Congressional Committee That Funds the FDA Recognizes PPMD-Led Efforts on Benefit/Risk, Draft Guidance; Applauds Collaboration with Agency

For the first time ever, the appropriations bill that funds the Food and Drug Administration includes report language focused on Duchenne muscular dystrophy and championed by PPMD advocates.


This week the House Appropriations Committee approved the Fiscal Year 2015 Department of Agriculture and FDA spending bill, clearing the way for the full House to act on the measure. The bill report praises the collaboration between the Duchenne community and FDA to enhance understanding of benefit/risk considerations and to develop guidance to help accelerate access to Duchenne therapies.


This is a significant milestone for the Duchenne community that has worked so hard over the years on both the benefit/risk project and draft guidance that we will soon be submitting to FDA for review. This particular spending bill does not typically focus on specific diseases or conditions. However, the committee has recognized the landmark nature of this work and has commended the collaboration we have fostered with the FDA. We thank Congresswoman Doris Matsui and Congressman Peter King for championing this language, and we thank the many members of Congress who lent their support.


The full language is as follows:  

Duchenne Muscular Dystrophy—The Committee commends the collaboration between FDA and the Duchenne Muscular Dystrophy community to advance useful regulatory tools for benefit-risk considerations in this disease population and drug development guidance. The Committee supports the agency’s engagement with the patient population for these purposes and to enable the appropriate use of regulatory flexibility as provided in FDASIA.

Last year, PPMD led the first-ever scientific survey of the benefit/risk perspectives of Duchenne parents, surveying nearly 120 parents to better understand their risk thresholds. That information has been conveyed to FDA and will hopefully inform agency review of Duchenne drug applications.


In 2012, PPMD strongly supported many of the patient-focused drug development reforms included in the FDA Safety & Innovation Act (FDASIA) to help accelerate development of therapies for Duchenne and other rare diseases. And in the coming days, PPMD will be submitting the first-ever patient advocacy community-originated draft guidance on drug development to the FDA for their consideration.


This language is a meaningful result for our community to help accelerate access to therapies and treatments and to achieve our goal of ending Duchenne.


Please be sure to thank both our Champions, Representative Mastui and Representative King on social media for their unwavering support of our community!

Thank Congresswoman Doris Matsui!

Thank Congressman Peter King!

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