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What compassionate use means for gene therapies

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Acknowledgements

We thank A. Hall for assisting with research on companies developing gene therapies and A. L. Caplan for comments on a draft.

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Authors and Affiliations

Authors

Contributions

A.B.-H. conceived the idea for the paper. C.R.C., K.I.M., A.M., L.K., T.W., P.F. and A.B.-H. contributed to the outline and ideas for the project. C.R.C. drafted the paper. All authors worked collaboratively to edit and revise the paper.

Corresponding author

Correspondence to Carolyn Riley Chapman.

Ethics declarations

Competing interests

All authors are members of the NYU School of Medicine Working Group on Compassionate Use and Pre-approval Access (CUPA).

A.B.-H. serves as an unpaid chair and an unpaid committee member for the Compassionate Use Advisory Committees (CompAC), external panels of medical experts, bioethicists, and patient representatives formed by NYU 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. She is an associate fellow of the GE2P2 Global Foundation and a member of its Independent Bioethics Advisory Committee (IBAC). The IBAC provides bioethical consultative services to commercial and other biopharma organizations on clinical trials, on EA 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 and Reagan-Udall Foundation for the FDA. She receives payment for work on a data safety monitoring committee for a clinical trial sponsored by the US National Eye Institute. 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.

C.R.C. serves as the deputy chair of the CompAC Infectious Disease committee, part of CompAC (see above). NYU receives administrative funding from Janssen to facilitate CompAC; money goes to the Division of Medical Ethics to support committee infrastructure. C.R.C.’s salary is partially funded by this support. 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 the coordinator of the NYU component of this program. Her salary is partially funded by this support. She has also provided paid advisory services to GE2P2 Advisory Services pbc as an independent contractor.

P.F. is the founding president and CEO of Parent Project Muscular Dystrophy (PPMD). PPMD is a nonprofit organization focused solely on Duchenne muscular dystrophy, the most common fatal genetic childhood disorder. PPMD provides consulting services for protocol design, access and reimbursement for companies who have drug development programs focused on Duchenne muscular dystrophy. In addition, P.F. participates on the steering committee for Capricor Therapeutics and is a reviewer for the Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP).

L.K. serves as an unpaid deputy chair for CompAC (see above). She is an Associate Fellow of the GE2P2 Global Foundation and a member of its IBAC. The IBAC provides bioethical consultative services to commercial and other biopharma organizations on clinical trials, on EA programs for investigational medicines and therapies, and in other areas.

A.M. serves as the executive director of The Isaac Foundation, a patient advocacy organization that has provided research grants to researchers and projects studying gene therapy for Hunter syndrome and Maroteaux-Lamy syndrome. He is an associate fellow of the GE2P2 Global Foundation and a member of its IBAC. The IBAC provides bioethical consultative services to commercial and other biopharma organizations on clinical trials, on EA programs for investigational medicines and therapies, and in other areas. He is also a member of the board of Clinical Research Pathways, an organization that is committed to opening access to research and advancing treatments. He has been an expert witness to the US Congress on right-to-try legislation.

K.I.M. is president and CEO of Cognition Therapeutics, a developer of therapeutics for Alzheimer's disease and other neurocognitive disorders. He serves on the board of the Biotechnology Innovation Organization, where he chairs its Bioethics Committee and cochairs the Emerging Company Section’s Policy Subcommittee. He is a member of the boards of Zynerba Pharmaceuticals and Gamida Cell, Ltd. He has spoken widely on EA and RTT, and has testified before the US Congress on RTT legislation.

T.W. is executive vice president of Bionical Early Access Programs, a service provider for the pharmaceutical and biotech industry. The company specializes in providing consulting services on EA strategy, then designing and implementing global EA programs.

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Chapman, C.R., Moch, K.I., McFadyen, A. et al. What compassionate use means for gene therapies. Nat Biotechnol 37, 352–355 (2019). https://doi.org/10.1038/s41587-019-0081-7

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