Betibeglogne autotemcel (beti-cel, bluebird bio) is a potentially curative gene therapy for β-thalassemia that utilizes a lentiviral vector to insert a functioning version of the HBB gene into the patient’s own red blood cells. Patients must also receive myeloablative chemotherapy prior to
receiving beti-cel. The US Food and Drug Administration (FDA) accepted bluebird bio’s Biologics Licensing Application of beti-cel for priority review on November 22, 2021. The FDA Advisory Committee meeting was held in early June 2022, and the date by which a regulatory decision was due was set for August 19, 2022. The Institute for Clinical and Economic Review (ICER) conducted a systematic literature review and
cost-effectiveness analysis to evaluate the health and economic outcomes of beti-cel. Here we present the summary of our findings and highlight the policy discussion with key stakeholders held at a public meeting of the New England Comparative Effectiveness Advisory Council (New England CEPAC) on June 17, 2022.