A survey of pediatric hematologists/oncologists’ perspectives on single patient Expanded Access and Right to Try

Abstract

Declaration of conflicting interests: The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: A.B.-H. serves as an unpaid chair and an unpaid committee member for the NYU Compassionate Use Advisory Committees (CompAC), external panels of medical experts, bioethicists, and patient representatives formed by NYU Grossman School of Medicine in collaboration with Janssen Pharmaceuticals to advise Janssen about requests for compassionate use of its investigational medicines. The university receives administrative funding from Janssen to facilitate the CompAC program. At the time of this study, she was an associate fellow of the GE2P2 Global Foundation and a member of its Independent Bioethics Advisory Committee (IBAC), which provide bioethical consultative services to commercial and other biopharma organizations on clinical trials, expanded access programs for investigational medicines and therapies, and in other areas. She has accepted speaking fees and/or travel expenses from the American Association for Cancer Research; Amyloidosis Research Consortium; Biogen; European Organization for the Research and Treatment of Cancer; Johnson & Johnson; the Reagan-Udall Foundation for the FDA; and the US FDA. She receives payment for work on data safety monitoring committees, one for a clinical trial sponsored by the National Eye Institute and one run by NYU Langone Health. She has served as an unpaid advisor to numerous pharmaceutical and biotech companies, patient advocacy groups, and health policy organizations with regard to pre-approval access to investigational medical products. She was an unpaid member of the advisory board for Kids V Cancer’s Compassionate Use Navigator. C.R.C. served as the deputy chair of the CompAC Infectious Disease committee from August 2017 through August 2019. C.R.C.’s postdoctoral fellowship salary was partially supported by Janssen’s administrative funding. The Division of Medical Ethics also has a grant from WIRB/WCG to provide ethics education as part of an international research ethics fellows program. C.R.C. is paid for coordinating the NYU component of this program. She has also provided paid advisory services to GE2P2 Advisory Services pbc/GE2P2 Global Foundation. The remaining authors report no conflicts of interest. D.L. reports being a member of the National Cancer Institute’s National Council of Research Advocates, Co-Chair of NCI’s Patient Advocate Steering Committee as well as a member of the Pediatric and Adolescent Solid Tumor Steering Committee.