Important Considerations in Modeling the Cost-Effectiveness for the First Food and Drug Administration–Approved Gene Therapy and Implications for Future One-Time Therapies

Aug 1, 2019, 00:00
10.1016/j.jval.2018.12.013
https://www.valueinhealthjournal.com/article/S1098-3015(19)30178-0/fulltext
Title : Important Considerations in Modeling the Cost-Effectiveness for the First Food and Drug Administration–Approved Gene Therapy and Implications for Future One-Time Therapies
Citation : https://www.valueinhealthjournal.com/action/showCitFormats?pii=S1098-3015(19)30178-0&doi=10.1016/j.jval.2018.12.013
First page : 970
Section Title : CORRESPONDENCE
Open access? : No
Section Order : 970
To the Editor:

In their recent article titled “Cost utility of voretigene neparvovec for biallelic RPE65-mediated inherited retinal disease,” Zimmermann et al1 propose a cost-effectiveness model for the first gene therapy approved by the Food and Drug Administration (FDA) designed to treat an inherited genetic disorder. It is important to note that the publication consists of an abbreviated version of the analysis that the Institute for Clinical and Economic Review (ICER) released in February 2018, which culminated in a favorable review committee vote for voretigene neparvovec-rzyl (VN) of 9-3 of high/intermediate versus low long-term value for money. The committee’s vote was informed by ICER’s sensitivity analyses of their base-case model, which demonstrated that VN may be cost-effective when using appropriate health utilities, indirect costs that address the high societal impact of blindness, and/or a lifetime treatment effect. Nonetheless, Zimmermann et al conclude that “[a]t the current price, VN was unlikely to reach traditional cost-effectiveness standards compared with SoC [standard of care]."

Categories :
  • Cost-comparison, Effectiveness, Utility, Benefit Analysis
  • Genetic, Regenerative & Curative Therapies
  • Personalized & Precision Medicine
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