CELL INTELLIGENCE · 肿瘤细胞治疗研究

BACKGROUND: Advanced cell therapies often face high parameter uncertainty at launch, prompting calls to collect further data about long-term effectiveness and safety. However, the value of collecting these data to support resource allocation decision-making is not known. Therefore, this study aimed to appraise all published value of information analyses for advanced cell therapies. METHODS: A systematic review (PROSPERO: CRD42023446874) identified value of information analyses for advanced cell therapies between inception and 14 May 2025 (databases: Medline; Embase). Included studies reported the expected value of perfect information (EVPI), expected value of partial perfect information (EVPPI), expected value of sample information (EVSI) or expected net benefit of sampling (ENBS). Study design and value of information results were summarised in a narrative synthesis. Quality of reporting was assessed using the Consolidated Health Economic Evaluation Reporting Standards Value of Information (CHEERS-VOI) checklist. RESULTS: Three published value of information analyses were identified: tisagenlecleucel for relapsed/refractory acute lymphoblastic leukemia; tisagenlecleucel for relapsed/refractory diffuse large B-cell lymphoma, and tumor infiltrating lymphocyte cell therapy for advanced melanoma. The beneficiary populations were 6, 36, and 400 individuals per year, respectively. All studies reported EVPI; two studies reported EVPPI. Estimated base case population EVPI was: €314,455, €0, and €2,250,000, respectively. Estimated EVPPI indicated that input parameters for survival extrapolations were the most valuable targets for further research specifically during scenario analyses that explored a lower cost of treatment acquisition. CONCLUSIONS: Value of information analyses will help decision-makers, analysts, and manufacturers understand whether long-term data collection is worthwhile to reduce decision uncertainty for advanced cell therapies at launch. Current estimates indicated that the value of further research is likely to be low. The value of collecting additional data will likely increase if future advanced cell therapies are priced such that their corresponding incremental cost-effectiveness ratio aligns with a relevant cost-effectiveness threshold and if they are indicated for larger beneficiary populations.