12:00 AM
Jun 15, 2015
 |  BioCentury  |  Regulation

Patients lead the way

How a patient advocacy group led FDA to publish a draft guidance for DMD

FDA's draft guidance on Duchenne muscular dystrophy and related dystrophinopathies is the culmination of an extensive effort led by a patient advocacy group that enlisted the support of patients, caregivers, academia and industry.

DMD drug development has been hindered by a lack of defined outcome measures and understanding of surrogate markers or biomarkers that could support approval. To address this, FDA invited Parent Project Muscular Dystrophy (PPMD) and the DMD community to submit a proposed draft guidance that could clarify a clinical development pathway for approval.

FDA spokesperson Kristofer Baumgartner said in a statement to BioCentury that PPMD's submission is the first time in the Center for Drug Evaluation and Research's knowledge that an advocacy group has submitted a proposed draft guidance, and that this type of engagement is an example of how early input from patients and caregivers can contribute to drug development.

PPMD President and CEO Pat Furlong told BioCentury that the group and its collaborators spent years looking for ways to ensure that DMD studies and endpoints would be sensitive to both effective therapies, and benefits that are meaningful to patients and caregivers.

"What we didn't want to have happen is trials that would fail because there weren't sufficiently sensitive measures to understand what was happening, or what was of benefit, or what the patients wanted and needed to see," she said.

PPMD partnered with John Bridges, an associate professor at the Johns Hopkins Bloomberg School of Public Health, to conduct a study that would address one piece of the puzzle: how DMD patients and caregivers perceive benefits and risks. Results from the study were published in 2014 in Clinical Therapeutics.

Meanwhile, companies and research institutions in the space were gathering natural history data to help interpret endpoints that may not be consistently sensitive across different stages of the disease.

Both those topics came into play at a December 2013 public-private policy forum, at which PPMD laid out its case to FDA that the state of knowledge was mature enough for a guidance.

She said FDA declined to draft a guidance itself, citing time and resource constraints,...

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