What compassionate use means for gene therapies

Author:

Chapman Carolyn RileyORCID,Moch Kenneth I.,McFadyen Andrew,Kearns Lisa,Watson Tom,Furlong Pat,Bateman-House Alison

Publisher

Springer Science and Business Media LLC

Subject

Biomedical Engineering,Molecular Medicine,Applied Microbiology and Biotechnology,Bioengineering,Biotechnology

Reference26 articles.

1. 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 Long term follow-up after administration of human gene therapy products: draft guidance for industry.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).

2. 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).

3. U.S. Food and Drug Administration. Expanded access. https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm (25 August 2017).

4. 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).

5. Borysowski, J., Ehni, H. & Górski, A. BMC Med. 15, 136 (2017).

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