debra of America Announces Drug Development Meeting with the FDA

House Committee on Appropriations: Subcommittee on Labor, Health and Human Services, Education and Related Agencies Public Witness Testimony

Testimony of Brett Kopelan, Executive Director, debra of America - April 26, 2018

debra is excited to announce that we met directly with the U.S. Food and Drug Administration (FDA) on April 6. The general public was invited to attend and participate. This incredibly important meeting was our opportunity to educate the FDA about living daily with EB. We discussed symptoms, issues we face, the importance of therapy development and what it means to us. Recent legislation has directed the FDA to consider the voice of the patient in their decision making process about clinical trials and drug or therapy approvals. We can’t emphasize enough how important this meeting is. It’s called an Externally Led Patient Focused Drug Development Meeting and it will help guide the FDA in determining the regulatory pathway of all EB research and therapy development.

debra has put together two panels of those with EB and those caring for people with EB to present testimony to the FDA.

What is an Externally-Led Patient Focused Drug Development Meeting?

As part of FDA’s commitments under the Prescription Drug User Fee Act reauthorization of 2012, FDA has taken several steps to inform assessment of benefit-risk in the Center for Drug Evaluation and Research's regulatory decisions concerning new drugs. The Patient-Focused Drug Development (PFDD) initiative is part of FDA commitments under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The PFDD initiative aims to more systematically obtain the patient perspective on specific diseases and their treatments.

The patient perspective is critical in helping FDA understand the context in which regulatory decisions are made for new drugs. PFDD meetings give FDA an important opportunity to hear directly from patients, patient advocates, and caretakers about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, and patients’ experiences with currently available treatments. This input can inform FDA’s decisions and oversight both during drug development and during the review of a marketing application.