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Regulatory News | 03 September 2014 | By Alexander Gaffney, RAC
The US Food and Drug Administration (FDA) is calling for public comment on a new draft guidance document developed by patient advocates and intended to accelerate the development of therapies to treat Duchenne Muscular Dystrophy (DMD).
In June 2014, Parent Project Muscular Dystrophy (PPMD) and other stakeholders submitted a draft guidance document to FDA which they said would "help accelerate development and review of potential therapies for DMD."
The guidance, the first developed by a patient advocacy group, has been seen by some as a harbinger of other patient-developed guidance documents. FDA has recently been encouraging more patient involvement in the regulatory process through its patient-centered drug development program, which was founded in 2012 after the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA). The thinking of the program, as reflected in the PPMD guidance, is that patients living with a disease are better arbiters of meaningful outcomes than regulators are.
In other words, patients—not FDA—should be able to have a significant say in what makes a treatment "significant" in its effects.
The matter is especially pronounced in the DMD community, whose patients suffer from progressive muscular degeneration, often leading to paralysis. A drug made by Sarepta Therapeutics has been hailed by some patients as being effective in alleviating some of DMD's effects, but FDA has thus far declined to approve the drug, eteplirsen, citing a lack of acceptable data. DMD patients first responded by launching a campaign, "The Race to Yes," which sought to pressure FDA into approving eteplirsen.
But eventually DMD patients banded together to release the draft guidance, perhaps recognizing that an institutional regulatory policy for approving DMD drugs would not only create a path for approval of eteplirsen, but other drugs as well.
At the time of the draft guidance document's publication, Janet Woodcock, director of FDA's Center for Drug Evaluation and Research (CDER), said the agency was "appreciative of the input of Duchenne patients and patient advocates."
"Their input will enhance the essential data-driven process and evaluation of new therapies," she added.
For more, check out Regulatory Focus' feature article on the DMD guidance. (Subscription Content)
Now FDA is formally opening up PPMD's draft guidance for further public comment.
In a 3 September 2014 Federal Register notice, FDA indicated that the guidance will be posted to a public docket (FDA-2014-D-1264) on the government's regulations.gov website. There, the guidance will be open for comment for a 30-day period—somewhat shorter than the 60-90 day comment periods given to most government-authored guidance documents.
FDA said it "values the guidance provided by the DMD community and is posting the document to seek additional guidance and public comment." Public comments are invited on both the guidance and "all matters relating to … DMD drug development," FDA added.
After the close of the comment period, FDA typically considers all comments and determines how—or if—they should affect the guidance.
Tags: DMD, Draft Guidance, PPMD, DMD Guidance, Patient-Centered Drug Development