How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

Orphanet Journal of Rare Diseases

H. Lee Sweeney⁵  Holly Peay⁹  Craig McDonald²  Neera Gulati³  Timothy R. Franson⁶  Kevin M. Flanigan⁷  Ryan Fischer⁹  Justin R. Fallon⁴  Lawrence Charnas¹  John F. P. Bridges⁸  Pat Furlong⁹ 

[1] Shire Plc, 300 Shire Way, Lexington 02421, MA, USA;UC Davis NeuroNEXT, 4860 Y Street, Suite 3850, Sacramento 95817, CA, USA;Suneel’s Light Foundation, 5651 Main Street, Suite 8-152, Williamsville, New York 14221, USA;Department of Neuroscience, Brown University, 185 Meeting Street, Box GL-N, Providence 02912, RI, USA;University of Pennsylvania Perelman School of Medicine, Richards Bldg., Rm. B400, 3700 Hamilton Walk, Philadelphia 19104, PA, USA;YourEncore, 111 Monument Circle–Suite 1022, Indianapolis 46204, IN, USA;Nationwide Children’s Hospital, 700 N. Children’s Drive, Columbus 43205, OH, USA;Johns Hopkins Bloomberg School of Public Health, 624 N. Broadway, Rm 689, Baltimore 21205, MD, USA;Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, Hackensack NJ 07601, New Jersey

关键词: Drug development;    Patient engagement;    Clinical trial;    Rare disease;    Advocacy;    Public policy;    Duchenne muscular dystrophy;    Industry guidance;    FDA;   

Others  :  1219116
DOI  :  10.1186/s13023-015-0281-2

 received in 2015-02-26, accepted in 2015-04-30,  发布年份 2015

【 摘 要 】

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we describe how the Duchenne muscular dystrophy community–led by Parent Project Muscular Dystrophy–created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.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other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents.

【 授权许可】

   
2015 Furlong et al.

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