FDA Guidance Update: Representing the Duchenne Community

An update on developing "Draft Guidance on the Clinical Investigation of drugs and biologics for Duchenne and Becker muscular dystrophy"


One of PPMD’s priorities is speeding development of reliable diagnostics and safe and effective medications for all individuals diagnosed with Duchenne or Becker muscular dystrophy. PPMD believes that FDA must be more responsive to the experiences and preferences of Duchenne families and all those living with Duchenne.

PPMD is proud that FDA has asked us to lead the community in the development of policy guidance. FDA guidance documents, including this draft guidance, do not establish legally enforceable rights or responsibilities. Instead, guidance documents describe the Agency’s current thinking on a topic, as well as describes the current landscape in a given disease area. We believe developing Draft Guidance on the Clinical Investigation of drugs and biologics for Duchenne and Becker muscular dystrophy builds on our commitment to smarter and quicker drug review, starting with "Putting Patients First: Recommendations to speed responsible acces... as well as our white paper on “Benefit Risk Assessments in Rare Disorders”.


The Duchenne Policy Forum in December was a great success. Nineteen FDA representatives participated in the meeting, including Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research. The community was given time to share their needs and recommend changes. There were excellent comments from all stakeholders, including FDA. We are off to a great start and plan to build on that momentum.

Next Steps

We now start the process of writing the policy guidance. This is a difficult task and we have promised the document in May. We are hopeful the entire community will be engaged in this effort and expect to have a document for review and public comment in April. Everyone's View Welcome.

A six member Steering Committee is in place and members will be announced next week. The Steering Committee has been formed in order to ensure the outcomes of the policy forum result in a draft guidance worthy of submission to the FDA. The Steering Committee will provide overall strategic direction to the guidance development from research to publication and launch. Committee members will collectively set the tone and direction of the draft guidance, ensuring that all perspectives from across the Duchenne community are represented. They will ensure the orderly progression of work and that all participants operate in a respectful fashion.

Working groups, made up of representatives of each part of the Duchenne community will be responsible for assembling, reviewing and contributing to the writing of each topical section for the guidance document. Each of seven working groups will address particular topics relevant to the development of the guidance. Each working group will have a Chairman and be accountable to the Steering Committee. The entire process will be supported by PPMD staff and consultants.

Parents and/or individuals with the diagnosis of Duchenne muscular dystrophy will be included in the Steering Committee and every working group.

A Community Advisory Panel will be developed and will allow additional participation of parents and individuals with Duchenne. They will be responsible to review progress and comment twice before publication. All are welcome.

On Monday, February 24, members of the Steering Committee and working groups will provide progress to date and may request additional comments and opinion about particular topics. This will take place in conjunction with our Annual Advocacy Conference.

Future Participation

As the guidance is developed, it will be posted to encourage public comment and to allow for broad scale participation.
Thanks to all of you who have already volunteered to help. Others are needed and everyone’s invited to participate. Please contact me at ryan@parentprojectmd.org if you wish to be a part of this important effort.

We will provide periodic blogs and web postings so that you can stay as informed as possible about the progress of this landmark document.

Up next: we will announce the Steering Committee members and working group topics.

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