Context:

The French regulatory agency is in charge of a compassionate use program (CUP) which enables access to innovative drugs before their marketing authorization. This CUP foresees a data collection, under the company’s responsibility, on conditions of use, efficacy and safety. These data need to be submitted by pharmaceutical companies to the French authority for health (HAS), in charge of Health Technology Assessment.

This analysis aims to evaluate how these data are taken into account when providing recommendations for reimbursement and pricing purposes.

Methods:

This analysis was performed on drugs available through a CUP and assessed by HAS between 01/01/2018 and 31/12/2018. Data were extracted from HAS internal database.

Results:

19 drugs available through a CUP were assessed by HAS representing 26 therapeutic indications. CUP data were available in 25/26 submission files and reported in the assessment document. Half of these dossiers reported data on conditions of use only and in 7 dossiers data on efficacy and safety were also submitted.

In 5 cases, CUP data were explicitly mentioned in the appraisal (scores or estimation of the target population). The weight of CUP data on appraisals was particularly important when the feasibility of randomized controlled trial was limited, such as in the evaluation of naloxone. In the remaining 20 dossiers, CUP data were not specifically mentioned in the appraisal as they were focused on conditions of use and not detailing efficacy/safety results.

Conclusion:

In most cases, data collected thought the CUP are submitted to the HAS and are systematically reported in the assessment document.

However, the quality of these data is unequal; therefore they cannot be systematically used in the appraisal. HAS would like to see the quality of CUP data increased, as they represent one of the unique way to collect real life data at the time of the first assessment.