Breakthrough therapy designation: Exploring the qualifying criteria

Established by the Food and Drug Administration Safety and Innovation Act of 2012, breakthrough therapy designation (BTD) is one of several programs developed by the U.S. Food and Drug Administration (FDA) to speed up the development and review of drugs and biologics that address unmet medical needs. In order to qualify for this designation, the treatment must address a serious or life-threatening illness. In addition, the manufacturer (i.e., sponsor) must provide early clinical evidence that the treatment is a substantial improvement over currently available therapies. The FDA is working to further clarify how it applies the qualifying criteria to breakthrough designation applications.

On April 24, under a cooperative agreement with FDA, the Center for Health Policy convened a public meeting to discuss the qualifying criteria for this special designation. Using examples from oncology, neurology, psychiatry, and hematology, the workshop highlighted considerations for the BTD application process, the evaluation process, and factors for acceptance or rejection. The discussion also focused on key strategies for ensuring that the qualifying criteria are understood across a broad range of stakeholder groups.

Past Event

Breakthrough therapy designation: Two and a half years in

Breakthrough therapy designation: Two and a half years in

Applying the breakthrough therapy criteria: Oncology

Applying the breakthrough therapy criteria: Neurology

Applying the breakthrough therapy criteria: Anti-infective/psychiatry/hematology

Summary and discussion of lessons learned

Agenda

Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug Administration

Economist - U.S. Food and Drug Administration

Operations Research Analyst - U.S. Food and Drug Administration

Oak Ridge Institute for Science and Education Fellow - U.S. Food and Drug Administration

Operations Research Analyst - U.S. Food and Drug Administration

Medical Officer, Division of Oncology Products II - U.S. Food and Drug Administration

Professor of Medicine; Harvard Medical School - Director, Ludwig Center at Harvard; Senior VP for Experimental Therapeutics, Dana-Farber Cancer Institute

Medical Officer, Division of Oncology Products II - U.S. Food and Drug Administration

Medical Director, Clinical Research and Development - Ariad Pharmaceuticals

Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug Administration

Deputy Assistant Secretary, European and Eurasian Affairs - U.S. State Department

Chairperson and Founder - Friends of Cancer Research

Clinical Team Lead, Division of Oncology Products II - U.S. Food and Drug Administration

Chief Science Officer, Medical & Scientific Relations - Alzheimer's Association

Clinical Team Leader, Division of Neurology Products - U.S. Food and Drug Administration

Senior Vice President, Global Head of Drug Regulatory Affairs and U.S. Head of Development - Novartis

Director, Office of Translational Research - National Institute for Neurological Disorders and Stroke

Deputy Director for Clinical Science, Center for Drug Evaluation and Research - U.S. Food and Drug Administration

Clinical Team Lead, Division of Anti-Infective Products - U.S. Food and Drug Administration

Vice President, Regulatory Science - Bluebird Bio

Adjunct Assistant Professor of Medicine, Hematology/Oncology; University of Pennsylvania - Co-Founder and Executive Director; Castleman Disease Collaborative Network

Senior Director, U.S. Regulatory Policy and Advocacy - GlaxoSmithKline plc

Chief, General Medicine Branch, Office of Cellular, Tissue and Gene Therapies - U.S. Food and Drug Administration

Director, Office of New Drugs - U.S. Food and Drug Administration

Clinical Team Lead, Division of Psychiatric Products - U.S. Food and Drug Administration

Associate Clinical Professor of Medicine; George Washington University School of Medicine - Senior Medical Scientist; Leidos Biomedical Research

Professor, Department of Psychiatry; Harbor-UCLA Medical Center - Chief Science Officer; SyneuRx International (Taiwan) Corporation

Executive Director - Friends of Cancer Research

Head of Global Regulatory Affairs - Janssen Pharmaceuticals

Director, Office of Surveillance and Epidemiology - U.S. Food and Drug Administration

Senior Vice President, Science Policy - Biotechnology Industry Organization

Deputy Director, Center for Biologics Evaluation and Research - U.S. Food and Drug Administration

Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug Administration

Principal, Drug and Biological Drug Products - Greenleaf Health LLC

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