FDA’s Patient Focused Drug Development Initiative – Request for Public Comment

PPMD and other groups submit joint statement making the case for neuromuscular disorders affecting children

As many advocates know from earlier blog postings, PPMD has been deeply and passionately engaged to enhance how the Food and Drug Administration (FDA) goes about its business of reviewing potential therapies and treatments for patients with Duchenne. Our work is focusing on ensuring that the agency moves as expeditiously as possible and takes into account the potential benefits of a treatment as well as the risks, something that is particularly critical with life-threatening conditions that lack other treatment options.

PPMD's efforts are multi-pronged and include ongoing and direct engagement with leaders in the neurological products review section of the agency, dialogue with industry working to develop therapies and treatments, and Congressional engagement. Earlier this year, PPMD was very active in helping to enact a number of measures through the FDA user fee law to provide the agency with the authority and direction to move as quickly as possible in conducting product reviews, including engaging more directly with patients and considering a wider variety of data and evidence in submissions. While the enactment of the law is a major step forward, much of the specifics remain to be written over the coming months and even years through a number of proposed new rules, guidance,and regulation.

PPMD is paying close attention to ensure the desires of our patients and families are heard loudly and clearly. Most recently, PPMD worked closely with A-T Children's Project, Friedreich's Ataxia Research Alliance (FARA), the Muscular Dystorphy Association (MDA), and the National Organization for Rare Disease (NORD) on a joint letter in response to a request for comment from FDA to urge that the agency strongly consider convening a public meeting dedicated exclusively to Degenerative Neuromuscular Disorders Affecting Childrenas part of the agency's patient-focused drug development initiative.

The disorders in this disease area would include:

  1. the Muscular Dystrophies such as Duchenne muscular dystrophy, Becker muscular dystrophy, myotonic muscular dystrophy, congenital muscular dystrophy, Emery-Dreifuss muscular dystrophy, fascioscapulohumeral muscular dystrophy, and limb girdle muscular dystrophy;
  2. Friedreich ataxia;
  3. ataxia telangiectasia;
  4. spinal muscular atrophy;
  5. Charcot-Marie-Tooth disease;
  6. congenital myasthenia gravis;
  7. congenital myopathies; and
  8. severe pediatric onset forms of metabolic diseases of muscle, such as mitochondrial myopathies.

We believe that the key factors of severity of disease and impact on quality of life – as well as unmet medical needs – are so close to identical,that the stated objectives of the public meetings to be scheduled over the next five years could be accomplished for this whole set of disorders in a single meeting.

With so many rare diseases competing to be the focus of one of the 20 meetings, PPMD and its allies believe the best and most efficient way to tackle the benefit risk analysis of many of these similar diseases is to group them together. This will save time and resources, two things that are on short supply to the FDA, our community, and many other organizations. In the meantime, as the FDA works through their mandate, PPMD is launching its own benefit risk project with a survey for families and patients, set to launch in November.

In addition to the collaborative comment letter, PPMD continues to work across the rare disease community to ensure that Duchenne concerns are heard on this and other FDA issues. PPMD has also been participating in recent public meetings with the agency. This is advocacy that will likely increase as more proposed rules and regulations are issued in the coming months.


Ryan Fischer, Director of Outreach & Advocacy
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