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Strength Happens Together: PPMD Submits FDA Draft Guidance on Duchenne

June, 25, 2014 – Today is a landmark day for PPMD and the entire Duchenne community. We are so pleased that at our organization’s 20 year anniversary – after 2 decades of hard work and resolute persistence within the policy, legislative and regulatory arena – we are today able to provide to the US Food and Drug Administration the first-ever patient-initiated guidance to help accelerate development and review of potential therapies for Duchenne muscular dystrophy.

This guidance is a major milestone for the community and truly incorporates the patient voice – the community’s voice – in well-documented and quantifiable ways in pursuit of our common goal – to end Duchenne.

Why Develop a Guidance?

In February 2013, the European Medicines Agency (EMA) developed a guideline document focused on the “clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy.”

We felt that the FDA should have similar guidance and that our well-versed and passionate patient community could come together to provide as much input as possible to inform the document. On December 12, 2013, we convened a national PPMD-FDA Duchenne Policy Forum – with 20 FDA staff members in attendance – where the community made its needs and preferences in drug development known to the Agency. An agreement was reached that we – the Duchenne Community – led by PPMD, would develop the first-ever patient initiated draft guidance on Duchenne.

After an intensive five month long process, overseen by a steering committee, developed by working groups, and reviewed by a community advisory board, today we officially submitted to the FDA the Duchenne muscular dystrophy community’s draft of the Guidance for Industry: Duchenne Muscular Dystrophy: Developing Drugs for Treatment over the Spectrum of Disease, written to help accelerate the development and review of potential therapies for Duchenne.

Community Collaboration

We had over 80 dedicated expert stakeholders working together with one goal in mind – to provide the strongest guidance with the clearest patient voice to the FDA. The 80 plus experts represented many different organizations and perspectives – from parents and patients, medical experts, academics and researchers, the biopharmaceutical industry and dedicated advocates from across the muscular dystrophy community.

The submission of the document also included an “Imperatives” cover note, the voice of the patient and caregiver, it sets the tone and drives home that this is a patient-community centered approach to providing the FDA with critical and quantifiable patient and caregiver insight to impact drug development and review.

We need to ensure that we do everything possible to speed the development of ALL therapies in the pipeline, at all stages of development, and better inform companies about clinical trial design.

What Now?

This guidance is intended to serve as a focus for continued discussions among the FDA, the medical industry, sponsors, academic community, the patient and caregiver community, and the public.

We hope that, like the EMA Guidance document from 2013, the FDA also develops its own guidance specifically in this rare disease area – based on the input that we as a community have provided. 

We expect to have a follow on meeting about the guidance with the community and the agency by end of year.

This is a groundbreaking day for our community; we should be proud of what we’ve accomplished. We must continue to work together to provide much-needed patient and caregiver input as potential therapies move through the pipeline. 



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