OBJECTIVES

In 2006, European Medicines Agency (EMA) put in place a special marketing authorization called “approval under exceptional circumstances (EC)” for promising products with limited clinical evidence. The aim of this study was to identify how the French Health authority (HAS) reacts to drugs approved under EC, in terms of assessment of Actual Benefit (AB) and Improvement of Actual Benefit (IAB).

2. METHODS

All drugs approved under EC were identified from EMA website up until October 2019, their available French Health Technology Assessments reports were downloaded from HAS website and reviewed. Drug information were extracted in addition to HAS decision on AB, IAB and clinical evidence limitations.

3. RESULTS

A total of 34 drugs were approved under EC, among which 7 were withdrawn (21%). In France, 22 drugs were assessed by the HAS (81.5%). Only 3 drugs received a negative recommendation, mainly due to limited patient number, lack of efficacy, and uncertainties concerning the safety. AB was appraised as important for 13 drugs. Among them, IAB was rated as 1 (major) for Orphacol^Ò, 2 (important) for Strensiq^Ò, Atriance^Ò, Elaprase^Ò, and Evoltra^Ò, 3 (moderate) for Naglazyme^Ò and Brineura^Ò, 4 (minor) for Myalepta^Ò, Qarziba^Ò, Lojuxta^Ò, Vyndaqel^Ò, and Vedrop^Ò, and 5 (weak) for Obizur^Ò. The majority (69%) had an orphan designation and were paediatric medicines, mainly treating endocrine, nutritional or metabolic diseases, and neoplasms. Most of these drugs (84,6%) had non-comparative trials and limited/lack of evidence on clinical efficacy benefit.

4. CONCLUSIONS

Even with limited clinical evidence, HAS issued positive recommendations for reimbursement of drugs approved under EC, allowing faster patient access to drugs with no available alternatives and high unmet need. In France, single-arm trial and limited efficacy data does not seem an obstacle for reimbursement of these promising drugs for untreated diseases.