The French Authorization for Temporary Use (ATU) and Recommendation for Temporary (RTU) mechanisms enabling a derogation for coverage of medicines were substantially amended by the Social Security Financing Bill for 2021.
With the aim of speeding up market access for innovative medicines and allowing exceptional access for medicines that meet a therapeutic need on an ad hoc basis, the new mechanisms of early access authorization (EAA) and compassionate use were implemented as from 1 July 2021.
Almost a year after the implementation of such new early access mechanisms, the French High Health Authority (HAS) and the French National Agency for the Safety of Medicines and Health Products (ANSM) gave a very positive first report on EEA.
In more detail
EAA is an exceptional derogation mechanism that allows the early availability and early management of one (or more) indication(s) of a medicine, indicated for a serious, rare or incapacitating diseases. Four cumulative conditions must be met to qualify for an EAA: (i) absence of an appropriate treatment; (ii) impossibility of delaying the treatment; (iii) strong presumption of efficacy and safety of the medicine; and (iv) presumed innovative nature of the medicine.
The EEA mechanism can be applied both for indications for which a marketing authorization has already been granted but which is not yet reimbursed (in which case, the pharmaceutical company applying for the EEA has applied or commits to applying for the inclusion of the said indication on one of the lists of reimbursable medicines) and for indications for which no marketing authorization has yet been granted (in which case, the pharmaceutical company applying for the EEA has filed or commits to filing a marketing authorization application).
HAS is the leading competent authority for assessing the EEA applications. Nonetheless, ANSM remains involved and is required to issue an opinion regarding the presumption of efficacy and safety of the medicine where the concerned medicine has no marketing authorization.
Such mechanism directly benefits to patients, allowing them rapid access to medicines for which there is no alternative.
In 10 months, around 100 EEA applications have been filed and half of them have been processed with an 80% positive outcome rate. The average time for instruction and issuance of a decision is only 60 days, while the regulatory timeline is set at 90 days. In the context of the pandemic, the EEA applications related to products designed to treat severe forms of COVID-19 were processed in 35 days.
In light of this first positive report, as well as the experience gained with ANSM over the last 10 months, HAS updated its evaluation principles to sharpen and improve the system.