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How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration
|
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Published in |
Orphanet Journal of Rare Diseases, June 2015
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DOI | 10.1186/s13023-015-0281-2 |
Pubmed ID | |
Authors |
Pat Furlong, John F. P. Bridges, Lawrence Charnas, Justin R. Fallon, Ryan Fischer, Kevin M. Flanigan, Timothy R. Franson, Neera Gulati, Craig McDonald, Holly Peay, H. Lee Sweeney |
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we describe how the Duchenne muscular dystrophy community-led by Parent Project Muscular Dystrophy-created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.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other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
United Kingdom | 4 | 36% |
United States | 2 | 18% |
Greece | 1 | 9% |
Canada | 1 | 9% |
Unknown | 3 | 27% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 8 | 73% |
Science communicators (journalists, bloggers, editors) | 2 | 18% |
Scientists | 1 | 9% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 2 | 4% |
Unknown | 53 | 96% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 8 | 15% |
Student > Master | 6 | 11% |
Student > Bachelor | 6 | 11% |
Other | 4 | 7% |
Researcher | 3 | 5% |
Other | 10 | 18% |
Unknown | 18 | 33% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 11 | 20% |
Psychology | 4 | 7% |
Nursing and Health Professions | 4 | 7% |
Agricultural and Biological Sciences | 3 | 5% |
Biochemistry, Genetics and Molecular Biology | 3 | 5% |
Other | 9 | 16% |
Unknown | 21 | 38% |