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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.
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
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April 24
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FDA opening remarks
9:00 am - 9:15 am
RMRichard 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
9:15 am - 10:15 am
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Applying the breakthrough therapy criteria: Oncology
10:30 am - 12:00 pm
JCJennie Chang Medical Officer, Division of Oncology Products II - U.S. Food and Drug AdministrationGDGeorge Demetri Professor of Medicine; Harvard Medical School - Director, Ludwig Center at Harvard; Senior VP for Experimental Therapeutics, Dana-Farber Cancer InstituteDKDickran Kazandjian Medical Officer, Division of Oncology Products II - U.S. Food and Drug AdministrationDKDavid Kerstein Medical Director, Clinical Research and Development - Ariad PharmaceuticalsRMRichard Moscicki Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug AdministrationEREric Rubin Deputy Assistant Secretary, European and Eurasian Affairs - U.S. State DepartmentESEllen Sigal Chairperson and Founder - Friends of Cancer ResearchMTMarc Theoret Clinical Team Lead, Division of Oncology Products II - U.S. Food and Drug Administration -
Applying the breakthrough therapy criteria: Neurology
1:00 pm - 2:00 pm
MCMaria Carrillo Chief Science Officer, Medical & Scientific Relations - Alzheimer's AssociationRFRonald Farkas Clinical Team Leader, Division of Neurology Products - U.S. Food and Drug AdministrationRKRobert Kowalski Senior Vice President, Global Head of Drug Regulatory Affairs and U.S. Head of Development - NovartisRRRajesh Ranganathan Director, Office of Translational Research - National Institute for Neurological Disorders and StrokeRTRobert 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
2:00 pm - 3:30 pm
JAJohn Alexander Clinical Team Lead, Division of Anti-Infective Products - U.S. Food and Drug AdministrationAEAnne-Virginie Eggimann Vice President, Regulatory Science - Bluebird BioDFDavid Fajgenbaum Adjunct Assistant Professor of Medicine, Hematology/Oncology; University of Pennsylvania - Co-Founder and Executive Director; Castleman Disease Collaborative NetworkAGAndrew Gustafson Senior Director, U.S. Regulatory Policy and Advocacy - GlaxoSmithKline plcIIIlan Irony Chief, General Medicine Branch, Office of Cellular, Tissue and Gene Therapies - U.S. Food and Drug AdministrationJJJohn Jenkins Director, Office of New Drugs - U.S. Food and Drug AdministrationLKLucas Kempf Clinical Team Lead, Division of Psychiatric Products - U.S. Food and Drug AdministrationJPJohn Powers Associate Clinical Professor of Medicine; George Washington University School of Medicine - Senior Medical Scientist; Leidos Biomedical ResearchETEmil Tsai Professor, Department of Psychiatry; Harbor-UCLA Medical Center - Chief Science Officer; SyneuRx International (Taiwan) Corporation -
Summary and discussion of lessons learned
3:45 pm - 4:30 pm
JAJeff Allen Executive Director - Friends of Cancer ResearchKVBKarin Van Baelen Head of Global Regulatory Affairs - Janssen PharmaceuticalsGDPGerald Dal Pan Director, Office of Surveillance and Epidemiology - U.S. Food and Drug AdministrationKHKay Holcombe Senior Vice President, Science Policy - Biotechnology Industry OrganizationPMPeter Marks Deputy Director, Center for Biologics Evaluation and Research - U.S. Food and Drug AdministrationRMRichard Moscicki Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug AdministrationRSRachel Sherman Principal, Drug and Biological Drug Products - Greenleaf Health LLC
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