(48) The impact of the Cell and Gene Therapy Access Model on state Medicaid coverage of sickle cell disease gene therapies

Background: : The CMS Cell and Gene Therapy (CGT) Access Model supports value-based purchasing (VBP) between Medicaid and manufacturers, initially targeting SCD gene therapies (GTs) (exagamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel). SCD was chosen due to the disproportionate share of people with SCD covered by Medicaid and high therapy costs. The Model launched in May 2025 with 35 states covering 84% of Medicaid SCD beneficiaries. Participating states require CMS approval for VBPs and must adopt a standardized access policy based on CMS-negotiated key terms.

Objective: : To evaluate if the CGT Model (1) impacted Medicaid SCD GT coverage and prior authorization (PA) criteria, and (2) influenced these trends in states that recently gained VBP approval.

Methods: : In June 2024, January 2025, and May 2025, we reviewed state Medicaid websites to identify SCD GT coverage and PA criteria for changes that may have resulted from participation in the CGT Model. We compared SCD GT management changes in states receiving VBP approvals from CMS in 2024 and 2025.

Results: : From June 2024 to May 2025, states with SCD GT covered status increased from 9 to 20 and preferred status increased from 6 to 15. In May 2025, 9 of 15 states cover both GTs with no preferred product, up from 3 of 6 in June 2024. 4 states prefer exagamglogene and 2 prefer lovotibeglogene. Following the January 2025 review, 2 states moved from preferring 1 GT to preferring both while 1 moved from non-preferring both to preferring 1.
Vaso-occlusive crisis (VOC) thresholds were used in 21 of 22 states. 9 of 22 states followed clinical trials: ≥4 VOCs in 2 years for lovotibeglogene; ≥2 VOCs/year in 2 years for exagamglogene. Colorado required 7 VOCs in 2 years.
5 of 22 states did not require genetic confirmation of SCD or failure of hydroxyurea. 10 of 22 states had criteria disqualifying the patient if a sibling donor was available and permitted stem cell transplant. 3 states have criteria related to infertility risks.
Of the states receiving VBP CMS approval in 2024 and 2025 with PA criteria, 4 of 6 had similar PA criteria, including VOC thresholds aligned with product labeling.

Conclusions: : States showed changes in SCD gene therapy coverage and preferences which are potentially linked to CGT Model participation. However, varying PA criteria suggest the Model’s key terms haven’t significantly influenced requirements—though timing may play a role. States with recent VBP approvals tend to have aligned PA criteria; these approvals may support broader VBP adoption beyond SCD and the CGT Model.

Sponsorship:: IPD Analytics