OBJECTIVES: Advanced cell therapies offering a potential curative treatment for severe diseases are now entering health care systems. The clinical and economic evidence supporting these high-cost advanced therapy medicinal products at launch is often limited. There are frequent appeals for further data collection to reduce uncertainty, most notably regarding longer-term clinical outcomes driven by the duration and persistence of treatment effects. However, the value of collecting these long-term data is not always clear when framed in the context of reducing decision uncertainty. Value of information (VOI) analyses can estimate whether this further data collection would be valuable. This study aimed to identify and evaluate all published VOI analyses for advanced cell therapies.

METHODS: A systematic review identified all published VOI analyses for advanced cell therapies. Medline and Embase were searched electronically from inception until May-2023. The search strategy comprised terms for VOI methods and advanced cell therapies (proprietary and international non-proprietary names). Data extracted comprised descriptive information for each study alongside VOI methods and results. Descriptive information was summarised quantitatively; VOI results were summarised by a narrative synthesis.

RESULTS: Three published VOI analyses for advanced cell therapies were identified: tisagenlecleucel for relapsed/refractory diffuse large B-cell lymphoma; tisagenlecleucel for relapsed/refractory acute lymphoblastic leukaemia; and tumour infiltrating lymphocyte cell therapy for advanced melanoma. All studies reported a low population expected value of perfect information (EVPI) at list prices (€0.00; €314,455; approximately €3 million, respectively). After reducing the list price in sensitivity analyses, two studies found the expected value of partial perfect information (EVPPI) was greatest for the survival analysis parameters.

CONCLUSIONS: Further research for advanced cell therapies is unlikely to be valuable when incremental cost-effectiveness ratios (ICERs) greatly exceed thresholds for cost-effectiveness. List price negotiations bringing ICERs in-line with prevailing cost-effectiveness thresholds will increase the value of undertaking further research to reduce uncertainties in longer-term outcomes.