OBJECTIVES

Cell and gene therapies (CGTs) are novel, complex treatments that pose specific challenges for payers. With the increasing number of high-cost CGTs likely to strain budgets, multinational HTA collaborations to review methods or conduct joint assessments could become more important and common for these therapies. This research compared assessments of CGTs across HTA organisations to understand how assessment methodologies vary and how this impacts outcomes.

METHODS

Six HTA organisations were selected due to their use of cost-effectiveness and participation in international HTA collaborations (CADTH, ICER, NICE, SMC, TLV, ZIN). Information was sourced from official agency websites, peer reviewed publications and grey literature to understand each organisation’s methods and policies. HTA reports published by June 2020 from the scope organisations were retrieved for three CGTs case studies (tisagenlecleucel, axicabtagene ciloleucel, voretigene neparvovec) to investigate timelines, cost per QALY outcomes and recommendations.

RESULTS

Although all organisations studied used cost-effectiveness, there were differences in methodologies and policies, including the use of explicit vs implicit cost per QALY thresholds and how thresholds are employed. Differences in assessment times for CGTs were observed across organisations, ranging from ~4 months (CADTH) to 9-12 months (NICE). Significantly different cost per QALY estimates were reported across organisations for the three CGTs due to various factors, including: perception of long-term data, patient and clinician input, and weighting of societal impact. For voretigene neparvovec, cost per QALY estimates ranged from €426,000 (ICER) to €127,760 (NICE). Both the cost per QALY differences and the differences in thresholds resulted in varied reimbursement recommendations.

CONCLUSIONS

While several organisations employ process modifications for high-cost therapies, few are CGT-specific. Organisations differ in how they handle data uncertainties and higher upfront treatment costs, resulting in varied cost per QALY outcomes. International collaborations provide opportunities to share learnings as more CGTs undergo HTA appraisal.