The Double-Edged Sword of Extremely High Prices for Gene Therapies in Sickle Cell Disease-Reference-Cited by-同舟云学术

The Double-Edged Sword of Extremely High Prices for Gene Therapies in Sickle Cell Disease

Published:2024-09-03 Issue:9 Volume:332 Page:703
ISSN:0098-7484
Container-title:JAMA
language:en
Short-container-title:JAMA

Author:

Cliff Edward R. Scheffer¹,Tessema Frazer A.¹

Affiliation:

1. Program On Regulation, Therapeutics, And Law, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital and Harvard Medical School, Boston, Massachusetts

Abstract

This Viewpoint discusses the high cost of new gene therapies for sickle cell disease, the challenges these costs pose for health care access, and new policy approaches to ensure fair reimbursement for payers and manufacturers without further increasing health care costs or barriers to access for underserved populations.

Publisher

American Medical Association (AMA)

Link

https://jamanetwork.com/journals/jama/articlepdf/2821828/jama_cliff_2024_vp_240076_1724442750.63526.pdf

Reference7 articles.

1. Comparison of US federal and foundation funding of research for sickle cell disease and cystic fibrosis and factors associated with research productivity.;Farooq;JAMA Netw Open,2020

2. Distributional cost-effectiveness of equity-enhancing gene therapy in sickle cell disease in the United States.;Goshua;Ann Intern Med,2023

3. US Food and Drug Administration. FDA approves first gene therapies to treat patients with sickle cell disease. Published December 8, 2023. Accessed June 4, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease

4. Mast? J. How sickle cell became the first disease treated by CRISPR. STAT News. Published December 4, 2023. Accessed June 3, 2024. https://www.statnews.com/2023/12/04/sickle-cell-crispr-exa-cel-casgevy-history/

5. Miller? M, Fields? R. Insurance executives refused to pay for the cancer treatment that could have saved him: this is how they did it. ProPublica. Published November 15, 2023. Accessed June 4, 2024. https://www.propublica.org/article/priority-health-michigan-cart-insurance-vanpatten-denials