OBJECTIVES: In France, patients benefit from early access to innovation, ahead of the marketing authorization approval, via the compassionate use program (ATU). Medicinal products indicated in a severe, rare and/or chronic disease are likely to be eligible mainly when there is no suitable treatment available and when both efficacy and safety are relevant. ATU approval is associated with a data collection protocol (PUT). Real world data (RWD) have to be collected during ATU. HTA agency is resquesting ATU data during the reimbursement assessment.

We investigate how ATU data are valuated by the Transparency Commission (TC) in France.

METHODS: We performed a retrospective analysis of TC assessments published on the HAS website in 2019. We read and collected information about how these ATU data were value in the reimbursement appraisal.

RESULTS: Amongst the 130 TC evaluations reviewed, 18 products were available in an ATU program (50% being onco-hematology drugs). For 90% of the products, the TC concluded clinically relevant comparator(s) exist(s). 78% of the ATU products were valued with a medical improvement rating (ASMR IV [minor] or better). The median target population was very low: 51 years (min-max: 0-1679)

The data valued by the TC were: patients’ characteristics and clinical outcome, efficacy, safety and prescription data (treatment line, prescribers, etc.). All of these data were displayed for only 2 products. For these 2 products, the TC valued the data collected.

CONCLUSIONS: Collecting ATU data allows to gather early real world evidence data, which are an asset for successful market access. Those data are considered as valuable by TC and mentioned in the apraisal.

The challenge is to optimize the collection of relevant ATU data for market access considering the short time available to set up an ATU, and TC dossier submission mandatory deadlines.