Objectives: Theoretically, combination therapies would face greater difficulty in demonstrating cost effectiveness because the backbone therapy is often priced close to the relevant country’s cost-effectiveness threshold. Unless the backbone therapy’s cost is reduced, the combination may not be cost-effective even if the add-on therapy is priced at zero. We set out to verify it this is true in practice by assessing reimbursement outcomes for combination therapies vs monotherapies in lung cancer and multiple myeloma.

Methods: Reimbursement status and level of reimbursement for all drugs in lung cancer and multiple myeloma, which were first priced between 1 January 2011 and 31 December 2020, were assessed in France, Germany, Italy, Spain and the United Kingdom using data from the IHS Markit POLI database. The reimbursement status review was supplemented by Amélioration du Service Médical Rendu (ASMR) ratings in France, Federal Joint Committee (G-BA) ratings in Germany and NICE guidance in the UK to assess the likely pressure on prices for those combination therapies that gained reimbursement.

Results: In lung cancer combination therapies were more likely to be rejected for reimbursement compared to monotherapies. Across the five countries, 20 out of the 56 combination therapy presentations (equivalent to 35.7%) were rejected for reimbursement compared to 11.4% (31 out of 271) for monotherapy presentations. In multiple myeloma, 5.3% of combination therapy presentations (7 out of 132) were rejected for reimbursement, while every single monotherapy was approved for reimbursement. Combination therapies also had less favourable ASMR and G-BA ratings.

Conclusions: This review of reimbursement decisions and cost-effectiveness assessment outcomes for drugs approved over a 10-year period in the top five European markets confirms that combination therapies in lung cancer and multiple myeloma face greater difficulty in demonstrating cost-effectiveness compared to monotherapies. Even when approved for reimbursement, combination therapies are subject to greater pressure on prices.