Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin in red blood cells that distorts them into a sickle shape. This sickling leads to various complications, including painful VOCs. While hydroxyurea—often in combination with prophylactic penicillin, analgesics, and/or blood transfusions—has been the standard of care for SCD, the FDA’s approval of Emmaus Life Sciences’ Endari (L-glutamine), Global Blood Therapeutics / Pfizer’s Oxbryta (voxelotor), and Novartis’s Adakveo (crizanlizumab) has provided patients with targeted treatment options. Moreover, gene therapies from Bluebird Bio (lovotibeglogene autotemcel [lovo-cel]) and Vertex / CRISPR Therapeutics (exagamglogene autotemcel [exa-cel]) are on the horizon, offering curative potential for SCD. Additionally, two emerging symptomatic therapies—inclacumab, a human MAb P-selectin inhibitor, and GBT021601, a next-generation hemoglobin S polymerization inhibitor, both from Global Blood Therapeutics / Pfizer—are likely to be efficacious targeted symptomatic treatments for SCD patients. This report explores the views and policies that physicians and payers have regarding approved SCD therapies and assesses hematologists’ expectations for and potential usage patterns of emerging gene and symptomatic therapies in the context of payers’ receptivity to and potential market access decisions regarding such treatments.
Geography: United States
Primary Research: Survey of 100 U.S. hematologists. Survey of 31 U.S. managed care organization (MCO) pharmacy and medical directors (PDs/MDs).
Key Drugs Covered: Adakveo (crizanlizumab), Endari (L-glutamine), Oxbryta (voxelotor), lovotibeglogene autotemcel (lovo-cel), exagamglogene autotemcel (exa-cel), inclacumab, GBT021601.
U.S. Access & Reimbursement provides integrated brand- and disease-level insight on reimbursement dynamics and the impact of U.S. payer policy on physician prescribing behavior in the market access environment. Each report includes up-to-date analyses of drug coverage and restriction policies as well as payer and prescriber perspectives on key marketed drugs and their receptivity to emerging therapies.