PPMD Calls for Greater Transparency and Accountability of FDA Patient Engagement Efforts

WASHINGTON, Dec. 8, 2014 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne) is urging Congress to build upon gains in recent years to further strengthen and enhance programs to ensure the patient voice is heeded during the drug development process during a Congressional briefing tomorrow at noon.

A champion of policies to enhance the voice of the patient within vital Food and Drug Administration (FDA) deliberations, PPMD is advocating for provisions within the 21st Century Cures Initiatives focused on measuring how the agency is or is not using new patient engagement tools. PPMD and other stakeholders will be presenting on the reform proposal during the briefing tomorrow afternoon.

Over the past year, PPMD has led the Duchenne and larger rare disease community by organizing a comprehensive multi-stakeholder effort to draft and submit to the FDA a first-ever patient advocacy initiated draft guidance for industry developing therapies for Duchenne. The draft guidance was submitted to FDA in late June and a public comment period closed in early October.

In addition to the draft guidance, PPMD has partnered with Johns Hopkins University to produce a scientific survey assessing Duchenne primary caregivers' views on benefit/risk of potential therapies. FDA officials have commended these products and have urged other patient communities to consider taking similar actions.

"We have seen significant progress over the years in how the FDA pays attention to the voice of the patient. PPMD has been focused relentlessly on this issue and has produced several groundbreaking tools intended to achieve this goal. Now, we must know that this work is bearing fruit and that FDA will actually be using these new tools as they review therapies for Duchenne and beyond," said PPMD Founding President & CEO Pat Furlong.

Dubbed the Patient-Impact Assessment Act, PPMD would like to see a simple and publicly accessible measurement tool developed whereby FDA reviews could note how they did or did not take various patient-focused drug development tools and resources into account in evaluating products and making their decisions.

"Such an assessment tool would shed light into how Patient Focused Drug Development (PFDD) tools and authorities are or are not being used and would help identify gaps or shortcomings in the space," Furlong said.

In addition to PPMD, leaders from FasterCures and the Pharmaceutical Research and Manufacturers of America (PhRMA) will also be presenting on related PFDD efforts and recommendations.

"Across the whole continuum of discovery, development, and delivery a new focus on the patient is changing culture, practice, and policy. PPMD has pioneered a new model for advancing the science of patient input, one that will help transform the way in which patient perspectives are collected and understood. FasterCures champions a broader partnership of patient, industry, and government organizations to further define the science of patient input and use it to inform decision-making at all levels; the 21^st Century Cures Initiative has reinforced the potential for this collaborative approach," said Kim McCleary, director of strategic initiatives at FasterCures.

The briefing will be held tomorrow from noon to 1 p.m. in room 2218 of the Rayburn House Office Building. To learn more about PPMD's advocacy agenda, visit the PPMD website.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500-5,000 live male births (about 20,000 new cases worldwide each year). Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures.

Duchenne results in progressive loss of strength and is caused by a mutation in the gene that encodes for dystrophin. Because dystrophin is absent, the muscle cells are easily damaged. The progressive muscle weakness leads to serious medical problems, particularly issues relating to the heart and lungs. Young men with Duchenne typically live into their late twenties.

Duchenne can be passed from parent to child, but approximately 35 percent of cases occur because of a random spontaneous mutation. In other words, it can affect anyone. Although there are medical treatments that may help slow its progression, there is currently no cure for Duchenne.

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.

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SOURCE Parent Project Muscular Dystrophy

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