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Advocacy Grand Rounds: Accelerated Approval Reforms: Balancing Timely Access and True Clinical Benefit
April 21 @ 1:00 pm–2:00 pm EDT
Since 1992, the U.S. Food and Drug Administration (FDA) has implemented the expedited approval pathway of accelerated approval. Under accelerated approval, manufacturers can receive faster approval of a drug based on a surrogate marker thought to be predictive of clinical benefit. In return for earlier market entry of drugs, manufacturers are required to complete postapproval studies to confirm that the drug does indeed have clinical benefit. However, the FDA has not made sure that the pharmaceutical industry has not upheld its end of the bargain. Since accelerated approval was introduced in 1992, almost half of the drugs approved through this pathway have not yet been confirmed to be clinically beneficial for patients as of 2020. Of these, one-fifth have been on the market, available for patients to use for more than 5 years. Even when these mandated confirmatory trials are completed and have failed to confirm the drug’s clinical benefit, the FDA has often not withdrawn these approvals. Both this prolonged uncertainty of these drugs’ clinical benefit as well as the FDA allowing the drugs to be prescribed even after they’ve failed confirmatory trials harms patients, both medically and financially. In light of the recent controversial, accelerated approval of aducanumab, Congress has introduced proposals to reform this expedited review pathway to balance timely access to new drugs and ensure patients truly beneficial treatments.
Joseph S. Ross, MD, MHS
Joseph S. Ross, MD, MHS, is a Professor of Medicine (General Medicine) and of Public Health (Health Policy and Management) at the Yale School of Medicine, an Associate Physician of the Center for Outcomes Research and Evaluation at Yale-New Haven Health System, and Co-Director of the National Clinician Scholars Program at Yale. With expertise in health services and outcomes research and the translation of clinical research into practice, his research examines the use and delivery of higher quality care and issues related to pharmaceutical and medical device regulation, evidence development, postmarket surveillance, and clinical adoption. Dr. Ross co-directs the Yale-Mayo Clinic Center for Excellence in Regulatory Science and Innovation (CERSI), the Yale Open Data Access (YODA) Project, and the Collaboration for Research Integrity and Transparency (CRIT) at Yale Law School, and leads efforts at Yale-New Haven Health System in collaboration with the National Evaluation System for health Technology (NEST). He has published more than 500 articles in peer-reviewed biomedical journals, co-founded the preprint server medRxiv, and is currently the U.S. Outreach and Research Editor at BMJ.
Holly Fernandez Lynch, JD, MBE
Holly Fernandez Lynch, JD, MBE, is Assistant Professor of Medical Ethics and Law at the University of Pennsylvania. Her scholarship focuses on clinical research ethics and regulation, priority setting in research, access to investigational medicines outside clinical trials, FDA pharmaceutical policy, and the ethics of gatekeeping in health care. She is founder and co-chair of the Consortium to Advance Effective Research Ethics Oversight (www.AEREO.org), an organization working to evaluate and improve IRB quality and effectiveness, and an active member of the NYU Working Group on Compassionate Use and Preapproval Access (CUPA). She serves as a member of the boards of Public Responsibility in Medicine & Research (PRIM&R) and the American Society for Law, Medicine, and Ethics, and as “ethicist in residence” at the Robert Wood Johnson Foundation. Professor Fernandez Lynch has worked as an attorney in private practice, a bioethicist serving NIH’s Division of AIDS, a senior policy analyst with President Obama’s Commission for the Study of Bioethical Issues, and executive director of Harvard Law School’s bioethics and health law research program. She was named a Greenwall Faculty Scholar in 2019 and elected a fellow of the Hastings Center in 2021.