This is a preview of subscription content, access via your institution
Relevant articles
Open Access articles citing this article.
-
Results from Expanded Access Programs: A Review of Academic Literature
Drugs Open Access 18 May 2023
-
Regulating clinical trials in a resource-limited setting during the Ebola public health emergency in Sierra Leone
Trials Open Access 06 June 2022
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$29.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Rent or buy this article
Prices vary by article type
from$1.95
to$39.95
Prices may be subject to local taxes which are calculated during checkout
References
U.S. Department of Department of Health and Human Services, Food and Drug Administration, Center for Biologics Evaluation and Research. Long term follow-up after administration of human gene therapy products: draft guidance for industry. https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-bio-gen/documents/document/ucm610797.pdf (2018).
MIT NEWDIGS Initiative. FoCUS Project: financing of cures in the US. Research Brief 2017F211.v011. https://newdigs.mit.edu/sites/default/files/FoCUS_Research_Brief_2017F211v011.pdf (2017).
U.S. Food and Drug Administration. Expanded access. https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm (25 August 2017).
Caplan, A. & Moch, K. Rescue me: the challenge of compassionate use in the social media era. Health Affairs Blog https://www.healthaffairs.org/do/10.1377/hblog20140827.041027/full/ (2014).
Borysowski, J., Ehni, H. & Górski, A. BMC Med. 15, 136 (2017).
Caplan, A. L. et al. J. Med. Ethics 44, 761–767 (2018).
Miller, J. E. et al. BMJ Open 7, e017917 (2017).
ASCO. ASCO and Friends of Cancer Research release comprehensive recommendations to broaden eligibility criteria for cancer clinical trials. https://www.asco.org/advocacy-policy/asco-in-action/asco-and-friends-cancer-research-release-comprehensive (2017).
Department of Health and Human Services, National Institutes of Health, Recombinant DNA Advisory Committee. Minutes of meeting, January 14, 1993. https://osp.od.nih.gov/wp-content/uploads/2014/03/193rac.pdf (1993).
Qasim, W. et al. Sci. Transl. Med. 9, eaaj2013 (2017).
Brau, R. & Tzeng, I. Orphan drug commercial models. Life Science Leader https://www.lifescienceleader.com/doc/orphan-drug-commercial-models-0001 (16 March 2018).
Feuerstein, A. Here come the right-to-try profiteers. The FDA is powerless to stop them. STAT News https://www.statnews.com/2018/06/20/right-to-try-opportunism/ (20 June 2018).
Kim, T. Goldman Sachs asks in biotech research report: ‘Is curing patients a sustainable business model?’ CNBC https://www.cnbc.com/2018/04/11/goldman-asks-is-curing-patients-a-sustainable-business-model.html (11 April 2018).
Mullin, E. A year after approval, gene-therapy cure gets its first customer. MIT Technology Review https://www.technologyreview.com/s/604295/a-year-after-approval-gene-therapy-cure-gets-its-first-customer/ (3 May 2017).
Kimmelman, J. Gene Therapy of Cancer: Methods and Protocols (eds. Walther, W. & Stein, U. S.) 423–445 (Humana, Totowa, NJ, USA, 2009).
Usdin, S. Beyond compassion: the case for using expanded access protocols to generate real world data. BioCentury https://www.biocentury.com/biocentury/regulation/2017-09-29/case-using-expanded-access-protocols-generate-real-world-data (29 September 2017).
Sutter, S. Expanded access programs eyed for data-gathering purposes. Pink Sheet Pharma Intelligence https://pink.pharmaintelligence.informa.com/PS122926/Expanded-Access-Programs-Eyed-For-DataGathering-Purposes (18 April 2018).
Rawson, K. Expanded access data can support approval decisions, US FDA says. Pink Sheet Pharma Intelligence https://pink.pharmaintelligence.informa.com/PS124296/Expanded-Access-Data-Can-Support-Approval-Decisions-US-FDA-Says (21 November 2018).
Cicalese, M. P. et al. Mol. Ther. 26, 917–931 (2018).
Department of Health and Human Services, NIH. Fed. Regist. 83, 41082–41093; https://www.federalregister.gov/documents/2018/08/17/2018-17760/national-institutes-of-health-nih-office-of-science-policy-osp-recombinant-or-synthetic-nucleic-acid (2018).
Jenkins, C. et al. Mol. Ther 24, 1173–1177 (2016).
Hitchcock, T. Manufacturing of AAV vectors for gene therapy. Genetic Engineering & Biotechnology News https://www.genengnews.com/gen-articles/manufacturing-of-aav-vectors-for-gene-therapy/6095 (1 July 2017).
Mullin, E. Gene therapy could make cancer care more unequal, and this map shows why. MIT Technology Review https://www.technologyreview.com/s/609890/gene-therapy-could-make-cancer-care-more-unequal-and-this-map-shows-why/ (12 January 2018).
Moch, K.I., McFadyen, A. & Caplan, A. Here’s how to structure successful right-to-try laws. The Hill http://thehill.com/opinion/healthcare/377794-heres-how-to-structure-successful-right-to-try-laws (11 March 2018).
Cortez, M. Gene therapies that could transform diseases get easier FDA path. Bloomberg https://www.bloomberg.com/news/articles/2018-05-22/gene-therapies-that-could-transform-diseases-get-easier-fda-path (22 May 2018).
Chapman, C.R. Is it time for the FDA to consider a differentiated approval system? Health Affairs Blog https://www.healthaffairs.org/do/10.1377/hblog20171129.293917/full/ (2017).
Acknowledgements
We thank A. Hall for assisting with research on companies developing gene therapies and A. L. Caplan for comments on a draft.
Author information
Authors and Affiliations
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
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.
Rights and permissions
About this article
Cite this article
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
Published:
Issue Date:
DOI: https://doi.org/10.1038/s41587-019-0081-7