Systematic review of the economic evaluations of novel therapeutic agents in multiple myeloma: what is the reporting quality?

WHAT IS KNOWN AND OBJECTIVE: Given the increasing healthcare costs and the recent introduction of novel agents in the treatment for multiple myeloma (MM), an incurable haematologic malignancy, more efficient use of existing resources is fundamental. The objective of this study was to systematically review economic evaluations of the use of novel agents in MM and assess their reporting quality.

METHODS: A literature search was performed in PubMed/Medline, Latin American and Caribbean Health Sciences Literature, Cost-Effectiveness Analysis Registry and the National Health Services Economic Evaluation Database for economic evaluations up to June 2015. The search strategy included Medical Subject Headings terms or text words related to MM, economic evaluations and drugs. Full economic evaluations of bortezomib, thalidomide or lenalidomide in patients with MM that were published in English, Portuguese or Spanish were included. Two independent authors performed study selection, data extraction and quality assessment using 24 items from the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement.

RESULTS AND DISCUSSION: Of the 132 potentially relevant records identified, eight satisfied the inclusion criteria. Most studies were cost-effectiveness analyses combined with cost-utility analyses (n = 6) from the public payer perspective (n = 4) and were performed in Europe (n = 6) on patients with refractory or relapsed MM (n = 5). All studies were based on economic models, with four of them using discrete event simulation. We found bortezomib-based therapies to be one of the more commonly selected treatment strategies for comparison (n = 7). Overall, the intervention was more effective and costlier than the alternative strategy (average of $54 630 per life year; $68 261 per quality-adjusted life year-QALY). The CHEERS' total score was 14·6 (SD = 2·6) with the most frequent problems being the lack of precision measures for all model parameters, no evaluation of heterogeneity of the results by subgroup analyses and no description of the role the funder in the identification, design, conduct and reporting of the analysis.

WHAT IS NEW AND CONCLUSION: Most analyses of the novel therapeutic agents determined that they were cost-effective in MM at a threshold of up to $100 000/QALY. Nevertheless, the poor reporting quality of the economic studies requires improvement to ensure greater transparency.