PPMD to Lead Duchenne Community in Developing Draft Guidance to Accelerate Drug Development and FDA Review

Dec 16, 2013, 14:00 ET from Parent Project Muscular Dystrophy

Guidance to be Submitted to FDA by Mid-2014; Annual PPMD Advocacy Conference to Include Community Review 

HACKENSACK, N.J., Dec. 16, 2013 /PRNewswire-USNewswire/ -- During a landmark policy forum held with  U.S. Food and Drug Administration (FDA) leaders last week, Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne), announced it would lead a community-wide effort to develop guidance to inform and accelerate the agency's review of Duchenne candidate therapies.

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The guidance is part of a longstanding, comprehensive, and strategic effort by PPMD focused on accelerating drug development to deliver safe and effective therapies to patients with Duchenne as quickly as possible.

The draft guidance comes on the heels of the first-ever scientific survey of the Duchenne parent community on benefit/risk tolerance. It also comes amidst ongoing PPMD engagement with FDA leaders and Congress to ensure the perspective and concerns of the Duchenne patient community are heard in this most important area.

"We will not rest until the FDA has all of the tools it needs to accelerate its review of Duchenne therapies so effective treatments can be delivered to our children," PPMD Founding President Pat Furlong said. "We have engaged the agency over several months to identify and develop the tools and knowledge they have told us they need, and we have worked extensively to understand the needs of industry developing these products. The partnership on display in last week's policy forum must lead to success in the drug approval process."

With multiple candidate therapies in clinical testing, clear guidance from FDA to industry is needed now. During the forum, FDA provided extensive comments to aid this process. The draft guidance document will include engagement of company, patient, provider, and other stakeholder groups and is intended to present clear direction to industry on a host of important issues such as:

  • Scientifically validated endpoints that can be used to design clinical trials;
  • Adaptive and other modified trial designs recognizing the small patient populations of Duchenne and other rare diseases; and
  • Eliminate the use of placebo.

The guidance framework will be released in mid-February in advance of the annual PPMD Advocacy Conference. A portion of that event will be dedicated to rolling out the guidance concepts and beginning a community-wide public comment process. The feedback received from the stakeholders will be integrated into the draft guidance to be delivered to FDA in May 2014 for the agency's further consideration.  During the Advocacy Conference, PPMD also will update Congress on the guidance progress while urging passage of the Muscular Dystrophy Community Assistance, Research and Education Amendments of 2013.

"One thing our children do not have is time. PPMD will continue to lead the community forward by developing draft guidance for the FDA to consider. With the input of patients, parents, researchers, clinicians, and industry, we will develop a critical tool that we believe will help speed new therapies into their lives," Furlong added.

About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.

SOURCE Parent Project Muscular Dystrophy