title: How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.
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we describe how the Duchenne muscular dystrophy community-led by Parent Project Muscular Dystrophy-created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.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other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents.
authors.affiliation: Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, Hackensack, NJ 07601, New Jersey. Pat@parentprojectmd.org.
authors.forename: Pat
authors.initials: P
authors.lastname: Furlong
authors.affiliation: Johns Hopkins Bloomberg School of Public Health, 624 N. Broadway, Rm 689, Baltimore, MD, 21205, USA. jbridge7@jhu.edu.
authors.forename: John F P
authors.initials: JF
authors.lastname: Bridges
authors.affiliation: Shire Plc, 300 Shire Way, Lexington, MA, 02421, USA. .lcharnas@shire.com.
authors.forename: Lawrence
authors.initials: L
authors.lastname: Charnas
authors.affiliation: Department of Neuroscience, Brown University, 185 Meeting Street, Box GL-N, Providence, RI, 02912, USA. justin_fallon@brown.edu.
authors.forename: Justin R
authors.initials: JR
authors.lastname: Fallon
authors.affiliation: Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, Hackensack, NJ 07601, New Jersey. ryan@parentprojectmd.org.
authors.forename: Ryan
authors.initials: R
authors.lastname: Fischer
authors.affiliation: Nationwide Children's Hospital, 700 N. Children's Drive, Columbus, OH, 43205, USA. Kevin.Flanigan@nationwidechildrens.org.
authors.forename: Kevin M
authors.initials: KM
authors.lastname: Flanigan
authors.affiliation: YourEncore, 111 Monument Circle-Suite 1022, Indianapolis, IN, 46204, USA. tim.franson@yourencore.com.
authors.forename: Timothy R
authors.initials: TR
authors.lastname: Franson
authors.affiliation: Suneel's Light Foundation, 5651 Main Street, Suite 8-152, Williamsville, New York, 14221, USA. npractice2@aol.com.
authors.forename: Neera
authors.initials: N
authors.lastname: Gulati
authors.affiliation: UC Davis NeuroNEXT, 4860 Y Street, Suite 3850, Sacramento, CA, 95817, USA. craig.mcdonald@ucdmc.ucdavis.edu.
authors.forename: Craig
authors.initials: C
authors.lastname: McDonald
authors.affiliation: Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, Hackensack, NJ 07601, New Jersey. holly@parentprojectmd.org.
authors.forename: Holly
authors.initials: H
authors.lastname: Peay
authors.affiliation: University of Pennsylvania Perelman School of Medicine, Richards Bldg., Rm. B400, 3700 Hamilton Walk, Philadelphia, PA, 19104, USA. lsweeney@mail.med.upenn.edu.
authors.forename: H Lee
authors.initials: HL
authors.lastname: Sweeney
mesh_terms: D020388:Muscular Dystrophy, Duchenne; D010344:Patient Advocacy; D014481:United States; D014486:United States Food and Drug Administration