An increasing number of anti-cancer medications are indicated for multiple tumors. Existing pharmacoeconomic evaluations routinely examine the cost-effectiveness (CE) and budget impact (BI) of such drugs by indication, as and when each indication is reviewed. The impact of indication-specific conclusions on the holistic value of such medications across all indicated patients is not currently evaluated, yet is important to stakeholders including health technology assessment (HTA) agencies, payers and patients. We introduce a holistic framework that considers the value of multiple indications together at a product level. Application of this approach is illustrated via an example across multiple indications for a novel, targeted anti-cancer therapy (pembrolizumab) in Canada.

Previously-HTA-evaluated indication-specific CE and BI models serve as the foundation for this multi-indication model. Comparing to standard of care (SoC) per indication, the model evaluates the potential BI, clinical outcomes and CE of pembrolizumab among the individual indications along with the overall multi-indication patient population from the perspective of a third-party payer. For the contextual model, incremental costs and quality-adjusted life years (QALYs) were weighted using indication populations derived from national incidence rates.

The indication-specific incremental cost-effectiveness ratios (ICER) from CE analyses of ipilimumab-treated advanced melanoma, ipilimumab-naïve advanced melanoma, second-line non-small cell lung cancer (NSCLC), first-line NSCLC and fourth-line classical Hodgkin lymphoma range from USD 52 K to USD 163 K per QALY. Accounting for the relative contributions of the various sizes of indication-specific patient populations results in an overall ICER for pembrolizumab vs. SoC of USD 100 K.

A holistic model can provide stakeholders with a tool to evaluate the overall value of multi-indication drugs. Results enable an understanding of the outcomes and economic consequences of treatment with pembrolizumab versus SoC by both individual indications and across all indications. Insights from this contextual approach will enable data from less-developed clinical trials to be considered when previously they might have gone unevaluated by decision-makers.