Presentation (CTI)

OBJECTIVES: Gene therapies offer significant clinical potential, yet pose substantial reimbursement challenges of high prices and uncertain long-term results. The National Institute for Health and Care Excellence (NICE) has typically appraised these therapies via the highly specialised technology (HST) route for ultra-rare and debilitating conditions (employing higher cost-effectiveness thresholds and lower evidence requirements), or via the single technology appraisal (STA) route and the Cancer Drugs Fund (CDF) for cancers not meeting HST criteria. However, NICE has recently recommended gene therapies for non-cancer conditions via standard STA, despite list prices >£1.6 million. We assessed the factors considered and whether the Innovative Medicines Fund (IMF) was employed to enable NICE’s recommendations for high-cost, non-cancer therapies not meeting the criteria for HST.
METHODS: NICE STA guidance on gene therapies for non-cancer conditions as of June 2025 was reviewed.
RESULTS: Three STAs were identified: Casgevy for severe sickle cell disease (TA1044), Casgevy for transfusion-dependent beta-thalassaemia (TA1003), and Hemgenix for moderately severe or severe haemophilia B (TA989). NICE recommended managed access for all three via the IMF (confidential discounts), rather than routine use. The committees for both Casgevy appraisals considered higher cost-effectiveness estimates than usual (≤£35,000 per quality-adjusted life-year for sickle cell disease) due to the innovative treatment paradigm and potential impact on health inequalities, such as those related to ethnic background. Despite highly uncertain estimates, NICE concluded there was potential for cost-effectiveness in all three appraisals, and additional data collection could resolve some uncertainties (e.g., long-term effects). Companies were advised to use the committee’s preferred assumptions for future guidance review. The therapies will be available with interim time-limited funding (≤5 years) provided by the IMF.
CONCLUSIONS: High-cost gene therapies can be recommended by NICE via standard STA under managed access if there is plausible cost-effectiveness potential and new evidence could feasibly be collected to address enough uncertainties.