OBJECTIVES : Single-arm studies (S-AS) can serve as pivotal trials although they are not designed to demonstrate the clinical added value (CAV) of a medicine. We conducted a retrospective study that aimed to show which kind of conclusions the French health technology assessment (HTA) body is drawing from this limited dataset.

METHODS : We analyzed the opinions with a sole S-AS provided for the HTA among all assessments carried out between 2018 and end of Q1 2020 for new drugs and new indications.

RESULTS : Among 645 opinions, 22 were exclusively based on a S-AS. Half of them were for antineoplastic agents and 19/22 targeted a population <600. There was no alternative in 4/22 and the medical need was considered as covered in 3/22 opinions. An indirect comparison was provided for 50% of the dossiers.

4/22 reimbursement recommendations were negative versus 5% for all new medicines. An additional CAV was granted for 9/18, including 3 conclusions for the 2 CAR-T (Yescarta® and Kymriah®). The CAV conclusions have been more favorable when an indirect comparison was performed (6/9 versus 3/9). The HTA body strongly criticized the absence of a direct comparison, especially when a direct comparison was feasible.

CONCLUSIONS : In a minority of medicine assessments, there was only a S-AS. Even though some of these medicines have obtained the recognition of a CAV, the conclusions were less favorable, especially in the absence of an external comparator arm. Improving the appropriate methodological choices when performing only a S-AS should be explored.