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

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