- France has made changes to its long-standing Authorization for Temporary Use (ATU) and Recommendation for Temporary Use (RTU) systems via the Social Security Finance Bill 2021 (PLFSS 2021) – and the changes are set to be implemented from next month
- The French alternative reimbursement pathway is currently divided into six systems, but under the reforms, the methods will be combined and just two new mechanisms will be introduced: Early Access Authorization (EAA) and Compassionate Use (CU)
- The new EAA system will be used primarily for innovative medicines and will be subject to the relevant company’s commitment to apply for an MA or for registration on a reimbursement list, within a specific timeline. The CU pathway will instead be used for certain medicines in specific therapeutic indications and for which, subject to exceptions, no development process is contemplated
PARIS, France – France has made changes to its long-standing Authorization for Temporary Use (ATU) and Recommendation for Temporary Use (RTU) systems via the Social Security Finance Bill 2021 (PLFSS 2021).
As it stands, the ATU and RTU schemes offer early access opportunities for patients who are in need of medicines that are going through authorization in France. The scheme has so far allowed for rapid access to targeted therapies, immunotherapy, and cell and gene therapies, which have prolonged patient survival or offered a cure for certain serious illness such as leukemia and lymphoma.
The systems have been in place since 1994, with various patient access options such as nominative ATU (nATU) or, more frequently, cohort ATU (cATU). These types of authorization inevitably require follow-up and data collection. Additionally, manufacturers must then submit a market authorization (MA) application within a pre-determined period.
The French alternative reimbursement pathway is currently divided into six systems, but under the reforms the methods will be combined and just two new mechanisms will be introduced:
- Early access authorization (EAA)
- Compassionate use (CU)
Both will come into force on a date to be determined by decree, but no later than 1 July 2021.
Early Access Authorization
The new EAA system will be used primarily for innovative medicines and will be subject to the relevant company’s commitment to apply for an MA or for registration on a reimbursement list, within a specific timeline.
To be granted an EAA under the new system, a therapy or product must meet the following criteria:
- Absence of an appropriate treatment
- Impossibility to delay the treatment
- Strong presumption of efficacy and safety of the medicine
- Presumed innovative nature of the medicine
France’s National Authority for Health (HAS) will act as the competent authority undertaking the assessments, but in the event of medicines that do not have a MA, the French National Agency for the Safety of Health Products (ANSM) will also be required to give an opinion. This change ultimately gives HAS more decision-making power towards manufacturers.
Free and unilateral price setting will henceforth be decided by the relevant pharmaceutical company for submission. Additionally, a mechanism of rebates paid by the company is maintained but will be simplified compared to the ATU system. For example, if the final negotiated price is lower than the price set during the EAA talks, the drugmaker will be required to pay back the difference.
The CU pathway will instead be used for certain medicines in specific therapeutic indications and for which, subject to exceptions, no development process is contemplated.
The authorization will be given on the following terms:
- The absence of appropriate treatment to medicines that,
- Subject to exceptions are not subject to research involving the human person for commercial purposes and
- For which efficacy and safety are presumed to be favorable
Once these criteria have been met, there are two available pathways for the product to follow to gain patient access.
The compassionate access authorization: upon the request of the prescribing physician, ANSM may authorize the use of a medicine that does not have an MA for the treatment of a serious, rare or incapacitating disease for a named patient;
The compassionate prescription framework: ANSM may establish, on its own initiative or upon the request of a minister, a compassionate prescription framework for a medicine that has an MA in other indications, in order to secure a prescription that does not comply with this authorization.
If the therapy is not yet covered by the French reimbursement system, it will be covered on the basis of the price set freely and unilaterally by the pharmaceutical company that submitted the application, or on the basis of an annual flat rate per patient set by the decree.
Additionally, a limit on the total number of CAP requests for the nATUs per product has been introduced. However, if this number is reached the company will be given the option to file an application for an EAA.
The PLFSS published on December 14, 2020, first penciled in the upcoming changes to the system.
Following the implementation date of July 1, 2021, ATUs with an expiry date after this date will not be eligible for renewal and will have to reapply.
The law ultimately sets the bar for evidence requirements higher than it previously was, both in terms of quality and quantity, potentially leading to a lower number of products being approved through these mechanisms in the future. However, this has yet to be seen.
According to the official text of the PLFSS, the new measures will help create a more cohesive system that entails:
- Rapid access for patients to drugs in advance (although upstream of common law) in a secure environment for use outside the usual
- Marketing authorization and paid for by health insurance,
- Readability for prescribers,
- Attractiveness and predictability for companies with innovative drugs,
- Financial sustainability for health insurance
The upheaval of the existing system aims to “simplify demands”, as well as “harmonize procedures”, according to the document.
The processes currently in use have been deemed overly complex in the past, leading French MPs to decide on replacing the schemes with newer, more streamlined systems. On top of the complexity, MPs cited a lack of readability for all stakeholders as a reason for the reforms.