REFINEMENT OF CRITERIA FOR INNOVATIVENESS IN THE AUTORISATION TEMPORAIRE D’UTILISATION (ATU) SCHEME
The French law for temporary authorization for use (ATU) for early access to medicines has been active since 1992.
The ATU scheme provides early access to medicines and carries several advantages when preparing launch of a new treatment:
- Access to medicines for patients with immediate unmet needs.
- Enhanced patient monitoring and evidence generation for French clinical settings.
- Opportunities for physicians to familiarize themselves with new innovative treatments.
In July 2021, the HAS took on the responsibilities of implementing the ATU schemes.
In May 2022, the HAS and ANSM concluded a performance review of the early access schemes and based on this review, updates to the criteria for presumption of innovation were made.
Applicants for early access to innovative medicines will now need to show context to current therapeutic practice and unmet clinical need in addition to existing criteria about new modalities with substantial clinical benefits.
After May 2022 and announced in 2021, two additional criteria for ‘presumption of innovativeness’ have been introduced – (1) requirement to show clinical benefit in the context of currently available therapies and (2) demonstrate fulfillment of an unmet medical need. These may require additional attention from manufacturers planning AAP applications in the future.
Advantages for manufacturers
- – Associated expedited HTA assessment by HAS
– Additional evidence generation supporting HTA in France
– Early familiarization in the health system supporting access in a competitive setting
Disadvantages for manufacturers
- – Potential delays and additional costs for inclusion of French clinical trial sites to fulfill innovativeness criteria related to local therapeutic context and to show strong unmet need
Challenges for manufacturers
- – Planning an AAP will require plans for comparative assessments to establish benefit over currently available therapy, a potential added risk to successful launch of a new drug.
- – Increased investments may be needed for executing the AAP.
- – Lack of appropriate comparators for non-pharmaceutical treatment options.
- – Introduction of selection bias when choosing appropriate comparator treatments.
- – Physicians will need to be convinced of the efficacy of new innovative treatment options over long-established therapy regimens.
- – A high unmet clinical need for innovative treatments must be demonstrated for approval.
AAP = Autorisation d’accès précoce; ANSM = Agence nationale de sécurité du médicament et des produits de santé; HAS = Haute Autorité de Santé
References: https://www.legifrance.gouv.fr/jorf/article_jo/JORFARTI000042665373; https://ansm.sante.fr/actualites/autorisation-dacces-precoce-aux-medicaments-un-premier-bilan-positif-et-des-principes-devaluation-affines; https://www.insideeulifesciences.com/2021/07/06/new-early-access-and-off-label-use-rules-in-france/