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. 2021 Apr 7;29(4):1367-1369.
doi: 10.1016/j.ymthe.2021.03.008. Epub 2021 Mar 13.

Gene therapy companies have an ethical obligation to develop expanded access policies

Lisa Kearns  1 , Carolyn Riley Chapman  2 , Kenneth I Moch  3 , Arthur L Caplan  2 , Tom Watson  4 , Andrew McFadyen  5 , Pat Furlong  6 , Alison Bateman-House  2
Affiliations

Gene therapy companies have an ethical obligation to develop expanded access policies

Lisa Kearns et al. Mol Ther. .
No abstract available

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Conflict of interest statement

L.K., A.L.C., A.M., P.F., and A.B.-H. are members of the Working Group on Pediatric Gene Therapy and Medical Ethics (PGTME) at the Division of Medical Ethics at NYU Grossman School of Medicine. PGTME is funded by a gift from Parent Project Muscular Dystrophy (PPMD), of which Pat Furlong is founder and president.

Comment in

References

    1. Chapman C.R., Moch K.I., McFadyen A., Kearns L., Watson T., Furlong P., Bateman-House A. What compassionate use means for gene therapies. Nat. Biotechnol. 2019;37:352–355. - PubMed
    1. Darrow J.J., Sarpatwari A., Avorn J., Kesselheim A.S. Practical, legal, and ethical issues in expanded access to investigational drugs. N. Engl. J. Med. 2015;372:279–286. - PubMed
    1. Usdin S. 2018. FDA to facilitate access to unapproved drugs. BioCentury, December 14, 2018.https://www.biocentury.com/article/299854/how-fda-plans-to-help-patients...
    1. 114th Congress . 2016. 21st Century Cures Act: Public Law 114–255—Dec. 13, 2016.https://www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf
    1. U.S. Food and Drug Administration . 2020. Expanded access.https://www.fda.gov/news-events/public-health-focus/expanded-access