OBJECTIVES: To study the relationship between the ATU status, reimbursement decisions, and their underlying metrics (SMR ratings, ASMR levels, and reimbursement rates).

METHODS: A total of 1,645 reimbursement decisions from the last five years (2015 to 2019) were collected from the Haute Autorité de Santé (HAS) website irrespective of disease condition. Relevant data regarding the reimbursement decision, Temporary Authorization for Use (ATU) statuses, additional benefit (SMR) ratings, improvement in additional benefit (ASMR) levels, and reimbursement rates were then extracted from these documents to create a structured database. The ATU status was captured as a “Yes” or “No.” HAS SMR levels (substantial, moderate, low, and insufficient), ASMR ratings (a scale of 1 to 5), and reimbursement rates (100%, 65%, 35%, 30%, 15%, and 0%) were used. The impact of ATUs on reimbursement decisions was evaluated first. The underlying metrics were then studied. Chi-squared testing was further used to measure significance.

RESULTS: There was no correlation between ATUs and overall reimbursement decisions by HAS (p = 0.481). However, there was a correlation between drugs having an ATU and a better ASMR level (p < 0.001). There was also a correlation between drugs having an ATU and receiving a higher reimbursement rate, particularly 100% reimbursement (p < 0.001). In addition, drugs with a proposed ATU that received a negative reimbursement decision either did not present clinical data or presented data that were deemed insufficient by HAS.

CONCLUSIONS: With this analysis, it was observed that a positive ATU is associated with a better ASMR level and a higher reimbursement rate. It shows that the ATU pathway is often granted to drugs that then demonstrate some improvement in additional benefit, and the pathway may be associated with a financial benefit for pharmaceutical companies.