Update on FDA Program to Streamline Drug Approval for Life-Threatening Diseases


On April 12, Friends of Cancer Research held a briefing on Capitol Hill on the topic of Breakthrough Therapy Designation: Successes, Lessons Learned and Future Strategies. Through the breakthrough therapy program, which followed the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012, a new drug may be designated as a breakthrough therapy by the Food and Drug Administration (FDA) if it is intended to treat a serious or life-threatening disease and preliminary clinical evidence suggests it provides a substantial improvement over existing therapies.

Since the breakthrough therapy designation was implemented in 2012, the FDA has received over 300 requests, granted over 100 designations, and approved 40 treatments.

Senator Michael Bennet (D-CO) was the keynote speaker at the briefing. Since the Senator, along with Orrin Hatch (R-UT) and Richard Burr (R-NC), introduced the bill to create the designation, he has stayed involved with the program. Senator Bennet expressed his optimism for the program and for what can be achieved through breakthrough therapy designation.

The panel of speakers included Janet Woodcock, Director, Center for Drug Evaluation and Research, FDA; Ellen Sigal, Chair & Founder, Friends of Cancer Research; Mathias Hukkelhoven, Sr. VP, Global Regulatory, Safety and Biometrics, Bristol-Myers Squibb; David Ross, Senior Director, Global Pharmaceutical Regulatory Affairs, Abbvie; Stephanie Dunn Haney, Patient Advocate; and Kate Rawson, Senior Editor, Prevision Policy, served as the moderator.

The panel discussion focused primarily on the success of the program thus far. Drugs with breakthrough designation have seen much faster development and approval times. The designated drugs have shown to be effective and with the breakthrough designation, they are reaching the hands of patients more quickly.

Stephanie Dunn Haney was present on the panel and spoke of her lung cancer diagnosis at the age of 39. In the beginning she struggled with traditional chemotherapy. She has since been on two breakthrough therapies over the last five years, and she is now living a normal life at the age of 47, symptom free and with only minor side effects.

Of the 40 approved breakthrough therapies, half are for cancer. When asked why this is, the panel explained that ultimately science drives drug development and the science and sustained research behind cancer has been advancing. Designations are also given to non-cancer drugs, and the numbers will likely increase as research advances in other diseases.

When discussing the future of drug development and the breakthrough therapy designation, panelists agreed that integration and collaboration are very important. While disease experts are necessary, they have to be working together in order to successfully advance research and development.

This was the fourth briefing held by Friends of Cancer Research (Friends) since before the breakthrough designation program was implemented. Friends is a leading voice in advocating for policies and solutions that will get treatments to patients in the safest and quickest way possible.

The Meso Foundation is proud to work in collaboration with Friends of Cancer Research. To see others with whom we collaborate, click here.

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