February 2017 by SMCMA

PLUS: 2017 TECHNOLOGY BUZZWORDS • MHEALTH APPS • ARTIFICIAL INTELLIGENCE

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FEBRUARY 2017 - VOLUME 6, ISSUE 2

www.PhysiciansNewsNetwork.com EDITORIAL COMMITTEE

Russ Granich, MD | President Alexander Ding, MD | President-Elect Sara Whitehead, MD | Secretary- Treasurer Michael Norris, MD | Immediate Past President

On The Inside

Janet Chaikind, MD Uli Chettipally, MD Mamatha Chivukula, MD Paul Jemelian, MD Alex Lakowsky, MD Richard Moore, MD Joshua Parker, MD Xiushui (Mike) Ren, MD Brian Tang, MD Dirk Baumann, MD | AMA Alternate Delgate Scott A. Morrow, MD | Health Officer, County of San Mateo

9................Stanford Study : Artificial Intelligence and Healthcare

www.SMCMA.org facebook.com/smcma | twitter.com/SMCMedAssoc.

10..............AMA Guidelines for mHealth Apps

EDITORIAL

11..............Power Collaboration to Improve mHealth Apps

San Mateo County Physician is published ten times per year by Physicians News Network (PNN) and the San Mateo County Medical Association. Opinions expressed by authors are their own and not necessarily those of PNN or SMCMA. San Mateo County Physician reserves the right to edit contributions for clarity and length, as well as to reject any material submitted.

2................President’s Letter | Russ Granich, MD Preparing for the Unknown 4................The Connected Physician : 2017 Buzzwords You Need to Know

12..............Manipulating Orphan Drug Status

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F EB RUA RY 2017 | S A N M AT E O C O U N T Y P H Y S I C I A N 1

PRESIDENT’S MESSAGE

PREPARING FOR THE

UNKNOWN BY RUSS GRANICH, MD

PHOTO BY SCOTT BUSCHMAN

“I will be asking the healthcare leaders of San Mateo County as well as community leaders to become part of a group that will develop a strategy to preserve access to care for those in our community”

This presidential transition is full of unknowns. What is happening is unique to all of us; many old traditions have been discarded for what may become the new norm. One thing that may specifically affect us concerns, of course, healthcare and the Affordable Care Act. The ACA has been fraught with problems but at the same time gave coverage to 20 million people, eliminated pre-existing conditions, eliminated spending caps, created equal premiums for men and women and covered older children to age 26. The incoming administration as well as the Republican majority have declared they will replace “Obamacare.” There is also talk of increasing the age to qualify for Medicare and Social Security as well as eliminating the Children’s Health Insurance Program (CHIP), which provides care to 8.4 million children. This could potentially lead to many of our county residents losing coverage and

becoming stranded. In order to prepare for possible major changes in coverage and increased numbers of San Mateo County residents without coverage, the San Mateo County Medical Association is taking an unprecedented step. We will unite all the healthcare organizations in our county to come together and plan for contingencies. I will be asking the healthcare leaders of San Mateo County as well as community leaders to become part of a group that will develop a strategy to preserve access to care for those in our community, particularly those insured by Covered California and Medi-Cal. I have asked David Goldschmid, MD, an emergency physician and longtime leader and champion of patient rights, to chair this group. The purpose of this group is to be proactive and start planning on how to tackle some of these potential issues. The group will not be a political group. There is no intent to lobby Congress or to try to change the laws, but rather to respond and adapt. The biggest areas that will be impacted are outpatient clinics and emergency departments. This group will come together to plan for a crisis; the format in many ways is similar to disaster planning. Instead of reacting as individual organizations, we can come together to react as a county. When a specific crisis looms, we will gather to prepare and plan. We need to hope for the best but prepare for the worst. And never lose sight of our own mission as physicians to help others to the best of our abilities.

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CONNECTED PHYSICIAN YOU 2017 BUZZWORDS NEED TO KNOW By Sierra Hersek, PNN Staff Writer

I N T H E E V E R - C H A N G I N G W O R L D of connected care it is hard to keep up with new technologies or with the new ways in which familiar technologies are being repurposed. While some of the following terms may not be new to you, they are all worthy of a deeper understanding of how they may impact information sharing, data collection and patient care in 2017.

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The Connected Physician

It is also worth noting that many of these technologies are interconnected. For example, digital therapeutics can also be part of the Internet of Things.

DIGITAL THERAPEUTICS

Used to improve health outcomes by addressing chronic conditions through behavioral changes, digital therapeutics refers to the collective developments in behavioral economics, smartphone apps, gamification, biometric sensors, data analytics and artificial intelligence. In some cases, digital therapies require approval from the U.S. Food and Drug Administration, such as the recently approved medication/digital sensor device to track medical adherence designed by Proteus Digital Health in partnership with Otsuka Pharmaceutical Co. to administer Abilify, which treats schizophrenia, bipolar disorder and depression. Already a-half-billion-dollar market, digital therapeutics is expected to grow into a $6 billion market, according to a research report by Goldman Sachs. Not limited to devices, however, it also encompasses online preventative therapies. Substantiating the digital approach, Medicare recently announced it will cover both bricks-andmortar and virtually delivered diabetes prevention programs like online courses for pre-diabetes prevention.

between providers. Studies have shown that employing these technologies has resulted in dramatic reductions in malpractice claims and faster lab results after introducing EHRs, which offers the potential for significant cost savings and improved quality of care. In addition to informatics, analytics can be provided by converting big data into actionable insights. Practices can expect to see more informatics and analytics solutions hit the market in 2017. It will be important to conduct due diligence before investing in an informatics and/or analytics solution, according to Physicians Practice.

INTEROPERABILITY

Interoperability is the ability of health information systems to work together within and across organizational boundaries in order to advance the effective delivery of healthcare for individuals and communities. Closely tied to informatics, which is the collection of data, interoperability is about sharing that collected data easily between clinician, lab, hospital, pharmacy and patient regardless of the application.

DISEASE MANAGEMENT TECHNOLOGIES

Disease management technologies are connected technologies used to manage chronic illness. They provide physicians information through health information exchanges (HIE) and disease registries that capture and track key patient information to assist care team members in proactively managing patients. They also offer patientcentered applications such as patient portals and personal health records (PHRs). â&#x20AC;&#x153;Many disease management programs have been successful at improving self-care practices and reducing use of various healthcare services, including hospital admissions and emergency room visits. As a result, healthcare expenditures for certain populations with chronic conditions have decreased,â&#x20AC;? according to a study by Georgetown University Health Policy Institute.

INFORMATICS

Informatics is the science behind information technology, communications and healthcare to improve the quality and safety of patient care. This includes the use of electronic health records (EHRs), technology-based patient education, and the exchange of health records

Many disease management programs have been successful at improving self-care practices and reducing use of various healthcare services, including hospital admissions and emergency room visits. As a result, healthcare expenditures for certain populations with chronic conditions have decreased. -Study by Georgetown University Health Policy Institute

F EB RUA RY 2017 | S A N M AT E O C O U N T Y P H Y S I C I A N 5

The Connected Physician

“An interoperable health IT ecosystem makes the right data available to the right people at the right time across products and organizations in a way that can be relied upon and meaningfully used by recipients,” according to a report by the Office of the National Coordinator for Health Information Technology. “By 2024, individuals, care providers, communities and researchers should have an array of interoperable health IT products and services that allow the healthcare system to continuously learn and advance the goal of improved healthcare.”

THE CLOUD

“The cloud” is a general term for the delivery of hosted services over the Internet. While not a new term, the cloud in 2017 is especially important in healthcare due to the large shift to digital storage and data sharing. Cloud technology has accelerated the way the healthcare industry can use or share information across a network and is relevant to interoperability and informatics. According to a study by EMC, healthcare represents a significant percentage of the overall digital universe and is growing at a clip of 48% each year. Several variables are fueling this expansion, including the use of electronic medical records and development of advanced diagnostics including molecular and genomic testing and digital imaging. Policy initiatives such as the recently passed 21st Century Cures Act also promote the use of patient data to advance the discovery and delivery of preventative cures and treatments. According to Cloud Tech, the cloud platform is secure, maintaining the confidentiality of patient information; it is scalable, allowing economic growth as the data set increases and performance needs change; and it is collaborative, enabling researchers to work with external partners. A recent survey of healthcare industry IT and leadership professionals shows that 73% of industry professionals will use the cloud to host patient empowerment tools — another key driver toward the cloud. This means healthcare providers are increasingly using cloud-based applications to put into the hands of patients resources that will allow them to educate themselves, monitor their own health, and store and share their health record. 6 S A N M AT E O C O U N T Y P H Y S I C I A N | F EB RUA RY 2017

BLOCKCHAIN

Health IT is becoming an increasingly important factor for a physician’s daily practice, and with it are growing concerns about the security of electronic health records (EHRs) and the information they hold. “Blockchain,” one of the latest health IT buzzwords, is being touted by some as the answer to the constant threat of breached health data, but what exactly is it? A blockchain is a permanent record of online transactions. Using cryptography, each transaction is recorded and is considered a block. The ledger that links these transactions can be thought of as the chain. This record can be shared among a network of computers, and users on the network can add to the record of transactions. Instead of a database that is centrally located and maintains and manages records, the database is distributed to the networks. Transactions are kept secure via cryptography, and transactions have to be approved and verified by the network in a process called mining, according to Becker’s Hospital Review. Although originally created for the financial industry, blockchain has promise in healthcare due to its interoperability for all users of a network to access that network, and all pieces of information are verified and show the history of transactions. “Blockchain allows for interoperability at a new level for healthcare,” writes Bruce Broussard, CEO of Humana, in an article for LinkedIn. “Electronic Health Records (EHRs) connect right into the Blockchain, where information is not just integrated into the EHR from the hospital, but from providers and specialists. A primary care physician could

The Connected Physician

ARTIFICIAL INTELLIGENCE (AI)

access a complete medical history of the member, while the radiologist could be limited to only the specifics he or she needs to perform the task at hand. For each, it’s about accessing the right data at the right time, and the Blockchain technology could enable this type of specific ‘need-to-know’ medical history access.” Broussard notes blockchain could also transform the entire healthcare claims process. “There is no more backand-forth haggling with the health plan about what was paid, why it was paid or whether it should have been paid. With transparency and automation, greater efficiencies will lead to lower administration costs, faster claims and less money wasted,” he writes. Population health may also be a place to apply blockchain technology. Organizations can access patient databases on a large, population scale and eliminate the middleman, therefore better protecting the information. “Spending time and resources verifying members’ trustworthiness (e.g., HIE, all-payer claims database, local EMRs) no longer makes savvy business sense. Blockchain will leapfrog population health by providing trust where none exists for continuous access to patient records by directly linking information to clinical and financial outcomes,” reports CIO. “The integration of Blockchain into the health care system still has a long way to go before its potential can be fully realized. The good news is that the healthcare industry has already started to embrace the transformational power of technology,” concludes Broussard.

While applicable in a variety of settings, AI in healthcare promises a variety of life-enhancing innovations, including clinical decision support, patient monitoring and coaching, automated devices to assist in surgery or patient care, and management of healthcare systems. In essence AI may be more involved in everyday tasks; for example, Forbes predicts that AI will play a big role in diagnostic imaging by complementing radiologists with advanced interpretation and imaging informatics support. “For AI technologies, healthcare has long been viewed as a promising domain. AI-based applications could improve health outcomes and quality of life for millions of people in the coming years — but only if they gain the trust of doctors, nurses, patients, and if policy, regulatory and commercial obstacles are removed,” said a panel of experts at Stanford working on the One Hundred Year Study on Artificial Intelligence (AI100).

AUGMENTED REALITY (AR)

Virtual additions to the known physical world, often used in fitness and wellness apps, augmented reality is also being utilized for much more complex applications like surgical guidance. AR can also be used in conjunction with AI, allowing physicians to employ machine learning while strategizing an upcoming surgical procedure in a simulation. A surgical simulation can be built from actual medical images of the patient undergoing surgery. Surgeons can then virtually perform the procedure with life-like tissues that can react to digital surgery tools and thus plan ahead to avoid issues that were previously unforeseeable. Augmented reality is also used in educating medical students, offering them the most realistic surgical simulation. “It’s [AR] already impacting the way medical professionals are training and physicians diagnose and treat patients,” said Rodwin Pabello, director of product development at San Francisco-based software developer Viscira, in an interview with Healthcare IT News. “There are many different uses for the technology within healthcare, from application in operating rooms to educational fun and games, and that’s just scratching the surface.” F EB RUA RY 2017 | S A N M AT E O C O U N T Y P H Y S I C I A N 7

The Connected Physician

IBM Watson, one of the most well-known cognitive computing applications, is now working to improve outcomes for cancer patients. IBM Watson for Oncology was specifically designed to help oncologists involved in cancer treatment.

COGNITIVE COMPUTING

Cognitive computing is the ability to simulate the human thought process in a computerized model. Cognitive computing systems depend on various aspects of artificial intelligence (AI) such as machine learning, reasoning, natural language processing, speech and vision, humancomputer interaction, dialogue and narrative generation, and more. The machine learning algorithms learn and acquire knowledge from the massive amount of data fed into them, according to Forbes. IBM Watson, one of the most well-known cognitive computing applications, is now working to improve outcomes for cancer patients. IBM Watson for Oncology was specifically designed to help oncologists involved in cancer treatment. It can analyze the meaning and context of structured and unstructured data coming from a variety of inputs, including handwritten documents. In addition, Watson can sift through information from over 290 medical journals, over 200 textbooks, and 12 million pages of text.

INTERNET OF THINGS (IOT)

The IoT is the internetworking of physical devices (also referred to as “connected devices” and “smart devices”) 8 S A N M AT E O C O U N T Y P H Y S I C I A N | F EB RUA RY 2017

embedded with electronics, software, sensors, actuators, and network connectivity that enable these objects to collect and exchange data. In healthcare this might include everything from ultrasounds, heart monitors, thermometers, glucose monitors, electrocardiograms and cell phones when used to connect with healthcare providers or mHealth apps. According to Business Insider Intelligence, the installed base of healthcare IoT devices will grow to more than four devices for every human on Earth by the year 2020. As the IoT market matures, it is expected that healthcare will be a major beneficiary of the technology, and vendors are exploring capabilities and features that can benefit patients and healthcare professionals. Voice-based virtual assistants such as Apple’s Siri, Google’s Assistant and Microsoft’s Cortana have successfully made voice-enabled devices popular with smartphone users, including healthcare professionals and patients, and are now migrating into the home, office and healthcare settings. A rising star in this IoT category is the Amazon Echo, a family of smart speakers whose sales more than doubled from 2015 to 2016. Alexa, the voice-based virtual assistant that powers Echo devices, could be a useful tool for healthcare providers and patients, according to Amazon CEO Jeff Bezos. Alexa’s base technology can be used for healthcare at home, including voice-activated home health capabilities such as medication adherence, pain management, patient education, patient monitoring and caregiver coordination. Answering physicians’ questions at the Vanity Fair New Establishment Summit in San Francisco recently, Bezos said that he could see uses for Alexa at the hospital and in the home for help with medical compliance and other treatment tasks.

ALEXA IS ALREADY GETTING SOME USE IN THE HEALTHCARE SETTING: • Mount Sinai for finding physicians linked to the Mount Sinai Health Partners program • Boston Children’s Hospital Kids MD for children’s fever and medication advice • Zika Facts and HealthBuddy for diseasespecific information • Marvee care companion for aging family members • Health Care Genius for healthcare terminology like “What’s a deductible?”

The Connected Physician

Stanford Study

ARTIFICIAL INTELLIGENCE and HEALTHCARE By Sierra Hersek, PNN Staff Writer

Stanford University is leading the charge and inviting contributors from several institutions to begin a new project, “One Hundred Year Study on Artificial Intelligence (AI100).” In the first report, “Artificial Intelligence and Life in 2030,” researchers pondered the useful application of artificial intelligence (AI) and found healthcare to be a likely fit. “For AI technologies, healthcare has long been viewed as a promising domain. AI-based applications could improve health outcomes and quality of life for millions of people in the coming years — but only if they gain the trust of doctors, nurses, patients, and if policy, regulatory, and commercial obstacles are removed,” the panel of experts reported. AI in healthcare promises a variety of life-enhancing innovations, including clinical decision support, patient monitoring and coaching, automated devices to assist in surgery or patient care, and management of healthcare systems. Data collection is a major part of these advancements. From personal monitoring devices and mobile apps to electronic health records (EHRs) in clinical settings, there is a wealth of information to be collected and applied. But restrictions around how this information can be used have slowed progress, according to the study. “Using this data to enable more finely grained diagnostics and treatments for both individual patients and patient populations has proved difficult,” the panelists wrote. “Research and deployment have been slowed by outdated regulations and incentive structures. Poor human-computer interaction methods and the inherent difficulties and risks of implementing technologies in such a large and complex system have slowed realization of AI’s promise in healthcare. The reduction or removal of these obstacles, combined with innovations still on the horizon, have the potential to significantly improve health outcomes and quality of life for millions of people in the coming years.” In a clinical setting the report points to the development of EHRs as a bumpy progression. The panel says that a limited number of vendors control the EHR market, and user interfaces are widely considered substandard. The promise of new analytics using data from EHRs, including AI, remains largely unrealized due to these and other regulatory and structural barriers. In the next 15 years, AI advances, if coupled with sufficient data and well-targeted systems, promise to change the cognitive tasks assigned to human clinicians, according to HealthcareNewsIT. “The opportunity to exploit new learning methods, to create structured patterns of inference by mining the scientific literature automatically, and to create true cognitive assistants by supporting free-form dialogue has never been greater,” the report stated. The report concludes that significant AI-related advances have already had an impact on North American cities over the past 15 years, and even more substantial developments are expected in the next 15, provided they are judiciously managed. “Recent advances are largely due to the growth and analysis of large data sets enabled by the Internet, advances in sensory technologies, and more recently, applications of ‘deep learning,’” the report noted. “In the coming years, as the public encounters new AI applications in domains such as transportation and healthcare, they must be introduced in ways that build trust and understanding, and respect human and civil rights.”

AI in healthcare promises a variety of life-enhancing innovations, including clinical decision support, patient monitoring and coaching, automated devices to assist in surgery or patient care, and management of healthcare systems.

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The Connected Physician

AMA RELEASES GUIDELINES FOR USE OF

mHEALTH APPS and RELATED DEVICES By Sierra Hersek, PNN Staff Writer

I N A N E F F O R T T O P R OV I D E G U I DA N C E and clarity in the new era of mHealth and associated digital health devices, trackers and sensors, the American Medical Association (AMA) has announced eight guiding principles to help physicians navigate coverage, payment, and financial incentive mechanisms for mHealth apps. “The new AMA principles aim to foster the integration of digital health innovations into clinical practice by promoting coverage and payment policies that are contingent upon whether mHealth apps and related devices are evidence-based, validated, interoperable and actionable,” said AMA Immediate Past President Steven J. Stack, MD, in a statement. “It is essential for mHealth apps to support care delivery that is patient-centered, promotes care coordination and facilitates team-based communication.” The following eight principles will be used to support the use of mHealth apps and associated devices, trackers and sensors:

The AMA also noted that many physicians are unsure of their own liability when using, recommending, or prescribing mHealth apps. Accordingly, the AMA plans to “assess the potential liability risks to physicians for using, recommending, or prescribing mHealth apps, including risk under federal and state medical liability, privacy, and security laws.” While physicians are optimistic about digital health and its potential medical benefits, as reported on PNN, the AMA policy acknowledges the need to expand the evidence base necessary to show the accuracy, effectiveness, safety, and security of mHealth apps.

1. Support a strong patient-physician relationship. 2. Possess a clinical evidence base to ensure mHealth app safety and effectiveness. 3. Follow evidence-based practice guidelines to boost patient safety, quality of care and positive health outcomes. 4. Promote care delivery that is patient-centered, coordinated, and based on team communication. 5. Support data portability and interoperability to foster care coordination through medical home and accountable care models. 6. Follow state licensure laws and state medical practice laws and requirements in line with where the patient is using the app. 7. Mandate providers delivering services through the app hold licensure in the state where the patient receives services. 8. Ensure that the delivery of any services via the app be consistent with the state’s scope of practice laws.

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It is essential for mHealth apps to support care delivery that is patient-centered, promotes care coordination and facilitates team-based communication.

The Connected Physician

Power Collaboration By Sierra Hersek, PNN Staff Writer

AIMS TO IMPROVE mHEALTH

F O U R O R G A N I Z AT I O N S A R E F O R M I N G a new multistakeholder collaboration, Xcertia, dedicated to improving the

AHA

AMA

DHX

HIMSS

quality, safety and effectiveness of mobile health applications (apps). The initial supporters of the collaboration include the American Heart Association (AHA), American Medical Association (AMA), DHX Group, and the Healthcare Information and Management Systems Society (HIMSS). The collaboration, along with a diverse membership, will leverage the insights of clinicians, patients and industry experts to help improve patient care and increase access to data. “The collaboration builds on each organization’s ongoing efforts to foster safe, effective and reputable health technologies, while complementing our mutual commitment to advancing innovation in medicine, and improving the health of the nation,” the four organizations said in a joint statement. “Our combined expertise, along with a diverse membership, will leverage the insights of clinicians, patients and industry experts to help improve patient care and increase access to data.” This announcement came a month after the AMA released a new set of guidelines designed to support physicians using mHealth devices and apps while calling for better regulation of apps. Xcertia joins a growing number of organizations looking to shed light on the multitude of mHealth apps for both patients and providers, such as RxUniverse out of the Sinai App Lab at the Icahn School of Medicine at Mount Sinai Health System, as reported by PNN. Also, the Hacking Medicine Institute’s RANKED Health program, a nonprofit launched out of Harvard and MIT, evaluates apps based on both health outcomes and consumer use. But Xcertia is addressing the issue a little differently, its website states. Xcertia will not engage in certifying mHealth apps; it will encourage others to apply its principles and guidelines in the development and curation of safe and effective mobile health apps. Xcertia’s membership and governing board may look different from others as it will include broad representation from consumers, developers, payers, clinicians, academia and others with an interest in the development of guidelines for mobile health apps.

The collaboration builds on each organization’s ongoing efforts to foster safe, effective and reputable health technologies while complementing our mutual commitment to advancing innovation in medicine and improving the health of the nation.

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The Connected Physician

“MANIPULATING” ORPHAN DRUG STATUS TO HIKE PRICES By Sierra Hersek, PNN Staff Writer

T H E O R P H A N D R U G S A C T was passed over 30 years ago as a motivator for pharmaceutical companies to develop new drugs for rare diseases. It is an area often ignored by drug companies due to less people in need leading to a smaller profit margin.

Today, some pharmaceutical companies are using the term “orphan” to justify a drug’s high price, whether or not that drug is designated as such.”

When a drug receives “orphan” status, the manufacturer receives millions of dollars in government incentives plus seven years of exclusive rights to treat that rare disease. But many of these drugmakers, according to a Kaiser Health News (KHN) investigation, are manipulating a system intended to help desperate patients in need. The companies aren’t breaking the law, but they are using the Orphan Drug Act to their advantage in ways that its architects say they didn’t foresee or intend. Today, many orphan medicines, originally developed to treat diseases affecting fewer than 200,000 people, come with astronomical price tags, according to KHN. In addition, more than 70 drugs that now have orphan status were first approved by the Food and Drug Administration for mass market use and later determined to be effective for patients with a rare condition. The result is a niche market now monopolized by a medicine already being taken by millions and making huge profits in that market. “What was intended for a good purpose can be used for a purpose that’s harmful to patients who can’t afford drugs,” said former U.S. Rep. Henry Waxman, D-Calif., a primary sponsor of the 1983 Orphan Drug Act. “And it makes the whole cost of all of these pharmaceuticals much more expensive for everybody.” Annual sales from orphan drugs are expected to grow 12% a year through 2020 — a pace that general drugmakers could “only dream about,” market watcher EvaluatePharma said in its most recent orphan drug report. In 2014, the average annual price tag for orphan drugs was $111,820 versus $23,331 for mainstream drugs. Orphan drugs are isolated from many of the typical pressures that bring down prices. Insurance companies often have so few rare-disease patients on their books that even a very high-priced drug gets little attention. And pharmaceutical companies fund assistance programs that ensure patients do not forgo a treatment because of its price, according to the Washington Post. Today, some pharmaceutical companies are using the term “orphan” to justify a drug’s

1 2 S A N M AT E O C O U N T Y P H Y S I C I A N | F EB RUA RY 2017

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PHYSICIANSNEWSNETWORK.COM high price, whether or not that drug is designated as such. The market has come to expect higher prices for any drug that treats less patients, and the system lacks any real mechanism to counter the price increases. Valeant Pharmaceuticals International recently outlined its reasoning for a price hike for Syprine, a three-decade-old rare-disease drug that ultimately went from $652 for 100 capsules to $21,267 over a fiveyear period. The reason for the price hike: “Progressive pricing actions to bring it in line with comparable orphan products.” In the commentary “The Orphan Drug Act: Restoring the Mission to Rare Diseases,” published in the American Journal of Clinical Oncology, the authors wrote: “We identify a pattern of pharmaceutical companies submitting drugs to the Food and Drug Administration (FDA) as orphan drugs, but once approved, the drugs are used broadly off-label with the lucrative orphan drug protections and exclusivity benefits. Since the law was passed, the proportion of new FDA-approved drugs that were submitted as orphan drugs has increased, with a peak last year of 41% of all FDA-approved drugs approved as orphan drugs. On the basis of the current data, we suggest that patients with rare cancers and other diseases may suffer due to dilution of the incentives and benefits. We propose reform to increase submission scrutiny, decrease benefits based on offlabel use, and increase price transparency.” Drug companies argue that changes to incentives could lead to less energy given to rare disease drug treatment once again. This only spurs fear in patients and their advocates, but many experts believe the new market for orphan drugs gives companies virtually unlimited pricing power.

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