Ten months after reforms to the early access scheme (read more about the new scheme and what it means for manufacturers here) took effect (on 1st July 2021), the High Authority for Health (HAS) and National Agency for Drug Safety (ANSM) have published a preliminary analysis of the reforms.
In 10 months, around 100 requests for early access (pre- and post-marketing authorisation) have been submitted. As at 30 April, 50 decisions have been issued, 80% (40 decisions) of which ended in early access being granted – 15 of these decisions related to drugs pre-marketing authorisation while 25 were granted to drugs post-marketing authorisation. In terms of therapy areas, oncology/haematology had the highest number of positive decisions (20) followed by infectology (7). In total, the 40 positive decisions were spread across 11 therapy areas.
For the 50 decisions, 23 were supported by written contributions from patient associations. In terms of timelines, decisions were issued in 60 days on average (35 days for COVID-19 treatments). Key decisions highlighted by the ANSM include positive decisions for Trodelvy (sacituzumab govitecan) and Keytruda (pembrolizumab) in the treatment of triple negative breast cancer.
The HAS notes that, in view of its initial assessment of the new scheme, and taking into account feedback from the ANSM, manufacturers and patient associations, HAS is modifying its “assessment principles”.
Specifically, the new HAS “doctrine” (which sets out its assessment principles) introduces more flexibility to assess whether there is or not an appropriate treatment in the disease area. In exceptional cases, even if there are clinically relevant options to treat the disease, if the treatment has the potential to respond to a health emergency, the HAS reserves the right to not take into consideration the existing treatments as “appropriate treatment”.
The HAS notes that the other major change made to the doctrine relates to the “presumption of innovation”. “The doctrine now specifies that a medicinal product likely to be innovative within the framework of an early access authorisation must meet the following three conditions supplemented by the additions indicated in bold type:
- Constitute a new method of care likely to bring a substantial change to patients
- Have an appropriate development plan and present clinical results supporting the presumption of benefit for the patient in the context of the existing therapeutic strategy
- Fulfil a medical need that is either not or insufficiently covered”.
- www.ansm.sante.fr, “Autorisation d’accès précoce aux médicaments : un premier bilan positif et des principes d’évaluation affinés”, 20th May 2022