OBJECTIVES:

Tumor-agnostic immunotherapies such as pembrolizumab are known to provide several dozen treatment indications for a single compound. Usually, the first registered indication of a drug shapes reimbursement decisions of all its future indications. However, these later indications are rarely assessed in terms of value for money. Through examination of seven indications of the same compound, this study aims to elucidate the broader cost-effectiveness landscape of pembrolizumab following the automatic reimbursement of these additional indications in The Netherlands.

METHODS:

First, a full cost-effectiveness analysis evaluated the first-line treatment of pembrolizumab in locally advanced non-small cell lung cancer (NSCLC) using a Markov model. Then, these cost and utility inputs, and results of sensitivity analyses guided the overall cost-effectiveness assessment of pembrolizumab for seven other indications, including first- and second-line head and neck cancer, first-line renal cell carcinoma, melanoma, and NSCLC. Estimations of overall survival and progression-free survival, pembrolizumab- and standard-of-care costs, were tailored to each indication. Throughout all indications, utilities per health state, adverse event costs and end-of-life costs were kept constant. All analyses included cost and utility inputs of peer-reviewed studies.

RESULTS:

The first-line treatment of NSCLC generated an ICER of €126,474/QALY. Other indications produced ICERs ranging from approximately €136,000/QALY to €2,6 million/QALY, with two negative ICERs amounting to -€4,3 million/QALY. Upon deeper examination, no correlation existed between the magnitude of the ICER and the line of treatment, the treatment context or the cancer histology.

CONCLUSIONS:

This study found that various indications of the same immunotherapy can yield substantially different cost-effectiveness outcomes, most of which barely approach cost-effectiveness thresholds. Consequently, separate cost-effectiveness calculations per indication are recommended. In The Netherlands, established discounts covering all new indications plausibly prompted unfavourable financial agreements. For new immunotherapies, health authorities should use caution when applying a ‘one compound, one price’ reimbursement system.