How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration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, John F P Bridges, Lawrence Charnas, Justin R. Fallon, Ryan Fischer, Kevin M. Flanigan, Timothy R. Franson, Neera Gulati, Craig M McDonald, Holly Peay, H. Lee Sweeney

Research output: Contribution to journalArticle

22 Scopus citations

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we describe how the Duchenne muscular dystrophy community-led by Parent Project Muscular Dystrophy-created a proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.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other rare disease communities and advocacy organizations can use our experience as a model for developing their own draft guidance documents.

Original languageEnglish (US)
Article number281
JournalOrphanet Journal of Rare Diseases
Volume10
Issue number1
DOIs
StatePublished - Jun 24 2015

Keywords

  • Advocacy
  • Clinical trial
  • Drug development
  • Duchenne muscular dystrophy
  • FDA
  • Industry guidance
  • Patient engagement
  • Public policy
  • Rare disease

ASJC Scopus subject areas

  • Medicine(all)
  • Genetics(clinical)
  • Pharmacology (medical)

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    Furlong, P., Bridges, J. F. P., Charnas, L., Fallon, J. R., Fischer, R., Flanigan, K. M., Franson, T. R., Gulati, N., McDonald, C. M., Peay, H., & Sweeney, H. L. (2015). How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.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, 10(1), [281]. https://doi.org/10.1186/s13023-015-0281-2