June 2024 | Patent thickets, FTC's pharma actions, trust in FDA, and more
Program on Regulation, Therapeutics, and Law (PORTAL)
Part of the Division of Pharmacoepidemiology at Harvard Medical School and Brigham & Women's Hospital
"We should strive for a pharmaceutical system that rewards meaningful therapeutic breakthroughs…not small modifications to the plastic components of delivery devices or new tweaks to molecules that perform no better than existing therapies."
On May 21, PORTAL faculty William Feldman, MD, DPhil, MPH testified before the Senate Judiciary Committee during a hearing on strategies to improve competition in the prescription drug market. Watch his opening statement and read his written testimony.
Recent PORTAL Publications
Original Analysis
Daval CJR, Egilman AC, Sarpatwari A, Kesselheim AS | JAMA
This study characterized the Federal Trade Commission’s (FTC) oversight of the pharmaceutical industry. From 2000-2022, the FTC pursued 22 enforcement actions against firms for alleged anticompetitive practices and challenged 62 mergers, of which 61 were allowed to continue, 58 after divesting drugs to third-party competitors. The authors propose that Congressional action may be necessary to strengthen the FTC’s authority and enforcement capacity.
Horrow C, Gabriele SME, Tu SS, Sarpatwari A, Kesselheim AS | JAMA Internal Medicine
In this study, the authors reviewed the US patents and patent applications protecting the 10 highest-revenue brand-name drugs in 2021, finding that almost three-quarters were filed after FDA approval. These post-approval patents and those protecting parts of the drug other than the active ingredient contributed to dense patent thickets, delaying generic and biosimilar competition. These findings suggest that closer scrutiny of these post-approval patent applications and patents could prevent patent thickets that delay generic or biosimilar competition.
Feldman WB, Rand LZG, Carpenter D, Russo M, Bhaskar A, Lu Z, Campbell EG, Darrow J, Kesselheim AS | Clinical Pharmacology & Therapeutics
The authors surveyed more than 2,000 US respondents to better understand trust in the FDA, finding that FDA mistrust was associated with various participant characteristics, including female sex, rural communities, conservative political views, worse self-reported health, lower health care satisfaction, paying less attention to health and science news, and not having children under 18. These findings underscore the challenges in convincing a heterogeneous American public to trust the FDA.
Rand LZG, McGraw S, Wang J, Woloshin S, Wang SV, Darrow J, Kesselheim AS | Journal of the American Geriatrics Society
When new drugs are approved, there is often limited evidence from older patient populations, and data from routine clinical care could provide patient population-relevant information. In this study, the authors conducted focus groups with patients 65 years and older who take an anticoagulant to understand their perspective on the evidence of safety and effectiveness from clinical trials or routine clinical use data for similar patients.
Horrow C, Kesselheim AS | The Milbank Quarterly
This study discusses the regulatory framework for laboratory-developed tests (LDTs) and efforts to ensure their safety and validity. The authors highlight the need for a balanced framework that protects patients without stifling laboratory innovation and discuss the impact and rationale behind proposals like the Verifying Accurate Leading-edge IVCT Development (VALID) Act and the recent FDA final rule.
Druedahl LC, Price WN II, Minssen T, Sarpatwari A | JAMA Network Open
This cross-sectional study evaluated how drug developers have used artificial intelligence in pharmaceutical research and development. Out of 102,454 investigational and approved drugs in the Pharmaprojects database, developers reported using AI to aid in the development of 164 investigational drugs and 1 approved drug. The authors conclude that the use of AI in drug development has been modest and mostly limited to early-stage applications such as drug molecule discovery.
Mahesri M, Sarpatwari A, Huybrechts KF, Lii J, Lee SB, Toyserkani GA, LaCivita C, Zhou EH, Dal Pan GJ, Kesselheim AS, Bykov K | Drug Safety
The authors used claims data to evaluate patients’ use of and adherence to three teratogenic drugs subject to FDA-imposed risk evaluation and mitigation strategies (REMS). Patients who could become pregnant and initiated lenalidomide, thalidomide, and pomalidomide compromised 3% of all patients initiating these drugs. The authors did not observe evidence of adherence to pregnancy testing requirements in most patients of childbearing potential, pointing to the need for further research on how often patients do not adhere to pregnancy testing requirements when taking teratogenic drugs.
Viewpoint & Commentary
Hwang CS, Kesselheim AS, Rome BN | Journal of Law, Medicine & Ethics
Glucagon-like peptide-1 (GLP-1) receptor agonists are effective for treating obesity, but the high cost of these medications endangers the financial viability of our health care system. In this perspective, the authors argue that to ensure that these drugs are available to Medicare beneficiaries, pharmaceutical manufacturers must lower their prices.
领英推荐
Tibau A, Kesselheim AS | The Lancet Oncology
Two recent studies assess the impact of FDA’s Project Orbis on cancer drug approval times and the long-term survival benefits of cancer drugs approved based on immature survival data. In this associated commentary, the authors highlight that though using surrogate measures may reduce costs and expedite patient access to new treatments, these measures might not accurately predict overall survival benefits. Thus, rigorous post-approval testing and transparent data reporting are critical to ensure patients receive the most effective treatments.
Willke RJ, Pizzi LT, Rand LZ, Neumann P | Value in Health
Recent concerns about the quality-adjusted life year (QALY) being discriminatory against selected patient groups have resulted in US legislation and regulation that would ban the use of the QALY in government programs. The authors identify scientific support for the QALY and its usefulness as an input to healthcare decision-making, acknowledge its limitations, and argue that, when appropriately constructed and used, it will not be discriminatory against these patient groups.
Kumar S, Blake VK, Tu SS | Health Affairs Forefront
Recent court cases, including Dobbs v. Jackson Women’s Health Org. and Alliance for Hippocratic Medicine v. FDA, highlight a concerning trend of US courts ignoring or misusing medical evidence in ruling on health care regulation. In this review of the US Supreme Court’s abortion jurisprudence, the authors find a correlation between the citation of scientific evidence and the expansion of abortion rights, while the absence of such citations often resulted in abortion restrictions. They suggest that courts should always consider scientific evidence when making decisions about reproductive rights and underscore the need for greater action to ensure informed judicial rulings.
Cai C, Song Z | Health Affairs
Private equity investment in the US health care system has rapidly increased in the past 10 years, with concerning effects on clinical care and costs. In this commentary, the authors review a variety of evidence-based policies at the state and federal level intended to address these effects.
PORTAL in Action
Sarah Gabriele , Joseph Daval, and Dongzhe Hong attended the ISPOR—The Professional Society for Health Economics and Outcomes Research 2024 conference, with presentations on the cost of drug market exclusivity extensions, brand-over-generic preferred drug lists in Medicaid, and ways to improve FDA advisory committees.
William Feldman, MD, DPhil, MPH presented research on the impact of patents on the high cost of asthma and COPD inhalers at the Massachusetts General Hospital Pulmonary & Critical Care Medicine Grand Rounds.
Aaron S. Kesselheim, M.D., J.D., M.P.H. participated in several events last month, including a National Conference of State Legislatures panel on prescription drug importation, a discussion on drug-device combinations during the FDA Generic Drug Science and Research Initiatives workshop, and a presentation on product liability litigation at a UCSF-JHU Opioid Industry Documents Archive symposium. He also spoke at the Harvard Medical School Global Health Care Leaders program.
Dongzhe Hong shared research on the use of real-world evidence to address prescription drug affordability and access at a seminar hosted by the UF Online Pharmaceutical Outcomes & Policy Program .
Christopher Cai presented research on the potential savings of a $2 generic drug copay cap for Medicare patients at the Society of General Internal Medicine (SGIM) 2024 Annual Meeting. He was awarded the prestigious Mack Lipkin, Sr. Associate Member Scientific Presentation Award for this presentation.
Catherine Hwang also presented at SGIM, highlighting research into physicians’ awareness of changes to REMS-related lab testing practices during the COVID-19 pandemic.
Benjamin Rome presented two posters at SGIM, one on the impact of Medicare inflation rebates on drug price increases and another on the trends in the use and cost of biologics for the treatment of plaque psoriasis.
S. Sean Tu joined PORTAL collaborators and alumni, including Anjali D. and Rebecca E. Wolitz , for the Stanford Law School BioLawlapalooza, where he presented on ancillary product patents.
Edward Cliff visited the FDA for Project ODAC Odyssey, a mock oncologic drugs advisory committee focused on molecularly defined subgroups in oncology clinical trials.
About PORTAL
The Program On Regulation, Therapeutics, And Law (PORTAL) is one of the largest research centers in the U.S. studying drug prices, drug policy, and the clinical, economic, and legal aspects of medication use and costs. PORTAL is housed within the Division of Pharmacoepidemiology and Pharmacoeconomics in the Department of Medicine at Brigham and Women’s Hospital (BWH) and Harvard Medical School. To assure independence,?no members of PORTAL have any personal financial relationships with pharmaceutical or medical device manufacturers.
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