An Ethical and Financial Obligation for Sickle Cell Disease Gene Therapy in the United States
Authors: Austin Wesevich, Monica E. Peek, Mark J. Ratain
Journal: Annals of Internal Medicine (2023)

Ideas and Opinions5 December 2023An Ethical and Financial Obligation for Sickle Cell Disease Gene Therapy in the United StatesAustin Wesevich, MD, MPH, Monica E. Peek, MD, MPH, MSc, and Mark J. Ratain, MDAustin Wesevich, MD, MPHSection of Hematology/Oncology, Department of Medicine, University of Chicago, Chicago, Illinois (A.W., M.J.R.), Monica E. Peek, MD, MPH, MScSection of General Internal Medicine, Department of Medicine, University of Chicago, Chicago, Illinois (M.E.P.)., and Mark J. Ratain, MDSection of Hematology/Oncology, Department of Medicine, University of Chicago, Chicago, Illinois (A.W., M.J.R.)Author, Article, and Disclosure Informationhttps://doi.org/10.7326/M23-2428 SectionsAboutFull TextPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareFacebookTwitterLinkedInRedditEmail New and life-saving therapy for patients with sickle cell disease (SCD) will soon be within reach. Vertex Pharmaceuticals (collaborating with CRISPR Therapeutics) and bluebird bio have each submitted biologic license applications to the U.S. Food and Drug Administration (FDA) for SCD gene therapy, and a rapid decision is expected. These therapies will join stem cell transplantation as the only curative options for adults with SCD.A report from the Institute for Clinical and Economic Review predicts that the price of SCD gene therapy will be $2 million per patient (1). Given that more than 50 000 patients with SCD may be ...References1. Beaudoin F, Thokala P, Nikitin D, et al. Gene Therapies for Sickle Cell Disease: Effectiveness and Value; Evidence Report. Institute for Clinical and Economic Review; 21 August 2023. Accessed at https://icer.org/wp-content/uploads/2023/08/ICER_SCD_Final_Report_FOR_PUBLICATION_082123.pdf on 5 September 2023. Google Scholar2. CVS Health. Gene Therapy Pipeline 3Q 2022–1Q 2027: Projected Treatments and Approval Timelines. Accessed at https://payorsolutions.cvshealth.com/sites/default/files/q3-2022-gene-therapy-treatments-approval-timelines.pdf on 9 June 2023. Google Scholar3. Bradt P, Spackman E, Synnott PG, et al. Crizanlizumab, Voxelotor, and L-Glutamine for Sickle Cell Disease: Effectiveness and Value. Institute for Clinical and Economic Review; 23 January 2020. Accessed at https://icer.org/wp-content/uploads/2021/02/ICER_SCD_Evidence-Report_031220-FOR-PUBLICATION.pdf on 15 July 2023. Google Scholar4. Sayed BA, Shaffer NC, Lewis SE, et al. Assessing Equity to Drive Health Care Improvements: Learnings from the CMS Innovation Center. Center for Medicare & Medicaid Innovation. Accessed at https://innovation.cms.gov/data-and-reports/2023/assessing-equity-hc-improv-wp on 1 September 2023. Google Scholar5. Grady A, Fiori A, Patel D, et al. Profile of Medicaid enrollees with sickle cell disease: a high need, high cost population. PLoS One. 2021;16:e0257796. [PMID: 34705847] doi:10.1371/journal.pone.0257796 CrossrefMedlineGoogle Scholar6. Farooq F, Mogayzel PJ, Lanzkron S, et al. Comparison of US federal and foundation funding of research for sickle cell disease and cystic fibrosis and factors associated with research productivity. JAMA Netw Open. 2020;3:e201737. [PMID: 32219405] doi:10.1001/jamanetworkopen.2020.1737 CrossrefMedlineGoogle Scholar7. Bahr NC, Song J. The effect of structural violence on patients with sickle cell disease. J Health Care Poor Underserved. 2015;26:648-661. [PMID: 26320901] doi:10.1353/hpu.2015.0094 CrossrefMedlineGoogle Scholar8. Nolen LT, Beckman AL, Sandoe E. How Foundational Moments in Medicaid's History Reinforced Rather Than Eliminated Racial Health Disparities. Health Affairs Forefront. 1 September 2020. [10.1377/forefront.20200828.661111] Google Scholar9. Brennan TA, Wilson JM. The special case of gene therapy pricing. Nat Biotechnol. 2014;32:874-876. [PMID: 25203033] doi:10.1038/nbt.3003 CrossrefMedlineGoogle Scholar10. Becerra X. A Report in Response to the Executive Order on Lowering Prescription Drug Costs for Americans. U.S. Department of Health and Human Services. Accessed at https://innovation.cms.gov/data-and-reports/2023/eo-rx-drug-cost-response-report on 1 September 2023. Google Scholar Author, Article, and Disclosure InformationAuthors: Austin Wesevich, MD, MPH; Monica E. Peek, MD, MPH, MSc; Mark J. Ratain, MDAffiliations: Section of Hematology/Oncology, Department of Medicine, University of Chicago, Chicago, Illinois (A.W., M.J.R.)Section of General Internal Medicine, Department of Medicine, University of Chicago, Chicago, Illinois (M.E.P.).Financial Support: By award T32CA009566 from the National Cancer Institute (Dr. Wesevich).Disclosures: Disclosures can be viewed at www.acponline.org/authors/icmje/ConflictOfInterestForms.do?msNum=M23-2428.Corresponding Author: Mark J. Ratain, MD, Section of Hematology/Oncology, Department of Medicine, University of Chicago, 5841 South Maryland Avenue, MC 2115, Chicago, IL 60637; e-mail, mratain@bsd.uchicago.edu.Author Contributions: Conception and design: A. Wesevich, M.J. Ratain.Analysis and interpretation of the data: A. Wesevich.Drafting of the article: A. Wesevich, M.E. Peek, M.J. Ratain.Critical revision for important intellectual content: A. Wesevich, M.E. Peek, M.J. Ratain.Final approval of the article: A. Wesevich, M.E. Peek, M.J. Ratain.Collection and assembly of data: A. Wesevich.This article was published at Annals.org on 5 December 2023. PreviousarticleNextarticle Advertisement FiguresReferencesRelatedDetails Metrics Current Issue January 2024Volume 177, Issue 1 Page: 85-86 Keywords Food and Drug Administration Gene therapy Genetic disorders Health disparities Health equity Medicaid Medicare Sickle cell disease ePublished: 5 December 2023 Issue Published: January 2024 Copyright & PermissionsCopyright © 2023 by American College of Physicians. All Rights Reserved.PDF downloadLoading ...