OBJECTIVES

European Medicines Agency (EMA) can grant promising products with limited clinical evidence with a special marketing authorization called “approval under exceptional circumstances (EC)”. This process can accelerate patients' access to drugs addressing high unmet needs, while additional data are collected to reduce the uncertainty. This study aims to review the assessment of drugs approved under EC by French Health Authority (HAS), the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) and the challenge they faced.

2. METHODS

All drugs granted EC up until October 2019 were identified from EMA official website and their Health Technology Assessment (HTA) reports were downloaded from HAS, NICE and SMC official websites.

3. RESULTS

A total of 34 drugs were approved under EC among which 7 were withdrawn (21%). In France, 28 drugs were assessed by the HAS (82%), 19 were recommended (56%) among which 13 (68%) were appraised with an important actual benefit (AB). In England, 4 drugs were recommended (12%) and 1 not recommended (3%). In Scotland, 6 drugs were recommended (23%) and 7 were not recommended (21%) while 3 of them had an important benefit in France. Unmet medical need was one of the drivers of positive opinions; in most cases, it outweighed scientific uncertainties in France whereas in England and Scotland, “no sufficiently robust economic analysis provided” was the main limitation.

4. CONCLUSIONS

It is important to recognize that, while EC may be a path to expedited regulatory approval, it could be challenging from a market access perspective. Clinically driven HTA may be more favorable for these products. Engaging with HTA bodies at an early stage could help to reach the market with clinical evidence which can meet the HTA agencies’ expectations and therefore speed up patient access.