July 9, 2013.  

PPMD met with Dr. Janet Woodcock and leadership from the Office of New Drugs, Medical Policy and the Division of Neurology Products, as well as, staff from the Office of Strategic Programs, Office of Health and Constituent Affairs.   The purpose of the meeting was to continue our efforts to provide the agency relevant information and data about Duchenne that will encourage/enable them to be flexible in their review process.  

We discussed PPMD’s views about regulatory strategy, initiated in 2012 with the Board Policy, previous interactions with the agency,  PPMD’s White Paper – Putting Patients First: Recommendations to Speed Responsible Access to Therapies for Duchenne muscular dystrophy, and Emil Kakkis’ White Paper focused on Accelerated Approval.   We briefly mentioned the EMA draft guidance and our (Treat NMD, PPMD, UPPMD, MDC) official response, as well as, the recent Treat NMD/EMA meeting in London.   


This primary objective of the meeting was to focus on PPMD’s Benefit Risk Survey and Analysis, as well as, to update the FDA with regard to Natural History Data.


In previous meetings with the agency, we discussed the need for BETTER data related to benefit/risk (B/R).  While we all agreed, B/R factors often are different in rare disease compared to common disease, concrete data to serve as reliable reference points for Duchenne did not exist. The agency suggested this data would be helpful and PPMD’s Holly Peay in collaboration with John Bridges developed a methodology and survey instrument to assess B/R in Duchenne. Holly presented data from PPMD’s survey using this instrument, showing that parents, above all else prioritized muscle function, and suggesting that parents equated this to quality of life (QOL). Dr. Woodcock stated she was impressed with this work and suggested she had hoped advocacy organizations would be willing to do the heavy lifting in this area. After some discussion, it was recommended we expand the survey to a more diverse group of parents and another survey for the adolescents/young adults.  Dr. Woodcock suggested Holly connect directly with one of her colleagues for advice about survey details of interest to the Agency.  In a meeting later that day, Dr. Woodcock said:

PPMD is doing truly impressive work. This is what we need and we were really excited to think about where this will lead.


Craig McDonald presented natural history data, suggesting its predictive ability and the domino effect of functional decline over time. The loss of the ability to stand predicts loss of ambulation, loss of ambulation predicts loss of the ability to feed oneself, and the loss of the ability to feed, predicts the need for ventilation. Colleagues from CDER neurology were very engaged in this discussion and the concordance of these data sets (CINRG, PTC placebo, Goemans).  


The meeting ended with next steps and a plan to meet again to engage the community in an open forum to discuss a range of important topics. We are very optimistic about the opportunity to build on these important survey methods and findings to improve the drug development climate for all Duchenne stakeholders. 

PPMD’s regulatory team continues to meet regularly to develop those next steps and I will continue to report on any developments.

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