(45) Coverage Practices and Pricing of FDA approved Gene Therapies in the United States

Background: : Gene therapy presents immense promise, especially in the treatment of rare and serious genetic diseases that have limited or no treatment options. Since 2010, 43 cellular and gene therapies have been approved by the FDA with several others in the pipeline. Despite their clinical success, a number of challenges remain including coverage, affordability, and long-term cost effectiveness.

Objective: : The goals of the study are to (i) provide an overview of all the currently FDA-approved gene therapies in the US; (ii) identify any coverage discrepancies among major payers; and (iii) document any existing payer strategies that optimize patient accessibility and overall cost-effectiveness of these therapies.

Methods: : A literature review of FDA-approved gene therapies was conducted using publicly available data from the FDA website, payer coverage policies, and published literature (PubMed). Disease prevalence, therapeutic indications, drug pricing (Average Wholesale Price [AWP]), payer coverage practices and considerations like outcomes-based agreements (OBAs), budget impact analysis (BIA), and cost-effectiveness analyses were extracted. Any therapies related to cord blood or tissue engineered therapies were excluded from the review.

Results: : A total of 29 of the 43 gene therapies were included in the review. Most therapies were approved between 2017-2024 (n=27). For the 29 therapies, 23 different therapeutic indications were listed ranging from rare (Roctavian, Hemophilia A) to extremely common (Lantidra, Type 1 diabetes) indication. Drug prices ranged from $300 to over $9 million per unit with AWP per unit of 11 therapies exceeding $1 million. Interestingly, there was one BIA reported for gene therapy in sickle cell disease ($1.91 PMPM increase in spending). There were inconsistencies in Medicaid coverage across 16 states in terms of target population, coverage scope, and speed of coverage decisions in specific therapies (Zolgensma, Kymriah, and Luxturna) despite federal requirements to cover FDA approved drugs. There were challenges seen in coverage practices as most private insurers operated on a case-by-case basis, sometimes denying coverages and then approving the treatment later on.

Conclusions: : Gene therapies offer transformative alternatives in improving clinical outcomes in patients with rare genetic disorders. However, several barriers exist that delay patient access to these therapies. Coordinated federal strategies, greater price transparency, and innovative reimbursement models are needed to ensure adequate patient accessibility.

Sponsorship:: None