OBJECTIVES :

The French Health Authority “HAS/CEESP” performs health-economic evaluations for innovative drugs that could have a significant impact on health expenditure. Since 2013, four opinions dealing with renal cell carcinoma (RCC) treatments were published. Objectives were to compare the choices made by pharmaceutical manufacturers and the objections raised by the HAS in order to identify areas of improvement in the health-economic evaluation of RCC in France.

METHODS :

We analyzed the four opinions evaluating: nivolumab, nivolumab+ipilimumab, cabozantinib, and pembrolizumab+axitinib. The evaluations were conducted by the HAS/CEESP between November 2016 and March 2020. For each of the four opinions, the methodological choices were described, and the acceptability level was analysed.

RESULTS :

The efficiency opinions in RCC were analyzed in terms of objections raised by the HAS/CEESP. Although all four of them covered different indications in RCC, only pembrolizumab+axitinib raised major objections. Those were mainly related to the non-exhaustivity of comparators included and to the non-consideration of different risk groups in RCC. Important objections that were raised mainly covered the modelling of survival data. Robustness of indirect comparisons was also criticized, and the validity of hazard-ratios estimated was discussed in both nivolumab and nivolumab+ipilimumab efficiency opinions. External validation of the model, as well as sensitivity analyses, need to be thoroughly addressed. The remaining structural settings like QoL data and time horizon were also analysed and discussed. Objections raised regarding budget impact analyses were closely related to those raised for cost-effectiveness analyses.

CONCLUSIONS :

The HAS/CEESP stated that pembrolizumab+axitinib was not efficient but did not clearly state whether efficiency was reached in the remaining dossiers. Analysis of the objections highlighted the parameters for which further robustness is needed for future RCC economic evaluations.