Journal: Blood
This study is a cross-sectional analysis of how national health technology assessment (HTA) bodies in major economies evaluate and reimburse commercial CAR T‑cell therapies for hematologic malignancies.
Using public HTA documentation from 14 countries among G20 members plus Spain, Singapore, and Switzerland (data through 1 August 2025), the authors examined all 18 currently approved CAR T product–indication combinations. They assessed whether each product–indication pair received a positive or negative reimbursement recommendation and measured the time from initial US regulatory approval to HTA decision.
Key findings:
- Only 48% (122/252) of all possible country–product–indication combinations are currently recommended for public reimbursement.
- The median delay from regulatory approval to HTA decision is 1.54 years (IQR 1.15–2.59), indicating a substantial lag between approval and potential access.
- HTA bodies frequently questioned cost‑effectiveness due to:
- Reliance on single‑arm trials rather than randomized studies
- Small sample sizes
- Immature data on overall survival, long‑term safety, and quality of life
The study concludes that, even among higher-income and upper middle–income countries, there are pronounced disparities in access to CAR T therapies. It underscores the need for both scientific solutions (e.g., stronger evidence generation, more robust trial designs) and policy innovations (e.g., pricing, reimbursement models) to lower costs and expand access to these treatments.