Cost Effectiveness of Transplant, Conventional Chemotherapy, and Novel Agents in Multiple Myeloma: A Systematic Review

Background Treatments for multiple myeloma (MM) have been rapidly evolving. Newly developed treatment regimens are likely to be more effective but also cost more than conventional therapies. Objective We conducted a systematic review to compare the cost effectiveness of different classes of MM treatment. Methods We searched the PubMed, MEDLINE, Web of Science, and EMBASE databases for studies published during 1990–2018 comparing the cost effectiveness of transplant, chemotherapeutic and novel MM treatments. Titles and abstracts were independently reviewed for eligibility by two investigators. The quality of the included studies was evaluated using the 16-item, validated Quality of Health Economics Studies instrument. Results Twenty-four publications were included in the systematic review and summarized according to treatment regimen and line. For first-line treatment, transplant was the most cost-effective option for transplant-eligible MM patients [the incremental cost-effectiveness ratio (ICER) was $4053–€45,460 per quality-adjusted life-year (QALY) gained, and $3848–$72,852 per life-year gained (LYG)], and the ICER for novel agents compared with conventional chemotherapy was $59,076 per QALY and $220,681 per LYG. For second-line treatment, in comparisons of novel agent-based regimens, ICERs were inconsistent. However, bortezomib-based regimens, lenalidomide plus dexamethasone, and pomalidomide plus dexamethasone were each cost effective compared with dexamethasone alone (ICERs showed cost saving, £30,153 per QALY gained, and €39,911 per LYG, respectively). Conclusions For transplant-eligible MM patients, transplant is a cost-effective first-line treatment. More cost-effectiveness analyses comparing novel agents in the first-line treatment regimen are warranted to determine which agent or regimen is the most cost effective. In the second-line setting, it is unclear which novel agent-based regimen is most cost effective, but bortezomib-based regimens, lenalidomide plus dexamethasone, and pomalidomide plus dexamethasone were each cost effective compared with dexamethasone alone.