In France, drug assessment is performed by the Transparency Committee (TC) of the French National Authority for Health (HAS). It's based on two criteria: the clinical benefit (CB) for reimbursement recommendation and the clinical added value (CAV) serving the pricing decision. The CAV is rated on a 5-point scale, from I (major) to V (no CAV). A critical step in the CAV assessment is the identification of the clinically relevant comparators (CRC) serving the TC to recognize the appropriateness of the comparators chosen in the randomized controlled trials (RCT). The objective of this study is to investigate the comparator choice consequences on TC appraisals and pricing.

A retrospective, descriptive study included all oncology indications assessed by the TC between 2015 and 2017. Based on a pre-specified grid, items on the comparators were extracted from final TC's appraisals.

Among the 135 indications included, the assessed drugs had no CRC in 20% of cases. A RCT was submitted for 89 indications (66%) whose 67 (76%) were conducted versus a CRC. A CRC was identified by the TC for 70% of the 46 indications without RCT. An important/moderate CAV (II-III) was granted when there was a RCT versus a CRC in 70% of cases, versus 50% and 43% for minor (IV) and no CAV respectively. The public price was reduced by 13.5% in average compared to the claimed price without impact of the CAV level (n = 18).

In oncology, comparative data assessed by the TC met its expectations (RCT versus CRC) in a majority of cases. When there is no RCT or a comparison versus a non-relevant comparator the CAV appraisal is decreased. Surprisingly this study hasn't shown any impact of this decrease on the public price. A wider analysis in different medical areas would need to be performed to better investigate these results.