debra of America Announces Drug Development Meeting with the FDA

Please Read, Sign Up, and Have your Voice Heard!

debra is excited to announce that we will be meeting directly with the U.S. Food and Drug Administration (FDA) on April 6 and you are invited to attend and participate. This incredibly important meeting is our opportunity to educate the FDA about living daily with EB. We will discuss 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. We may be adding more panelists and we need you, the EB community to be in the audience where you will actively participate through live polling, question and answer periods, and commentary. This is a great opportunity to get involved in one of the most important ways we can.

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.

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.

When and where is this meeting?

The meeting will take place on April 6, 2018 from 12-5:30 PM at the Marriott College Park Hotel & Conference Center in Maryland.

Will debra provide financial assistance to those who attend?

debra will pay for a one-night hotel stay (April 5 or 6), food on April 6, and a travel stipend may be awarded on a case-by-case basis. For more information about financial assitance, please contact brett [at] debra [dot] org.