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
. 2021 Feb;27(2):276-280.
doi: 10.18553/jmcp.2021.27.2.276.

The effectiveness and value of novel treatments for cystic fibrosis

Affiliations

The effectiveness and value of novel treatments for cystic fibrosis

Jeffrey A Tice et al. J Manag Care Spec Pharm. 2021 Feb.

Abstract

DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, California Health Care Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, America's Health Insurance Plans, Anthem, Allergan, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare. Seidner, Rind, and Pearson are employed by ICER. Tice reports contracts to his institution, University of California, San Francisco, from ICER during the conduct of this study. Wherry has nothing to disclose.

PubMed Disclaimer

Conflict of interest statement

Funding for this summary was contributed by Arnold Ventures, California Health Care Foundation, Harvard Pilgrim Health Care, and Kaiser Foundation Health Plan to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER’s annual policy summit is supported by dues from Aetna, America’s Health Insurance Plans, Anthem, Allergan, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Boehringer-Ingelheim, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, HealthFirst, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Pfizer, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare.

Seidner, Rind, and Pearson are employed by ICER. Tice reports contracts to his institution, University of California, San Francisco, from ICER during the conduct of this study. Wherry has nothing to disclose.

Figures

FIGURE 1
FIGURE 1
Network Meta-Analysis Results for ppFEV1 Comparing CF Modulator Therapy to Placebo

References

    1. Cystic Fibrosis Foundation. Cystic Fibrosis Foundation Patient Registry 2018 Annual Data Report. 2019. Accessed January 9, 2021. https://www.cff.org/Research/Researcher-Resources/Patient-Registry/2018-...
    1. Patel S, Sinha IP, Dwan K, Echevarria C, Schechter M, Southern KW. Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis. Cochrane Database Syst Rev. 2015(3):CD009841. - PubMed
    1. Agrawal A, Mehta D, Sikachi R, Du D, Wang J. Nationwide trends of hospitalizations for cystic fibrosis in the United States from 2003 to 2013. Intractable Rare Dis Res. 2017;6(3):191-98. - PMC - PubMed
    1. Heijerman H, McKone E, Downey D, et al. . Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blink, randomised, phase 3 trial. Lancet. 2019;394(10212):1940-48. - PMC - PubMed
    1. Keating D, Marigowda G, Burr L, et al. . VX-445-Tezacaftor-ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med. 2018;379(17):1612-20. - PMC - PubMed

MeSH terms