Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2018 Dec 5;26(12):2717-2726.
doi: 10.1016/j.ymthe.2018.10.017. Epub 2018 Oct 30.

Addressing the Value of Gene Therapy and Enhancing Patient Access to Transformative Treatments

Rachel Salzman  1 , Francesca Cook  2 , Timothy Hunt  3 , Harry L Malech  4 , Philip Reilly  5 , Betsy Foss-Campbell  6 , David Barrett  7
Affiliations
Review

Addressing the Value of Gene Therapy and Enhancing Patient Access to Transformative Treatments

Rachel Salzman et al. Mol Ther. .

Abstract

Although high upfront costs for the high value of gene therapy have resulted in concerns about sufficient reimbursement to allow patient access to these therapies, the significant benefits of gene therapies will not be realized unless patients have access to them. Stakeholders are discussing these issues, and the payment models being developed for the newly approved gene therapies provide an early indication of the flexibility that will be needed from treatment manufacturers, payers, and policy makers to optimize patient access. Maximizing patient access to effective gene therapies is one integral part of the overall mission of the American Society of Gene and Cell Therapy, along with maximizing the quality of therapies and minimizing their costs.

Keywords: benefits; cost; coverage; patient access; payer; payment; payor; position; reimbursement; value.

PubMed Disclaimer

References

    1. U.S. Food and Drug Administration. (2017). BLA approval letter for tisagenlecleucal BL #1256046/0. August 30, 2017, STN: BL 125646/0. https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapy....
    1. U.S. Food and Drug Administration. (2017). BLA approval letter for axicabtagene ciloleucel BL #125643/0. October 18, 2017, STN: BL 125643/0. https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapy....
    1. U.S. Food and Drug Administration. (2017). BLA approval letter for voretigene neparvovec-rzyl BL #125610/0. December 19, 2017, STN BL 125610/0. https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapy....
    1. Massachusetts Institute of Technology New Drug Design Paradigms Initiative FoCUS Project. (2017). Existing gene therapy pipeline likely to yield dozens of approved products within five years. Research Brief 2017F211.v011. https://newdigs.mit.edu/sites/default/files/FoCUS_Research_Brief_2017F21....
    1. Daniel, G., Leschly, N., Marrazzo, J., and McClellan, M. (2017). Advancing gene therapies and curative health care through value-based payment reform. Health Affairs. https://www.healthaffairs.org/do/10.1377/hblog20171027.83602/full/?cooki.... - DOI