Presentation (CTI)

OBJECTIVES: Regulators classify gene therapies as Breakthroughs, though payers may disagree. This study compared treatment criteria in payer policies to FDA-approved indications to assess payer-regulator alignment on the value of Hemophilia B gene therapies.
METHODS: Approved Hemophilia B gene therapies were selected for review and key data abstracted: FDA approval date, labeled indication from the FDA Approved Products database; pivotal trial inclusion / exclusion criteria from clinicaltrails.gov. Policies, coverage criteria, and publication dates were collected from payer websites (United, Elevance, Aetna, Cigna, HCSC, BCBS Michigan). Policy publication dates were confirmed using document revision history (where available), else via web archive. Coverage criteria for each product were compared against the FDA labeled indications and trial criteria. We defined policies as “restrictive” if there were additional patient eligibility criteria compared to the approved label and determined if patients had to meet or exceed trial criteria to gain access. Policy date minus FDA approval date defined time to policy. Descriptive statistics were generated.
RESULTS: The study included two FDA-approved Hemophilia gene therapies: Hemgenix (22November2022) and Beqvez (25April2024). All policies (12/12) were classified as restrictive, as 100% narrowed the covered patient population compared to FDA labels, and 67% (8/12) limited treatment eligibility to a population meeting (n=3) or exceeding (n=5) trial criteria. Level of restrictiveness was similar for each therapy, with exception of BCBS Michigan, which listed Beqvez as a “preferred product.” Most payers (4/6) took longer to publish the first Beqvez policy, though mean time to initial policy was similar (92.5 days Hemgenix, 97 days Beqvez).
CONCLUSIONS: US payer policies defined a narrow patient population for Hemophilia B gene therapies compared to product FDA labels, sometimes restricting eligibility beyond clinical trial criteria. This targeted review identified misalignment between payers and regulators on the clinical value of Hemophilia B Breakthroughs.