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Open Access 01-12-2015 | Review

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration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

Published in: Orphanet Journal of Rare Diseases | Issue 1/2015

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we describe how the Duchenne muscular dystrophy community–led by Parent Project Muscular Dystrophy–created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.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other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents.

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Title: How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration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: 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
Publication date: 01-12-2015
Publisher: BioMed Central
Published in: Orphanet Journal of Rare Diseases / Issue 1/2015
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/s13023-015-0281-2

At a glance: The STEP trials

Springer Medicine

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

Abstract

At a glance: The STEP trials

Springer Medicine

Abstract

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