PPMD President Pat Furlong Testifies Before Key Congressional Committee

HACKENSACK, N.J., July 11, 2014 /PRNewswire-USNewswire/ -- Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne), will testify today before a key Congressional committee focused on accelerating development and delivery of therapies to patients.

Furlong will appear before the House Energy and Commerce Committee's Subcommittee on Health as part of the committee's ongoing 21^st Century Cures Initiative. She will be one of four patient advocacy representatives selected to testify and will spotlight PPMD's cutting-edge benefit/risk survey and guidance development projects.

"I am honored that PPMD was chosen to offer the Duchenne patient perspective as to what we can – and must – do to accelerate the development and delivery of therapies to patients in need. This is the core mission of PPMD as we strive to end Duchenne," Furlong said. "The Energy and Commerce Committee has been a spark behind many reforms, including the Children's Health Act, MD CARE Act and the FDA Safety & Innovation Act that have helped get the field to where it is today, and I am confident the 21^st Century Cures Initiative will help us address additional gaps to accelerate progress further."

Furlong will focus on two PPMD-led projects that build upon the patient-focused drug development provisions within FDASIA and that seek to address major barriers. These projects are:

• The first-ever scientific survey of the Duchenne community on benefit and risk preferences; and
• The first-ever, patient-initiated effort to develop a draft guidance document for FDA to use with industry sponsors.

Both projects represent a first for a rare disease and are part of PPMD's ongoing advocacy agenda that focuses on encouraging regulatory agencies to incorporate the patient perspective in the decision-making process. Both involved multiple stakeholders including patients and caregivers, clinicians, researchers, industry, and government and are strong examples of public-private partnerships. Now, the Duchenne community expects FDA to step up its role so both projects achieve their desired impact.

In addition to urging FDA to move forward on both the survey and guidance, Furlong will suggest additional ways Congress and FDA can deepen the inclusion of the patient voice in FDA decision-making and will suggest ways to accelerate the drug development process, particularly during the clinical trials stages.

"The Duchenne and larger rare disease communities need therapies and we need them right now. With about 7,000 rare diseases, we simply don't have the time or money for a business-as-usual approach. I urge the committee to recognize this and look forward to continuing to help advance this most important work," she said.

For more about today's hearing, visit the Energy & Commerce Committee website. To learn more about PPMD's draft guidance for the FDA, visit PPMD's website.

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 (PPMD)