An exciting update to the revised community-led Duchenne Guidance for FDA is that it has been officially submitted to the agency!
As you know, this Community Draft Guidance will serve as an update to the original guidance that was submitted in July 2014. Because of so many advancements in knowledge, understanding, care, clinical trials, and approvals, it is important for us to now “modernize” the Community-Led Guidance of 2014 to reflect this new landscape in Duchenne and Becker therapy development. This critical community guidance update process has involved over 100 stakeholders and has taken over a year to compile and submit.
This new version includes updates to sections of the 2014 document as well as additional original sections covering cardiac, gene therapy, and informed consent sections for the FDA’s consideration.
PPMD thanks the FDA for their receptive response to our original 2014 submission. We have requested that the FDA officially open a new docket for this revised community guidance initiative to allow for broad public attention and commentary. We will continue to keep you updated as this process moves along, but we are ecstatic about this important step being completed.View the submitted draft guidance >
About The Revised Guidance
The process of updating the Community Guidance has involved over 100 stakeholders including patients, caregivers, clinicians, researchers, drug companies, genetic counselors, and regulatory experts. This unprecedented effort brought together the leading experts in Duchenne serving on working groups in the following areas:
- Benefit Risk Preferences and Patient Experience
- Gene Therapy
- Natural History, Trial Design, Outcome Measures
Members of each working group can be viewed here.
Two advisory boards were also involved in the update overseeing the process and commenting on each section of the Guidance; A Pharmaceutical Advisory Board comprised of 21 companies currently developing therapies for Duchenne and Becker, and a Community Advisory Board that included 20 Duchenne partner organizations.
|Community Advisory Board||Pharmacy Advisory Board|
|ActionDuchenne||Antisense Therapeutics Limited|
|Best Day Ever Foundation||Astellas GT|
|Charley's Fund||Avidity Bio|
|Coalition Duchenne||Cumberland Pharma|
|Cure Duchenne||Daiichi Sankyo|
|EveryLife Foundation for Rare Diseases||Eli Lilly|
|JB's Keys to DMD||EntradaTX|
|Kindness Over Muscular Dystrophy||Pfizer|
|Little Hercules Foundation||PTC Bio|
|MD Canada||Regenex Bio|
|Muscular Dystrophy Association||Reveragen|
|My DMD Hero||Roche|
|Save Our Sons||Solid Bio|
|The Hope for Gus Foundation||Vertex|