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
. 2019 Jan;25(1):10-15.
doi: 10.18553/jmcp.2019.25.1.010.

The Effectiveness and Value of Patisiran and Inotersen for Hereditary Transthyretin Amyloidosis

Kristin Mickle  1 , Karen E Lasser  2 , Jeffrey S Hoch  3 , Lauren E Cipriano  4 , William B Dreitlein  1 , Steven D Pearson  1
Affiliations
Review

The Effectiveness and Value of Patisiran and Inotersen for Hereditary Transthyretin Amyloidosis

Kristin Mickle et al. J Manag Care Spec Pharm. 2019 Jan.

Abstract

Funding for this summary was contributed by the Laura and John Arnold Foundation, Blue Shield of California, and California Health Care Foundation 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, AHIP, Anthem, Blue Shield of California, CVS Caremark, Express Scripts, Harvard Pilgrim Health Care, Cambia Health Solutions, United Healthcare, Kaiser Permanente, Premera Blue Cross, AstraZeneca, Genentech, GlaxoSmithKline, Johnson & Johnson, Merck, National Pharmaceutical Council, Prime Therapeutics, Sanofi, Spark Therapeutics, Health Care Service Corporation, Editas, Alnylam, Regeneron, Mallinkrodt, Biogen, HealthPartners, and Novartis. Mickle, Dreitlein, and Pearson are ICER employees. Lasser, Cipriano, and Hoch have nothing to disclose.

PubMed Disclaimer

Conflict of interest statement

Funding for this summary was contributed by the Laura and John Arnold Foundation, Blue Shield of California, and California Health Care Foundation 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, AHIP, Anthem, Blue Shield of California, CVS Caremark, Express Scripts, Harvard Pilgrim Health Care, Cambia Health Solutions, United Healthcare, Kaiser Permanente, Premera Blue Cross, AstraZeneca, Genentech, GlaxoSmithKline, Johnson & Johnson, Merck, National Pharmaceutical Council, Prime Therapeutics, Sanofi, Spark Therapeutics, Health Care Service Corporation, Editas, Alnylam, Regeneron, Mallinkrodt, Biogen, HealthPartners, and Novartis. Mickle, Dreitlein, and Pearson are ICER employees. Lasser, Cipriano, and Hoch have nothing to disclose.

Figures

FIGURE 1
FIGURE 1
The ICER Value Assessment Framework
See this image and copyright information in PMC

References

    1. Gertz MA. Hereditary ATTR amyloidosis: burden of illness and diagnostic challenges. Am J Manag Care. 2017;23(7 Suppl):S107-S112. - PubMed
    1. Hawkins PN, Ando Y, Dispenzeri A, Gonzalez-Duarte A, Adams D, Suhr OB. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015;47(8):625-38. - PMC - PubMed
    1. Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. . Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. New Engl J Med. 2018;379(1):11-21. - PubMed
    1. Quarta CC, Buxbaum JN, Shah AM, et al. . The amyloidogenic V122I transthyretin variant in elderly black Americans. New Engl J Med. 2015;372(1):21-29. - PMC - PubMed
    1. Lasser K, Hoch JS, Mickle K, et al. . Inotersen and patisiran for hereditary transthyretin amyloidosis: effectiveness and value. Final evidence report. Institute for Clinical and Economic Review. October 4, 2018. Available at: https://icer-review.org/wp-content/uploads/2018/02/ICER_Amyloidosis_Fina.... Accessed December 4, 2018.

MeSH terms

Supplementary concepts