Sickle Cell Disease – Access & Reimbursement – Access & Reimbursement – Sickle Cell Disease (US)

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, leading to various complications (e.g., painful vaso-occlusive crisis). While hydroxyurea has been the SOC for SCD, the FDA’s approval of Endari, Oxbryta, and Adakveo has given patients targeted treatment options. Moreover, new gene therapies Lyfgenia (Bluebird Bio) and Casgevy (Vertex / CRISPR Therapeutics) offer curative potential. Two emerging symptomatic therapies—inclacumab and GBT021601, both from Global Blood Therapeutics / Pfizer—are promising targeted SCD treatments. Further, Pyrukynd (mitapivat; Agios Pharmaceuticals) is being developed for SCD (currently approved for hemolytic anemia in adults with PK deficiency). This report explores hematologists’ and payers’ views and policies regarding approved SCD therapies and assesses expected use of new therapies in the context of potential coverage of such treatments.

Questions answered

• How are approved SCD therapies covered? What restrictions do payers impose, and how do market access dynamics influence surveyed hematologists’ prescribing?
• What price concessions and clinical data do payers want to favorably cover the gene therapies Lyfgenia and Casgevy and emerging symptomatic SCD therapies?
• How receptive are physicians to gene and emerging symptomatic therapies for SCD? What clinical and nonclinical factors will drive their use, and what payer controls will constrain prescribing?

Content highlights

Geography: United States

Primary research: Survey of 100 U.S. hematologists; survey of 30 U.S. managed care organization (MCO) pharmacy and medical directors (PDs/MDs)

Key drugs covered: Adakveo (crizanlizumab), Endari (L-glutamine), Oxbryta (voxelotor), Lyfgenia (lovotibeglogene autotemcel; lovo-cel), Casgevy (exagamglogene autotemcel; exa-cel), inclacumab, GBT021601, Pyrukynd (mitapivat)

Production description

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 therapies and their receptivity to emerging therapies.

Solution enhancement

Access & Reimbursement includes key analyses from Clarivate Real-World Data Product—comprehensive and timely data that provide clarity around the healthcare experiences and activities of hundreds of millions of patients, HCPs, and payers. Analyses include payer mix, percentage of claim rejections for key therapies, and reasons for claim rejection.