Register

April

24
2015

8:45 am EDT - 4:45 pm EDT

Past Event

Breakthrough therapy designation: Exploring the qualifying criteria

  • Friday, April 24, 2015

    8:45 am - 4:45 pm EDT

The Park Hyatt Hotel
Ballroom

24th and M Streets, NW
Washington, DC

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

  • April 24
    • FDA opening remarks

      9:00 am - 9:15 am

      RM
      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

      9:15 am - 10:15 am

      RC
      Ryan Conrad Economist - U.S. Food and Drug Administration
      AH
      Afi Harrington Operations Research Analyst - U.S. Food and Drug Administration
      GS
      Grace Stark Oak Ridge Institute for Science and Education Fellow - U.S. Food and Drug Administration
      KT
      Kim Taylor Operations Research Analyst - U.S. Food and Drug Administration
    • Applying the breakthrough therapy criteria: Oncology

      10:30 am - 12:00 pm

      JC
      Jennie Chang Medical Officer, Division of Oncology Products II - U.S. Food and Drug Administration
      GD
      George Demetri Professor of Medicine; Harvard Medical School - Director, Ludwig Center at Harvard; Senior VP for Experimental Therapeutics, Dana-Farber Cancer Institute
      DK
      Dickran Kazandjian Medical Officer, Division of Oncology Products II - U.S. Food and Drug Administration
      DK
      David Kerstein Medical Director, Clinical Research and Development - Ariad Pharmaceuticals
      RM
      Richard Moscicki Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug Administration
      ER
      Eric Rubin Deputy Assistant Secretary, European and Eurasian Affairs - U.S. State Department
      ES
      Ellen Sigal Chairperson and Founder - Friends of Cancer Research
      MT
      Marc 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

      MC
      Maria Carrillo Chief Science Officer, Medical & Scientific Relations - Alzheimer's Association
      RF
      Ronald Farkas Clinical Team Leader, Division of Neurology Products - U.S. Food and Drug Administration
      RK
      Robert Kowalski Senior Vice President, Global Head of Drug Regulatory Affairs and U.S. Head of Development - Novartis
      RR
      Rajesh Ranganathan Director, Office of Translational Research - National Institute for Neurological Disorders and Stroke
      RT
      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

      2:00 pm - 3:30 pm

      JA
      John Alexander Clinical Team Lead, Division of Anti-Infective Products - U.S. Food and Drug Administration
      AE
      Anne-Virginie Eggimann Vice President, Regulatory Science - Bluebird Bio
      DF
      David Fajgenbaum Adjunct Assistant Professor of Medicine, Hematology/Oncology; University of Pennsylvania - Co-Founder and Executive Director; Castleman Disease Collaborative Network
      AG
      Andrew Gustafson Senior Director, U.S. Regulatory Policy and Advocacy - GlaxoSmithKline plc
      II
      Ilan Irony Chief, General Medicine Branch, Office of Cellular, Tissue and Gene Therapies - U.S. Food and Drug Administration
      JJ
      John Jenkins Director, Office of New Drugs - U.S. Food and Drug Administration
      LK
      Lucas Kempf Clinical Team Lead, Division of Psychiatric Products - U.S. Food and Drug Administration
      JP
      John Powers Associate Clinical Professor of Medicine; George Washington University School of Medicine - Senior Medical Scientist; Leidos Biomedical Research
      ET
      Emil 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

      JA
      Jeff Allen Executive Director - Friends of Cancer Research
      KVB
      Karin Van Baelen Head of Global Regulatory Affairs - Janssen Pharmaceuticals
      GDP
      Gerald Dal Pan Director, Office of Surveillance and Epidemiology - U.S. Food and Drug Administration
      KH
      Kay Holcombe Senior Vice President, Science Policy - Biotechnology Industry Organization
      PM
      Peter Marks Deputy Director, Center for Biologics Evaluation and Research - U.S. Food and Drug Administration
      RM
      Richard Moscicki Deputy Center Director for Science Operations, Center for Drug Evaluation and Research - U.S. Food and Drug Administration
      RS
      Rachel Sherman Principal, Drug and Biological Drug Products - Greenleaf Health LLC