Meeting with the FDA: Our topics for discussion

On July 9, PPMD will be meeting with individuals from FDA to include CDER, Division of Neurology, Office of Strategic Programs and Staff from the Office of health and Constituent Affairs.

The topics for discussion will include a review of PPMD’s Board Policy, developed in 2011. This policy embraces the following principles supporting an enhanced regulatory framework to advance development of drugs for rare diseases:


  • Duchenne patients want, need and deserve therapies that are effective and safe in treating the symptoms and altering the trajectory of Duchenne.

  • Enhanced collaborative dialogue and the sharing of expertise between reviewers, industry sponsors, patient advocates and research organizations across the drug development and review process are critical for achieving progress. Duchenne patients and their caregivers have unique perspectives on the natural history, progression and care of this disease that may be of use to regulators considering the benefits and risks of new therapies.

  • The Duchenne community (parents and patients) understands and respects the importance of reliable standards for the assessment and approval of new treatments for all diseases, including Duchenne.

  • Unique benefit-risk considerations inherent with a progressive and fatal condition like Duchenne must be considered when evaluating the safety and efficacy of a compound.

  • Flexibility and scientific judgment, as appropriate, by FDA reviewers should be encouraged when evaluating investigational studies and marketing applications for drugs for rare diseases, including Duchenne, given the inherent limitations in conducting large trials.

  • Randomized, double-blind, placebo-controlled clinical trials are critical. In Duchenne and other rare diseases, adaptive designs and other innovative approaches are needed to better measure the safety and effectiveness of experimental agents.


We intend to discuss:


  1. White Paper: Putting Patients’ First: Recommendations to speed responsible access to new therapies for Duchenne muscular dystrophy and other rare, serious and life-threatening neurologic disorders. Specifically the issue of Adaptive Approval, as recommended in the PCAST report to the president.

  2. Benefit/Risk: In previous meetings with the agency, PPMD/FDA discussed B/R as it applies to Duchenne (Rare Disorders), suggesting B/R is different in Rare vs Common conditions. FDA agreed, though suggested they had no data to confirm the hypothesis. PPMD’s Holly Peay in collaboration with Dr. John Bridges (Hopkins) developed and piloted a survey instrument. Results and draft report will be provided to the agency

    a. How can the agency use this unique data?

    b. Are there additional questions/concerns the agency needs to learn?

    c. Would it be useful to expand the survey to a more diverse population and to young adults/adults with Duchenne?

  3. Natural History: Within the last 3 years, we have learned a great deal about the 6mwt, its relationship to TFT (timed function testing) and NSAA (north star assessment).  We have learned 30m decline in the 6mwt translates to 10% decline in function. These data were presented during a recent meeting in London with Treat NMD and representatives from EMA. Our intention is to ensure information provided to regulatory agencies is consistent.  It is our hope that the agency will embrace natural history data, to encourage novel trial design which includes boys across the spectrum of disease, to consider either eliminating use of placebo in RCT or shortening the time required for placebo studies and/or using the boy as his own control.

  4. Policy Forum: We would like to engage are hopeful the agency will agree to participate in a policy forum in the Fall (September), similar to that held in London, organized by Treat NMD/Duchenne Parent Project and PPMD in response to the Draft EMA guidelines.

    We intend to include topics such as – novel trial designs, surrogates and biomarkers, animal data, compassion (families with non-ambulatory children, families with more than one Duchenne child) and extrapolation to insure broad (inclusive) labeling to ensure all boys have access and reimbursement for therapies.

    Our goal is to support all drugs in development. We want to see them approved… all of them that are determined to be safe and effective. We believe that boys, our sons (and daughters) need and deserve access to therapies, that they should have options and we believe a cocktail of compounds will be required to halt progression. This is our goal, our mission and our commitment.

We intend to post a full report from the meeting on July 11.



Pat Furlong, Founding President, CEO
Read more PPMD Staff Blogs

Views: 710


You need to be a member of PPMD Community to add comments!

Join PPMD Community

Need help using this community site? Visit Ning's Help Page.



© 2020   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service