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Breakthrough therapy designation: Exploring the qualifying criteria

Past Event

Breakthrough therapy designation: Two and a half years in

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Breakthrough therapy designation: Two and a half years in

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Applying the breakthrough therapy criteria: Oncology

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Applying the breakthrough therapy criteria: Neurology

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

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Summary and discussion of lessons learned

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

R

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

R

Ryan Conrad

Economist - U.S. Food and Drug Administration

A

Afi Harrington

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

G

Grace Stark

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

K

Kim Taylor

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

Applying the breakthrough therapy criteria: Oncology

J

Jennie Chang

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

G

George Demetri

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

D

Dickran Kazandjian

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

D

David Kerstein

Medical Director, Clinical Research and Development - Ariad Pharmaceuticals

R

Richard Moscicki

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

E

Eric Rubin

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

E

Ellen Sigal

Chairperson and Founder - Friends of Cancer Research

M

Marc Theoret

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

Applying the breakthrough therapy criteria: Neurology

M

Maria Carrillo

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

R

Ronald Farkas

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

R

Robert Kowalski

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

R

Rajesh Ranganathan

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

R

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

J

John Alexander

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

D

David Fajgenbaum

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

A

Andrew Gustafson

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

I

Ilan Irony

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

J

John Jenkins

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

L

Lucas Kempf

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

J

John Powers

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

E

Emil Tsai

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

Summary and discussion of lessons learned

J

Jeff Allen

Executive Director - Friends of Cancer Research

G

Gerald Dal Pan

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

K

Kay Holcombe

Senior Vice President, Science Policy - Biotechnology Industry Organization

P

Peter Marks

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

R

Richard Moscicki

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

R

Rachel Sherman

Principal, Drug and Biological Drug Products - Greenleaf Health LLC

Closing remarks

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