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

Author:

Salzman Rachel,Cook Francesca,Hunt Timothy,Malech Harry L.,Reilly Philip,Foss-Campbell Betsy,Barrett David

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference50 articles.

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/CellularGeneTherapyProducts/ApprovedProducts/UCM574106.pdf.

2. 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/CellularGeneTherapyProducts/ApprovedProducts/UCM581259.pdf.

3. 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/CellularGeneTherapyProducts/ApprovedProducts/UCM589690.pdf.

4. 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_2017F211v011.pdf.

5. 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/?cookieSet=1.

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