A Pilot Experiment in Responding to Individual Patient Requests for Compassionate Use of an Unapproved Drug: The Compassionate Use Advisory Committee (CompAC). (March 2019)

• Record Type: Journal Article
• Authors: Caplan, Arthur; Bateman-House, Alison; Waldstreicher, Joanne; Fedor, Lisa; Sonty, Ramana; Roccia, Tito; Ukropec, Jon; Jansson, Rick
• Is Part Of: Therapeutic innovation & regulatory science. Volume 53:Number 2(2019:Mar.)
• Page Start: 243
• Page End: 248
• Publication Date: 2019-03
• Subjects: daratumumab -- preapproval access -- expanded access -- unapproved medications -- multiple myeloma; Drugs -- Periodicals; Pharmacology -- Periodicals; Drugs -- Research -- Periodicals; Drugs -- Testing -- Periodicals; 615.1
• DOI: 10.1177/2168479018759659
• Languages: English
• ISSNs: 2168-4790

Abstract

Background: Janssen Research & Development, LLC, part of the Janssen pharmaceutical companies of Johnson & Johnson, and NYU School of Medicine partnered to establish the Compassionate Use Advisory Committee (CompAC) to evaluate the use of an independent, external, expert committee in ensuring transparent, fair, beneficent, evidence-based, and patient-focused compassionate access to investigational medicines, a public health challenge that has been an ongoing issue for over 3 decades. Methods: To this end, NYU School of Medicine was responsible for the formation, member selection, and operation of CompAC, consisting of physicians, ethicists, and patient advocates, under Johnson & Johnson's sponsorship. Results: A pilot was successfully run using CompAC to provide recommendations on compassionate use access to a Johnson & Johnson oncology investigational asset called daratumumab. Conclusion: This innovative model provides a framework that can be emulated by the industry globally.