HACKENSACK, N.J., June 9, 2014 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) – the leading patient advocacy organization fighting to end Duchenne muscular dystrophy (Duchenne) – announced that a recent treatment preferences and benefit/risk study led by the organization has been published in Clinical Therapeutics. The article entitled, "A Community-Engaged Approach to Quantifying Caregiver Preferences for the Benefits and Risks of Emerging Therapies for Duchenne Muscular Dystrophy" concludes that caregivers are willing to accept a serious risk when balanced with noncurative slowing or stopping of the progression of muscle weakness, even absent improvement in lifespan. These preferences should inform the Food and Drug Administration's (FDA's) benefit–risk assessment of emerging Duchenne therapies. This study highlights the synergistic integration of traditional advocacy methods and scientific approach to quantify benefit–risk preferences.
Holly Peay, PPMD's Vice President of Education and Outreach and the lead author of the Clinical Therapeutics article, explains the need for this study: "Rare diseases, especially serious pediatric disorders like Duchenne with no approved treatments, are different than common diseases. The FDA is aware of the special issues in rare disorders, but needs input on how to assess them differently—especially when considering progressive pediatric disorders. PPMD is aggressively engaged with the FDA to keep the community voice in that process. We want to encourage the FDA to integrate the Duchenne community perspective into their assessment of benefit and risk. At the same time, we feel it is important to provide quantified data to the FDA to complement their data-driven process. These types of approaches allow us to move beyond individual testimony and provide a much broader perspective from many more participants."
The study was completed in collaboration with John F P Bridges, PhD, an expert in the application of stated-preference methods and engaging patients and caregivers in benefit-risk analysis.
The study and resulting article is part of a larger effort to explore treatment preferences and risk tolerance in the Duchenne community, in an effort to inform regulatory decision making. PPMD advocated aggressively for the new tools provided to the agency by Congress through the Food and Drug Safety and Innovation Act (FDASIA), including a provision on patient engagement called Patient Focused Drug Development. Since passage of FDASIA, PPMD has embarked on an enhanced community-centered strategy that includes the benefit risk study and an unprecedented effort to develop draft guidance on Duchenne for the Agency and industry.
The power of PPMD's regulatory advocacy is evident; for the first time ever, the appropriations bill that funds the FDA includes report language focused on Duchenne muscular dystrophy and championed by PPMD advocates. The appropriation bill "…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." (http://appropriations.house.gov/uploadedfiles/hrpt-113-hr-fy2015-agriculture.pdf, page 58)
PPMD's Founding President & CEO Pat Furlong added, "PPMD is committed to creating the unique tools and delivering the strategic advocacy that only the patient community can provide so that potential therapies in Duchenne can be approved safely and quickly."
Peay HL, Hollin I, Fischer R, Bridges JFB. A community-engaged approach to quantifying caregiver preferences for the benefits and risks of emerging therapies for Duchenne muscular dystrophy.Clinical Therapeutics 2014;36:624–637.
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.