Cost-effectiveness of healthcare interventions for rare cancers: Evidence from a systematic literature review and meta-analysis

Background: Rare cancers account for 20-24% of all cancer diagnoses in Europe and have been recently recognized as a public health issue due to the lower survival compared to common cancers. However, the evidence about the cost-effectiveness of interventions for rare cancers remains unclear. The aim of this study was to review economic evaluation studies of these interventions, assess their quality, and provide policy-makers with summary estimates about the value-for-money of these interventions. Methods: We systematically searched Medline, EMBASE and governmental reimbursement agencies by following the PRISMA guidance and selected economic evaluations of healthcare interventions for rare cancers based on predefined criteria. A template was developed to extract study and patient characteristics as well as reported outcomes and costs. The CHEERS checklist was used to assess the quality of the studies and costs were inflated to 2016 prices and converted to British Pound. A random effects meta-analysis, using study quality scores as weights, was performed to pool outcomes and costs and to explore differences between types of rare cancer and study origin. Results: Out of 1991 screened studies, 32 economic evaluations of interventions for sarcoma, malignant pleural mesothelioma and thyroid carcinoma were selected. Almost all of them evaluated drug treatment and surgeries (n=30; 94%) and were originated from North America (n=8; 49%) and Europe (n=7; 43%). Half of these studies were NICE reports and their results ranged from 20300 pound to 59000 pound per quality adjusted life year (QALY). The 16 published studies were assessed to be of mediocre quality, particularly in describing the assumptions underpinning decision-analytic models and the methods used to handle uncertainty or population heterogeneity. The meta-analysis of their results showed that the pooled incremental cost of these interventions was 3410 pound (95% CI 821- pound 7642) pound per patient per year. In term of outcomes, the pooled incremental QALY was 0.20 (95% CI 0.04-0.37). Conclusion: Compared to NICE suggested thresholds and cost-effectiveness ratios of reimbursed interventions for common cancers, interventions for rare cancers seem to be value-for-money. More collaborative research is needed to realise their full potential for improving efficiency and equity in healthcare.