Breakthrough therapy designation: Exploring the qualifying criteria
Past Event
Breakthrough therapy designation: Two and a half years in
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.
Agenda
Welcome, introduction and meeting objectives
FDA opening remarks
Richard Moscicki
Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug Administration
Breakthrough therapy designation: Two and a half years in
Ryan Conrad
Economist - U.S. Food and Drug Administration
Afi Harrington
Operations Research Analyst - U.S. Food and Drug Administration
Grace Stark
Oak Ridge Institute for Science and Education Fellow - U.S. Food and Drug Administration
Kim Taylor
Operations Research Analyst - U.S. Food and Drug Administration
Applying the breakthrough therapy criteria: Oncology
Jennie Chang
Medical Officer, Division of Oncology Products II - U.S. Food and Drug Administration
George Demetri
Professor of Medicine; Harvard Medical School - Director, Ludwig Center at Harvard; Senior VP for Experimental Therapeutics, Dana-Farber Cancer Institute
Dickran Kazandjian
Medical Officer, Division of Oncology Products II - U.S. Food and Drug Administration
David Kerstein
Medical Director, Clinical Research and Development - Ariad Pharmaceuticals
Richard Moscicki
Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug Administration
Eric Rubin
Deputy Assistant Secretary, European and Eurasian Affairs - U.S. State Department
Ellen Sigal
Chairperson and Founder - Friends of Cancer Research
Marc Theoret
Clinical Team Lead, Division of Oncology Products II - U.S. Food and Drug Administration
Applying the breakthrough therapy criteria: Neurology
Maria Carrillo
Chief Science Officer, Medical & Scientific Relations - Alzheimer's Association
Ronald Farkas
Clinical Team Leader, Division of Neurology Products - U.S. Food and Drug Administration
Robert Kowalski
Senior Vice President, Global Head of Drug Regulatory Affairs and U.S. Head of Development - Novartis
Rajesh Ranganathan
Director, Office of Translational Research - National Institute for Neurological Disorders and Stroke
Robert Temple
Deputy Director for Clinical Science, Center for Drug Evaluation and Research - U.S. Food and Drug Administration
Applying the breakthrough therapy criteria: Anti-infective/psychiatry/hematology
John Alexander
Clinical Team Lead, Division of Anti-Infective Products - U.S. Food and Drug Administration
Anne-Virginie Eggimann
Vice President, Regulatory Science - Bluebird Bio
David Fajgenbaum
Adjunct Assistant Professor of Medicine, Hematology/Oncology; University of Pennsylvania - Co-Founder and Executive Director; Castleman Disease Collaborative Network
Andrew Gustafson
Senior Director, U.S. Regulatory Policy and Advocacy - GlaxoSmithKline plc
Ilan Irony
Chief, General Medicine Branch, Office of Cellular, Tissue and Gene Therapies - U.S. Food and Drug Administration
John Jenkins
Director, Office of New Drugs - U.S. Food and Drug Administration
Lucas Kempf
Clinical Team Lead, Division of Psychiatric Products - U.S. Food and Drug Administration
John Powers
Associate Clinical Professor of Medicine; George Washington University School of Medicine - Senior Medical Scientist; Leidos Biomedical Research
Emil Tsai
Professor, Department of Psychiatry; Harbor-UCLA Medical Center - Chief Science Officer; SyneuRx International (Taiwan) Corporation
Summary and discussion of lessons learned
Jeff Allen
Executive Director - Friends of Cancer Research
Karin Van Baelen
Head of Global Regulatory Affairs - Janssen Pharmaceuticals
Gerald Dal Pan
Director, Office of Surveillance and Epidemiology - U.S. Food and Drug Administration
Kay Holcombe
Senior Vice President, Science Policy - Biotechnology Industry Organization
Peter Marks
Deputy Director, Center for Biologics Evaluation and Research - U.S. Food and Drug Administration
Richard Moscicki
Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug Administration
Rachel Sherman
Principal, Drug and Biological Drug Products - Greenleaf Health LLC
Closing remarks
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