Foundation and ClinicalTrials.gov Extol Virtues of Expanded Access for Patients

Reagan-Udall Foundation for the FDA

Public-private partnerships have the flexibility to increase patient equity in accessing investigational therapies and to create a neutral clearinghouse for educational and technical resources, says June Wasser, MA, executive director of the Reagan-Udall Foundation (RUF) for the Food and Drug Administration (FDA).

RUF was created by Congress in 2007 as an independent organization designed in part to support and promote regulatory science to advance the FDA’s mission. It also serves as a neutral third party to coordinate public-private partnerships with FDA and other stakeholders, Wasser told attendees of a webinar on “Pre-approval Access Patient Advocacy” on June 6 that was sponsored by Johnson & Johnson and NYU Langone Health.

RUF offers an Expanded Access Navigator tool serving as a source of information on the process physicians use to request expanded access to investigational therapies under development by biopharmaceutical companies. It also includes a directory for patients and physicians to find the expanded access policies and clinical trial record numbers of participating companies.

Rebecca J. Williams, PharmD, MPH, assistant director for ClinicalTrials.gov, a resource of the National Institutes of Health/U.S. National Library of Medicine, stressed the importance of easy access to such clinical trial information by members of the public and research community. Benefits include:

  • Meeting ethical obligations to human subjects (e.g., seeing that results will be used to help others)
  • Informing future research and research funding decisions
  • Mitigating information bias (e.g., non-publication of results deemed unfavorable)
  • Evaluating research integrity (e.g., adherence to protocol)
  • Preventing duplication of trials of unsafe or ineffective interventions
  • Providing access to data to support evidence-based medicine
  • Enhancing patient access to enrollment in clinical trials

Williams shared data as of May 4 showing that ClinicalTrials.gov has posted 389 expanded access records with a drug, biological, or genetic intervention. About half were U.S. only, while another 30% had no geographical information. The records included 228 unique sponsor names, 404 unique intervention names, and 452 unique condition names.

Author: Michael Causey