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.
FDA opening remarks
9:00 am - 9:15 am
Breakthrough therapy designation: Two and a half years in
9:15 am - 10:15 amGrace Stark Oak Ridge Institute for Science and Education Fellow - U.S. Food and Drug Administration
Applying the breakthrough therapy criteria: Oncology
10:30 am - 12:00 pmGeorge Demetri Professor of Medicine; Harvard Medical School - Director, Ludwig Center at Harvard; Senior VP for Experimental Therapeutics, Dana-Farber Cancer InstituteDickran Kazandjian Medical Officer, Division of Oncology Products II - U.S. Food and Drug Administration
Applying the breakthrough therapy criteria: Neurology
1:00 pm - 2:00 pmRonald Farkas Clinical Team Leader, Division of Neurology Products - U.S. Food and Drug AdministrationRobert Kowalski Senior Vice President, Global Head of Drug Regulatory Affairs and U.S. Head of Development - NovartisRajesh Ranganathan Director, Office of Translational Research - National Institute for Neurological Disorders and Stroke
Applying the breakthrough therapy criteria: Anti-infective/psychiatry/hematology
2:00 pm - 3:30 pmJohn Alexander Clinical Team Lead, Division of Anti-Infective Products - U.S. Food and Drug AdministrationDavid Fajgenbaum Adjunct Assistant Professor of Medicine, Hematology/Oncology; University of Pennsylvania - Co-Founder and Executive Director; Castleman Disease Collaborative NetworkIlan Irony Chief, General Medicine Branch, Office of Cellular, Tissue and Gene Therapies - U.S. Food and Drug AdministrationLucas Kempf Clinical Team Lead, Division of Psychiatric Products - U.S. Food and Drug AdministrationJohn Powers Associate Clinical Professor of Medicine; George Washington University School of Medicine - Senior Medical Scientist; Leidos Biomedical Research
Summary and discussion of lessons learned
3:45 pm - 4:30 pmGerald Dal Pan Director, Office of Surveillance and Epidemiology - U.S. Food and Drug AdministrationPeter Marks Deputy Director, Center for Biologics Evaluation and Research - U.S. Food and Drug Administration