As you know, PPMD recently facilitated a multi-stakeholder process involving patients, parents, investigators, industry, and other interested parties which culminated in a draft guidance document “Guidance for Industry: Duchenne Muscular Dystrophy, Developing Drugs for Treatment over the Spectrum of Disease” which was submitted to the Docket for consideration by FDA on June 26, 2014. Many of you participated by serving on the Community Advisory Board or including your feedback during the public comment period. This draft guidance has not only gotten the attention of the FDA, but of the rare disease community and mainstream media (read a great article published by The Wall Street Journal). It has quickly become a map for rare diseases as they, like us, seek to inform regulatory agencies about their conditions.
Subsequent to that submission, PPMD and the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) collaborated in convening a working group to explore contemporary issues in Duchenne cardiology (read a recap here). Attendees at that meeting included experts in pediatric and adult cardiology, clinical research, patient advocacy, and representatives of the FDA. During discussions at that meeting, the FDA indicated interest in receiving recommendations for cardiac surveillance and cardiac markers, to be included in current and future Duchenne clinical trials. In response to those questions, recommendations were developed by the Expert Working Group and submitted to the FDA for consideration for inclusion in the draft guidance. Almost immediately, we received a thank you for the recommendations and submitted them to the Docket.
PPMD appreciates FDA’s interest in these efforts to provide the agency with stakeholder views about trial processes. We are optimistic that this continued open dialogue with FDA leadership will keep Duchenne a priority of the agency. And again, we just want to thank you for working with us to keep the FDA informed and educated. Strength Happens Together.