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- Title
How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.
- Authors
Furlong, Pat; Bridges, John F. P.; Charnas, Lawrence; Fallon, Justin R.; Fischer, Ryan; Flanigan, Kevin M.; Franson, Timothy R.; Gulati, Neera; McDonald, Craig; Peay, Holly; Lee Sweeney, H.
- Abstract
Among the challenges confronting patients with rare diseases is a dearth of treatment options. The development of safe and effective new therapies is hampered by challenges associated with conducting clinical trials in small populations. In this article, we describe how the Duchenne muscular dystrophy community-led by Parent Project Muscular Dystrophy-created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration. This unprecedented undertaking involved a broad coalition of more than 80 stakeholders collaborating across nine time zones to produce a document in only 6 months. We hope that other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents.
- Subjects
DUCHENNE muscular dystrophy; PATIENT advocacy; UNITED States. Food &; Drug Administration; PRESSURE groups; CLINICAL trials; DRUG development
- Publication
Orphanet Journal of Rare Diseases, 2015, Vol 10, Issue 1, p1
- ISSN
1750-1172
- Publication type
Article
- DOI
10.1186/s13023-015-0281-2