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BioMed Central, Orphanet Journal of Rare Diseases, 1(10), 2015

DOI: 10.1186/s13023-015-0281-2

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How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

Journal article published in 2015 by Pat Furlong, John F. P. Bridges, Lawrence Charnas, Justin R. Fallon, Ryan Fischer, Kevin M. Flanigan, Timothy R. Franson, Neera Gulati, Craig McDonald ORCID, H. Lee Sweeney, Holly Peay, H. Lee Sweeney
This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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we describe how the Duchenne muscular dystrophy community–led by Parent Project Muscular Dystrophy–created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.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other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents.