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. 2015 Jun 24:10:82.
doi: 10.1186/s13023-015-0281-2.

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

Pat Furlong  1 , John F P Bridges  2 , Lawrence Charnas  3 , Justin R Fallon  4 , Ryan Fischer  5 , Kevin M Flanigan  6 , Timothy R Franson  7 , Neera Gulati  8 , Craig McDonald  9 , Holly Peay  10 , H Lee Sweeney  11
Affiliations

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

Pat Furlong et al. Orphanet J Rare Dis. .

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we describe how the Duchenne muscular dystrophy community-led by Parent Project Muscular Dystrophy-created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.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other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents.

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Figures

Fig. 1
Fig. 1
Draft guidance project timeline
Fig. 2
Fig. 2
Entities involved in Developing the draft industry guidance
Fig. 3
Fig. 3
Process for developing individual draft guidance document sections and master document
See this image and copyright information in PMC

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