PPMD Calls for New Collaborative Approach to Benefit/Risk Analysis of Duchenne Therapies at FDA

Landmark Benefit/Risk Study Delivered to Agency to Support Regulatory Changes

WASHINGTON, Oct. 9, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD)  – the leading patient advocacy organization fighting to end Duchenne muscular dystrophy (Duchenne) – is proposing a collaboration with the Food & Drug Administration (FDA) to initiate a rare disease benefit/risk pilot program using Duchenne as the initial therapeutic area.

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In making this call for a Duchenne benefit/risk pilot program, PPMD issued a paper, entitled Benefit-Risk Assessments in Rare Disorders: The Case for Therapeutic Development in Duchenne Muscular Dystrophy as the Prototype for New Approaches, that thoroughly explores the benefit/risk paradigms used in both the U.S. and Europe, including patient survey data from a recent PPMD study, and that offers recommendations for ways to modify these frameworks to accommodate the unique needs of rare disease communities.

"A robust and functioning system to evaluate and consider the benefits and risks of a potential therapy is critical to the process of reviewing and approving novel therapies," Parent Project Muscular Dystrophy Founding President & CEO Pat Furlong said. "In making such decisions, particularly on therapies intended to treat rare diseases, it is absolutely essential that reviewers and regulators fully understand the perspective of the patients and parents impacted by such decisions. We also aspire to understand from regulators how to better provide such patient perspectives for their considerations."

While the evaluation of a product's potential benefits and risks is similar whether the product is intended to treat a highly prevalent and well-understood condition or a rare disease, the nature of rare diseases necessitates that special considerations be made.

For example, rare diseases are often not as well-characterized because they lack large patient populations studied over long periods of time, and many rare diseases do not have any approved therapies or means of prevention to serve as points of reference. To compensate for these deficiencies, regulators and other stakeholders must embrace modified assessment frameworks that protect patient safety while at the same time supporting innovation and efforts to develop life-enhancing and life-saving medications.

"Duchenne offers the FDA the ideal candidate for a rare disease benefit/risk pilot program. While Duchenne is a rare and fatal disease with no approved treatments, the disease progression is steadily becoming better understood and a number of potential therapies are in various stages of the development and clinical trial process," Furlong said. "As FDA prepares to receive these Duchenne drug applications, a Duchenne benefit/risk pilot and complementary research and development initiatives with government, industry, and academia are vital to our success.  PPMD is committed to creating the unique tools and delivering the strategic advocacy that only the patient community can provide to this vital work."

To this end, PPMD recently completed the first-ever rigorous scientific survey of benefit/risk expectations of nearly 120 Duchenne parents/guardians, producing a unique data resource to help power the proposed pilot. Data from the study illustrates a relatively high level of risk tolerance if the potential benefit is slowing or stopping the progression of muscle weakness. The preliminary survey data was presented to FDA leadership in a meeting in July, and PPMD continues to work with the agency to apply this approach to new knowledge.

"Our survey and now this paper are very much in line with FDA's patient-focused drug development initiative. Undertaking a rare disease benefit/risk assessment pilot program with Duchenne as the initial focus will help achieve continued progress in our fight to end Duchenne and provide a framework and foundation upon which similar rare disease benefit/risk programs can be built. We look forward to collaborating with FDA on this initiative," Furlong said.

About Duchenne muscular dystrophy
Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal muscle disorder affecting boys and young men that causes the loss of muscle function, wheelchair dependency and a decline in respiratory and cardiac function. 

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