Abstract

Marketing authorisation holders are compelled to set a single uniform price for multi-indication pharmaceuticals entailing limited flexibility in adjusting the incremental cost-effectiveness ratio (ICER) of each indication. Indication-specific pricing has been suggested; however, this is often not feasible due to lack of detailed drug consumption data. In light of this, a novel method of expanding the ICER into a weighted average variant dependent on the sizes of the respective indications’ eligible patient populations has been suggested. The objectives of this early study were to examine the practical and methodological potentials of a weighted average ICER as a supplementary decision tool when assessing the cost-effectiveness of multi-indication pharmaceuticals. A weighted average ICER was computed with nivolumab as a case example, for six out of eight of nivolumab's monotherapy indications. The expected costs and Quality-adjusted Life Years (QALYs) were estimated using partitioned survival analysis to construct cost-utility analyses. Efficacy data was from registrational CheckMate trials. Costs included resource use at the hospital, adverse events and patient's time and transport and were estimated using a mixed micro- and macro costing approach. The pharmaceutical acquisition costs were based on list prices. Utility weights were estimated using EQ-5D-3L. This study succeeded in establishing a weighted average ICER for nivolumab at € 100,999/QALY. The use of a weighted average ICER could entail improved patient access of multi-indication pharmaceuticals and promote a supplier-incentive to research and develop in add-on indications with small patient populations. A weighted average ICER could decrease the transparency in the decision-making process and would require a shift in how the ICER is interpreted. This early study identified the practical and methodological potential of a weighted average ICER and suggests that the method could be applied as a supplementary decision tool when assessing the cost-effectiveness of multi-indication pharmaceuticals.

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