornung said. “If we could intervene with these kids at an early age, as the results suggest may well be possible, how much more highly functional would they be if we got them to treatment? Yet, according to our own survey as well as national payor data, insurers just aren’t covering this PDT.” A closer examination of the FDA approval process for Canvas Dx offers some clues as to why payors may be reluctant to cover the software. The agency assessed it through the de novo premarket review pathway “for low- to moderate-risk devices of a new type,” the FDA stated in a press release announcing the approval. “This action creates a new regulatory classification.”

Similar to other PDTs on the market, Ms. Hornung noted, this new approval pathway may have made some payors reluctant to embrace the technology.

As for the OUD software that Ms. Hornung chose to cite as another missed PDT opportunity, she pointed to the reSET-O app (Pear Therapeutics).

Approved by the FDA in December 2018, the app is intended to be used in addition to outpatient treatment from a health professional, in conjunction with buprenorphine and behavior modification therapy.

In clinical trials that led to the app’s approval, 82% of people with OUD who added reSET-O to their buprenorphine stayed in treatment versus 68% who did not.

Despite those data, “UHC says the app is unproven and not medically necessary,” Ms. Hornung said. Similarly, Aetna deemed the app as “experimental and investigational,” and national payor data show that attitude is fairly

21

Specialty Pharmacy Continuum • May/June 2022

CLINICAL

On the legislative front, the Access to Prescription Digital Therapeutics Act of 2022 (H.R. 7051 and S. 3791) has bipartisan support. The legislation includes several components that could help overcome payor reluctance, including the establishment of clearer payment codes and policies that would allow coverage

by Medicare and Medicaid, she noted.

“That’s huge, because payors have told us the uncertain reimbursement picture for PDTs is yet another reason for their reluctance to cover them,” Ms. Hornung said.

prevalent across the board, she noted.

Ms. Hornung lamented that payor stance. “Who in this audience can say they don’t know someone … who has been affected by the ongoing opioid epidemic in the United States today? This is an app that can help, based on solid clinical data. Yet, it’s not being covered adequately.”

In addition, advocacy efforts are gaining momentum, including the Digital Therapeutics Alliance, which was founded in 2017 to engage payors.

“Their sole mission is to increase awareness of digital therapeutics,” she said.

Given the potential for PDTs to make a difference in patients’ lives, “that’s a huge development that hopefully will yield results soon.”

These efforts are sorely needed because “digital health is becoming an essential part of how healthcare is delivered,” Ms. Hornung said. “It’s

very likely that [PDTs are] going to grow exponentially, and we expect further research and evidence to support these technologies. Digital health and digital therapeutics are here to stay and will help support patients through their new unique healthcare journey.”

—Additional reporting by Karen Blum The sources reported no relevant financial disclosures other than their stated employment.

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Gaining Some Traction Soumya Vishwanath, PharmD, the senior manager of formulary strategy for Magellan Rx Management, agreed that reimbursement for PDTs has been spotty, with only a “minimal number of plans” covering them. “There are some hesitations in regard to effective evaluation strategies of these products,” she said during a session on PDTs at the AMCP 2022 annual meeting. “However, this space has gained a lot of traction, especially during the COVID-19 pandemic.”

Payors consider several factors when deciding to cover the products, Dr. Vishwanath said: Safety. This critical factor involves looking at the digital application’s potential for harms, risks and side effects; security measures incorporated to protect information; and whether the product is cleared by the FDA. (To date, the agency has cleared 10 PDTs.) Clinical evidence. Payors will look at what type of evidence is available, how effective the product is compared with alternative treatments, and whether there is evidence for long-term use.

Applicability. Such determinations include the patient engagement rate; the product’s available platforms; whether data can be integrated into medical records; and whether the product reduces health disparities.

Reasons for Hope As payors continue to grapple with these PDT coverage determinations, several promising developments may speed acceptance of the technology, Ms. Hornung said.

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22

Specialty Pharmacy Continuum • May/June 2022

CLINICAL

Pharms Provide Significant Support in cGVHD

By Myles Starr

Chronic graft-versus-host disease (cGVHD), a difficultto-manage potential complication of allogeneic stem cell transplantation, requires multidisciplinary management, with pharmacists playing a key role on the care team.

Specifically, pharmacists can advise surgeons and oncologists on the most appropriate cGVHD treatments, check for potential drug interactions and help patients with the often emotionally and financially challenging work of obtaining the new and expensive drugs for the condition, according to three experts who shared these strategies with Specialty Pharmacy Continuum.

Given the prevalence of cGVHD, these collaborative efforts are essential. A 2018 survey of 84 articles related to cGVHD found the incidence of the condition ranged from 14% to as high as 90% between one and five years into treatment (Value Health 2018;21[3]:S441-S442).

Generally, cGVHD occurs 100 days or more after a stem cell transplant (Blood 2017;129[1]:30-37), according to Mohammad Maher Abdul Hay, MD, a hematologist at NYU Langone Medical Center, in New York City. The condition “is characterized by fibrosis and inflammation in a variety of potential sites in the body, ranging from the eyes to tendons to the GI [gastrointestinal] tract.” Typical symptoms, Dr. Hay noted, include nausea and vomiting, loss of appetite, eye dryness, lichen planus in the oral cavity and fibrosis in the lungs causing decreased physical exertion tolerance in activities such as walking. “Chronic GVHD, in contrast to acute GVHD, rarely affects the liver and is not accompanied by high bilirubin levels,” he said.

The risk for cGVHD depends on the donor recipient match. Mismatch-related donations usually occur with a haplotype-related transplant. For any treatment team concerned about GVHD, the most important and opportune times to prevent a patient acquiring the condition are when matching the transplant and during perioperative management of the patient’s immunosuppression, Dr. Hay noted. Although physicians—not pharmacists—determine the matching of donor and recipient, pharmacists do have an important role at this juncture, because prevention of cGVHD also relies heavily on choice of medications, he said.

During those early stages, “the most important role of the pharmacist is in assisting in the achievement of [a] therapeutic level of immunosuppressants,” said Doris M. Ponce, MD, a hematologic oncologist at Memorial Sloan Kettering Cancer Center, in New York City. Patients are started on

immunosuppressants before stem cell graft infusion, “and we have learned that early levels of immunosuppression are very critical in prevention of severe GVHD,” she said. “Thus, we aim to achieve and keep a stable therapeutic level throughout the early post-HCT [hematopoietic cell transplant] period.

Our pharmacist are responsible for monitoring and keeping an optimal level of the immunosuppressants. Pharmacists also offer support in how to adjust the dose of the other drugs that are frequently used concurrently ly in transplant.”

belumosudil. The same is true for lung inflammation. That medication in particular has an advantage over the others used to treat cGVHD, but it is only approved as a second- or third-line treatment.” (The prescribing information for belumosudil states that the drug is “indicated for the treatment of adult and pediatric patients 12 years and older with … chronic GVHD after failure of at least two prior lines of systemic therapy.”)

Assistance With Insurance The pharmacists’ role in treating cGVHD is not limited to clinical recommendations and monitoring. As noted, they can be important conduits of information between doctors, patients and

get the drug, and our pharmacy continuously adapts and improves as far as getting access to the medication. So, pharmacists’ involvement in managing cGVHD is really essential from many different perspectives.”

The coverage issues are not just anecdotal. In a 2018 multicenter survey, 34% of patients experienced delayed or denied insurance coverage for cGVHD treatments (Biol Blood Marrow Transplant 2019;25[3]:599-605). One frequent reason for denied claims involves sequence of therapy, Dr. Nix noted. “Many payors have therapy pathways built into their formulary that require a patient try and fail a therapy based on the consensus guidelines,” she said. “Pharmacists can

First-Line Treatment When cGVHD does develop,, first-line medications include corticosteroids. Because some patients are steroid-refractoryy and other patients who aree initially responsive to steroids ds eventually will need to stop them em to avoid complications, a change ange to second-line therapies, including cluding extracorporeal photopheresis, s, ibrutinib (Imbruvica, Janssen) and ruxolitinib (Jakafi, Incyte) sometimes is necessary, Dr. Hay noted.

“In the case of severe GVHD, in my experience, and also looking at the data, at least 30% of patients are going to need to use a second-line therapy, such as ibrutinib or ruxolitinib,” he said. “Of those, another 30% will need to go to [another alternative], such as belumosudil [Rezurock, Kadmon Pharmaceuticals]. Importantly, if there are interactions with other drugs, pharmacists will tell us. For example, with ibrutinib, they’ll note that the patient needs to have a certain number of platelets because there is a risk of bleeding.”

As for ruxolitinib, the drug can activate viruses, “so pharmacists will remind us that we need to follow up for possible cytomegalovirus infections with a patient,” Dr. Hay said.

Belumosudil, a kinase inhibitor approved by the FDA in July 16, 2021, represents “a breakthrough in treating cGVHD, partly because it has something different than anything we have seen in cGVHD—it actually can destroy fibrosis, while the others can only block inflammation; they don’t reverse fibrosis,” he stressed. “For example, some cGVHD patients present with tendon issues, so they cannot flex their arms because of fibrosis. This can be reversible with

Allogeneic transplants contain large amounts of donor immune cells that can trigger a reaction against the recipients’ cells and result in graft-versus-host disease.

Source: Crown Bioscience.

insurance companies. “Patients will view the relationship with their pharmacist differently from the relationship with their oncologist,” said Nancy M. Nix, PharmD, a pharmacy oncology clinical coordinator at Ballad Health, in Johnson City, Tenn. “They will tell you things about their medication that they won’t tell the doctor because they think doctors [focus primarily on] their physical care, and so they choose to tell the pharmacist about their drug care. But the physician really needs to know all this information before he or she makes a choice of treatment, and due to our role, we can relate this information to physicians.”

Dr. Ponce agreed that pharmacists can play a critical role in helping patients and providers navigate payment issues, including appealing denials of initial request for coverage. Especially for uninsured patients, pharmacists also are indispensable in enrolling patients in drug company discount programs, Dr. Ponce noted. “Many insurance companies ask for additional information, which our pharmacies will provide,” she said. “Many of these medicines come from a specialty pharmacy. There are very specific rules about how to

support their patients by providing clear information to the prescriber concerning what therapy must have been tried and failed prior to approval of a subsequent therapy.”

In taking such actions, pharmacists act as an important node between the prescriber, insurance companies and patients, Dr. Nix noted. But it is their clinical role that arguable can have the largest impact. “Prescribers have embraced the role of the pharmacist in being able to aid them in choosing, not just the most effective cGVHD and other treatments, but avoiding potential complications as much as possible,” she said. “Physicians who train at centers that have an integrated pharmacy will absolutely pick up the phone and say, ‘I’m not sure about the drugs they are taking. Can you look at the interactions?’ Doctors definitely view us as a resource to aid in the care of the patients and make it more streamlined overall.”

Drs. Abdul Hay and Nix reported no relevant financial disclosures. Dr. Ponce reported financial relationships with CareDx, Ceramedix, Evive Biotechnology (Shanghai) Ltd., Incyte and Kadmon Corporation.

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