National Spotlight on FDA and Duchenne Muscular Dystrophy: What This Means for Everyone with a Rare Disease

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FdaNext week, a Food and Drug Administration panel of experts will hold the first of two meetings to review two drugs designed to slow the muscle wasting effects associated with Duchenne Muscular Dystrophy (DMD).  These much-anticipated events provide hope for children and families living with this devastating and fatal disease, which currently has no treatment or cure.   There are also takeaways here for the wider rare community affected by the 7,000 known rare diseases/disorders, the majority of which are without any sort of treatment.

Earlier this week, STAT reporter Ed Silverman looked at how the DMD community, FDA and drug developers arrived at this point.  Pat Furlong, a NORD board member and chief executive of Parent Project Muscular Dystrophy (PPMD), a NORD member organization, cited as one reason 2012 legislation (the FDA Safety and Innovation Act) that “opened the door for patient involvement.”

NORD advocates for, and works to facilitate, stronger patient participation in every step of the drug development process.  This was a reoccurring topic at our recent Rare Diseases and Orphan Products Breakthrough Summit, where we covered how increased patient involvement in research via natural history studies and registries, clinical trial design, and working with regulators, along with scientific advances (see Precision Medicine), have helped to accelerate orphan drug development.  In 2014, the FDA approved more orphan drugs than ever before.  We are on track to exceed that number in 2015.

To learn more about how you can participate in next week’s events related to DMD, please visit our member organization’s website:

We will be watching Tuesday and believe that growing patient participation in the drug development process is one more reason for our community to be thankful this year.