Hostname: page-component-848d4c4894-r5zm4 Total loading time: 0 Render date: 2024-06-30T00:19:09.220Z Has data issue: false hasContentIssue false
  • English
  • Français

High-Priced Sickle Cell Gene Therapies Threaten to Exacerbate US Health Disparities and Establish New Pricing Precedents for Molecular Medicine

Published online by Cambridge University Press:  27 July 2022

Frazer A. Tessema ,
Ameet Sarpatwari ,
Leah Z. Rand  and
Aaron S. Kesselheim
Frazer A. Tessema
Affiliation:
PROGRAM ON REGULATION, THERAPEUTICS, AND LAW (PORTAL), DIVISION OF PHARMACOEPIDEMIOLOGY AND PHARMACOECONOMICS, DEPARTMENT OF MEDICINE, BRIGHAM AND WOMEN’S HOSPITAL AND HARVARD MEDICAL SCHOOL, BOSTON, MA, USA
Ameet Sarpatwari
Affiliation:
PROGRAM ON REGULATION, THERAPEUTICS, AND LAW (PORTAL), DIVISION OF PHARMACOEPIDEMIOLOGY AND PHARMACOECONOMICS, DEPARTMENT OF MEDICINE, BRIGHAM AND WOMEN’S HOSPITAL AND HARVARD MEDICAL SCHOOL, BOSTON, MA, USA
Leah Z. Rand
Affiliation:
PROGRAM ON REGULATION, THERAPEUTICS, AND LAW (PORTAL), DIVISION OF PHARMACOEPIDEMIOLOGY AND PHARMACOECONOMICS, DEPARTMENT OF MEDICINE, BRIGHAM AND WOMEN’S HOSPITAL AND HARVARD MEDICAL SCHOOL, BOSTON, MA, USA
Aaron S. Kesselheim
Affiliation:
PROGRAM ON REGULATION, THERAPEUTICS, AND LAW (PORTAL), DIVISION OF PHARMACOEPIDEMIOLOGY AND PHARMACOECONOMICS, DEPARTMENT OF MEDICINE, BRIGHAM AND WOMEN’S HOSPITAL AND HARVARD MEDICAL SCHOOL, BOSTON, MA, USA
Get access Rights & Permissions [Opens in a new window]

Abstract

Gene therapies to treat sickle cell disease are in development and are expected to have high costs. The large eligible population size — by far, the largest for a gene therapy — poses daunting budget challenges and threatens to exacerbate health disparities for Black patients, who make up the vast majority of American sickle cell patients.

Keywords

Sickle Cell DiseaseGene TherapyHealth Care DisparitiesDrug CostsHealth Care Reform
Type
Columns: Health Policy Portal
Information
Journal of Law, Medicine & Ethics , Volume 50 , Issue 2: Commercial Speech and Commercial Determinants of Health , Summer 2022 , pp. 380 - 384
Copyright
© 2022 The Author(s)

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

Footnotes

About This Column

Aaron Kesselheim serves as the editor for Health Policy Portal. Dr. Kesselheim is the JLME editor-in-chief and director of the Program On Regulation, Therapeutics, And Law at Brigham and Women’s Hospital/Harvard Medical School. This column features timely analyses and perspectives on issues at the intersection of medicine, law, and health policy that are directly relevant to patient care. If you would like to submit to this section of JLME, please contact Dr. Kesselheim at akesselheim@bwh.harvard.edu.

References

G. Kolata, “Two New Drugs Help Relieve Sickle-Cell Disease. But Who Will Pay?” The New York Times, December 7, 2019, updated December 9, 2019; Howard, J. et al., “A Phase 1/2 Ascending Dose Study and Open-Label Extension Study of Voxelotor in Patients with Sickle Cell Disease,” Blood 133, no. 17 (2019): 1865–1875; “FDA Approves Oxbryta (Voxelotor), the First Medicine Specifically Targeting the Root Cause of Sickle Cell Disease,” GlobeNewswire, November 25, 2019.Google Scholar
Abraham, A.A. and Tisdale, J.F., “Gene Therapy for Sickle Cell Disease: Moving from the Bench to the Bedside,” Blood 138, no. 11 (2021): 932–941.CrossRefGoogle Scholar
Id., O. Negre et al., “Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of Beta-Thalassemia and Sickle Cell Disease,” Current Gene Therapy 15, no. 1 (2015): 64–81.Google Scholar
Kolata, supra note 1; Cassidy, W., “How Will We Pay for the Coming Generation of Potentially Curative Gene Therapies?” STAT, June 12, 2019; K. Jensen, “Bluebird Proposes Installment Plan for LentiGlobin Gene Therapy,” Biopharma Dive, January 9, 2019.Google Scholar
Herman, B., “New Gene Therapy has $1.8 Million Price Tag,” Axios, 2019. June 14, 2019.Google Scholar
Cassidy, supra note 4; Herman, supra note 5; Kauf, T.L., Coates, T.D., Huazhi, L., Mody-Patel, N., and Hartzema, A.G., “The Cost of Health Care for Children and Adults with Sickle Cell Disease,” American Journal of Hematology 84, no. 6 (2009): 323–327.CrossRefGoogle ScholarPubMed
U.S. Centers for Disease Control and Prevention, “Data & Statistics on Sickle Cell Disease,” (2020).Google Scholar
“The Rise of Orphan Drugs,” AHIP (2019).Google Scholar
Id.Google Scholar
“The Price of Sovaldi And Its Impact On The U.S. Health Care System,” Committee on Finance: United States Senate (Washington, DC, 2015).Google Scholar
Henry, B., “Drug Pricing & Challenges to Hepatitis C Treatment Access,” Journal of Health and Biomedical Law 14 (2018): 265–283; J.M. Liao and M.A. Fischer, “Restrictions of Hepatitis C Treatment for Substance-Using Medicaid Patients: Cost Versus Ethics,” American Journal of Public Health 107 (2017): 893-899; A.L. Beckman et al., “New Hepatitis C Drugs Are Very Costly And Unavailable To Many State Prisoners,” Health Affairs (Millwood) 35, no. 10 (2016): 1893-1901.Google ScholarPubMed
Supra note 10.Google Scholar
Data & Statistics on Sickle Cell Disease, supra note 7; Reeves, S.L. et al., “Incidence, Demographic Characteristics, and Geographic Distribution of Sickle Cell Trait and Sickle Cell Anemia Births in Michigan, 1997-2014,” Molecular Genetic & Genomic Medicine 7, no. 8 (2019): e795.CrossRefGoogle ScholarPubMed
Data & Statistics on Sickle Cell Disease, supra note 7; Naik, R.P., et al., “Clinical Outcomes Associated With Sickle Cell Trait: A Systematic Review,” Annals of Intern Medicine 169, no. 9 (2018): 619–627.CrossRefGoogle ScholarPubMed
Herman, supra note 5; Kaltenboeck, A.and Bach, P.B., “Value-Based Pricing for Drugs: Theme and Variations,” JAMA 319, no. 21 (2018): 2165–2166; R. Knox, “New Cancer Treatments Top $500,000 AND Raise Daunting Questions About How To Pay,” WBUR, 2018, January 11, 2018.CrossRefGoogle ScholarPubMed
Lubeck, D. et al., “Estimated Life Expectancy and Income of Patients With Sickle Cell Disease Compared With Those Without Sickle Cell Disease,” JAMA Network Open 2 (2019): e1915374; F. Farooq, P.J. Mogayzel, S. Lanzkron, C. Haywood, and J.J. Strouse, “Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity,” JAMA Network Open 3 (2020): e201737; J. Cohen, “Sickle Cell Disease: After Years Of Neglect Some Promise For Sufferers,” Forbes February 17, 2020.Google ScholarPubMed
National Institutes of Health, “NIH Researchers Create New Viral Vector for Improved Gene Therapy in Sickle Cell Disease,” NIH, 2019; Uchida, N. et al., “Development of a Forward-Oriented Therapeutic Lentiviral Vector for Hemoglobin Disorders,” Nature Communications 10, no. 1 (2019): 4479.CrossRefGoogle ScholarPubMed
National Institutes of Health, “NIH Launches New Collaboration to Develop Gene-Based Cures for Sickle Cell Disease and HIV on Global Scale,” NIH, 2019.Google Scholar
Samazan, T., “Government Funding of CRISPR Research and Policy Changes,” Instrument Business Outlook, January 4, 2019; Doudna, J. and Marson, A., “Federal Funding for Basic Research Led to the Gene-Editing Revolution. Don’t Cut It.” Vox, April 22, 2017.Google Scholar
Bluebird bio, “Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy,” Clinicaltrials.gov, U.S. National Library of Medicine, 2020, Bluebird bio, “A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in Severe Sickle Cell Disease,” Clinicaltrials.gov, U.S. National Library of Medicine, 2020; Thompson, A.A. et al., “Gene Therapy in Patients with Transfusion-Dependent beta-Thalassemia,” New England Journal of Medicine 378, no. 16 (2018): 1479–1493.CrossRefGoogle ScholarPubMed
Artiga, S., Orgera, K., and Damico, A., “Changes in Health Coverage by Race and Ethnicity Since the ACA, 2010-2018,” Kaiser Family Foundation, 2020; E. Berchick, E. Hood, and J. Barnett, Health Insurance Coverage in the United States: 2017, Current Population Reports, Washington, DC: U.S. Census Bureau, September 2018, Report No.: P60-264; E. Berchick, J. Barnett, and R. Upton, Health Insurance Coverage in the United States: 2018, Current Population Reports, Washington, DC: U.S. Department of Commerce, November 2019, Report No.: P60-267.Google Scholar
Essien, U.R., Dusetzina, S.B., and Gellad, W.F., “A Policy Prescription for Reducing Health Disparities-Achieving Pharmacoequity,” JAMA 326, no. 18 (2021).CrossRefGoogle ScholarPubMed
Wilson-Frederick, S., Hulihan, M., and Anderson, K., “Prevalence of Sickle Cell Disease among Medicaid Beneficiaries in 2012,” Data Highlight, Washington, DC: Centers For Medicare & Medicaid Services (CMS), Office of Minority Health; June 2019, Data Highlight No.16.Google Scholar
Bruen, B., Brantley, E., Thompson, V., Steinmetz, E., and Helmchen, L., High-Cost Hepatitis C Drugs in Medicaid: Final Report . Medicaid and CHIP Payment and Access Commission (MACPAC), Washington, DC: George Washington University; January 2017, Report for MACPAC Contract # MACP16406T2.Google Scholar
State of Illinois Budget Summary, Fiscal Year 2021, Commission On Government Forecasting & Accountability; Illinois General Assembly.Google Scholar
Sickle Cell Program, University of Illinois – Chicago. Department of Medicine, Division of Hematology and Oncology, 2021.Google Scholar
Cassidy, supra note 4.Google Scholar
Cassidy, supra note 4; M.R. Trusheim, W. M. Cassidy, and Bach, P.B., “Alternative State-Level Financing for Hepatitis C Treatment-The ‘Netflix Model,’” JAMA 320, no. 19 (2018): 1977–1978.Google ScholarPubMed
Cassidy, supra note 4.Google Scholar
Cassidy, supra note 4.Google Scholar
Sarpatwari, J. Avorn, and Kesselheim, A.S., “Accounting for US Public Funding in Drug Development: How Can We Better Balance Access, Affordability, and Innovation?” BMJ 371 (2020): m3841 (2020); G.C. Alexander et al., “Reducing Branded Prescription Drug Prices: A Review of Policy Options,” Pharmacotherapy 37, no. 11 (2017): 1469-1478.Google ScholarPubMed
Bradt, P. et al., Crizanlizumab, Voxelotor, and L-Glutamine for Sickle Cell Disease: Effectiveness and Value , Boston, MA: Institute For Clinical And Economic Review (ICER); March 2020 (updated Feb. 2021), Evidence Report – Crizanlizumab, Voxelotor, and L-Glutamine for SCD.Google Scholar