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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
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

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.

Type
Columns: Health Policy Portal
Copyright
© 2022 The Author(s)

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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.

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