It has always been a priority for the French authorities to provide compassionate use or exceptional use access to patients suffering from serious and disabling diseases. For more than 20 years, early access was provided under a program known as Temporary Authorization for Use (ATU); however, the program has become increasingly complex over the years.
In response, the two French regulatory authorities, the National Agency for the Safety of Medicines and Health Products (ANSM) and the French Agency for the Safety of Health Products (HAS), have taken steps to speed up and simplify access. The new program, which has also been developed with the support of patients’ associations, also seeks to improve scientific rigor when collecting the patients data and to ensure visibility of the following criteria:
- Serious, rare, or disabling disease
- No existing treatment or adapted-use treatments
- Urgency of treatment
- Strongly presumed safety and tolerance of treatment
The two new early access (Accès Précoce) or compassionate use programs (Accès Compassionnel) came into force on July 1, 2021. The early access program, which replaces post-ATU, cohort ATU (ATUc), ATU extension of indication (ATUei) and temporary coverage (PECT), is for innovative medicinal products for which clinical data is available or where data collection is continuing. Companies undertake to make the product available to patients within two months of being granted early access and to submit a request of marketing authorization within two years.
The second program, a compassionate use program, replaces the named patient ATU (ATUn) and RTU and is granted for medicinal products that should not be marketed in the concerned indication, where no development is ongoing, and where patients cannot receive the product in the context of a clinical trial, but where a patient is expected to benefit from the treatment.
Early access submission process
To be eligible for early access, companies must submit their request to the authorities via a dedicated platform (“SESAME”). While not required, it is advisable that companies make a pre-submission appointment with HAS, followed by a submission request. The company will be informed of the administrative receivability of its request within 10 days via “SESAME”.
The evaluation process takes up to three months. To start with, ANSM conducts a benefit-risk assessment at the end of which they either provide a negative opinion, where they refuse the request and share that opinion with HAS, where they provide no opinion (a silent response), which amounts to a negative response, or where they provide a positive opinion, which again is shared with HAS.
The approval process then moves to HAS, which bases its criteria on severity of condition, whether it is a rare, disabling illness, if there is no therapeutic alternative, and whether the product is innovative. Again, response can be negative, silent, which in this case means the request is approved, or a positive opinion granting the authorization request. The laboratory commits to make available the medicines within 2 months after getting Authorisation for early access, to submit a Marketing Authorisation Application within 2 years max after getting the Authorisation for early access.
With compassionate access, the evaluation process is, carried out by ANSM based on the same criteria, provided that the ANSM is able to presume a favorable benefit/risk ratio for a serious, rare, or incapacitating disease. There a re two types of requests: one is usually made by the physician and authorization is valid for up to a year, although it can be renewed, for a named patient, or the request is made at the initiative of other health actors (ANSM, Minister, Professional Councils…) in order to secure practices of prescribing a drug that does not comply with the MA, the authorization is issued for a period of 3 years, although it can be renewed.
Authorization for early access and compassionate use is subject to a therapeutic protocol of use and collection of data approved by HAS and ANSM and regular reporting is provided to the ANSM and HAS on pre-defined basis. This new process allows an automatic and immediate coverage of the patients by the Health Insurance.
Since the early access program is very new, details have yet to be shared by the authorities. However, this is a positive move for French patients in need of urgent treatment. As more information about the programs are made available, ProductLife Group will share insights with our customers to help you serve your patients.