FAQ
- All
- AP1 & AP2
- Compassionate Use
- Early Access
- Europe Market Insights
- Expanded Access
- France Market Insights
France does not have an FDA. The equivalent authority of the FDA in France is the ANSM (Agence nationale de sécurité du médicament et des produits de santé), which regulates medicines and health products.
Sources
- ANSM – https://ansm.sante.fr
AP1 (Accès Précoce 1) is the pre-marketing authorization pathway under France’s Early Access Program (AAP). It allows innovative medicines without marketing authorization (MAA) yet to be used for serious, rare, or disabling conditions when no satisfactory alternative exists within a cohort framework.
Key takeaways – AP1 (pre-MAA Early Access)
- Purpose: Enables early patient access to innovative therapies still awaiting MAA.
- Mechanism: As part of the broader AAP framework (since July 2021), AP1 is solely for products that do not yet have MAA, unlike AP2 which applies post-MAA.
- Conditions: Must fulfill all the following criteria:
- Presumed favorable benefit‑risk profile (i.e. Ph2/3 results)
- Treatment for serious, rare, or disabling disease
- No appropriate alternative available
- Treatment cannot be delayed
- Presumed innovation relative to clinically relevant comparators
Sources
- Cevidra (Early Access Programs in France) – AP1 vs AP2 distinction
https://www.cevidra.com/early-access-programs-in-france/
AP2 is part of France’s Early Access Program (AAP). It applies to medicines that already have marketing authorization (MAA) but are still awaiting reimbursement approval and/or completion of final price negotiations. It ensures patients within a cohort framework can access the treatment in the meantime, with obligations for real-world data collection and temporary pricing rules.
Differences between AP1 and AP2 in France:
- AP1: Early Access before marketing authorization (MAA) → allows use of an innovative medicine still under regulatory review.
- AP2: Early Access after marketing authorization (MAA) but are still awaiting reimbursement approval and/or completion of final price negotiations → ensures patient access during the pricing/HTA process.
Both require: serious/rare/disabling disease, no alternative, urgent need, strong presumption of efficacy/safety, and real-world data collection (PUT-RD).
AAP is a French program allowing access to innovative medicines before marketing authorization, under specific conditions, with decisions made by HAS following ANSM’s opinion. (Haute Autorité de Santé).
Discover more about AAP : full definition here.
The Early Access Authorization (AAP) is a regulatory framework that enables patients with serious, rare, or disabling diseases to access innovative medicines before marketing authorization (MAA) or reimbursement. It addresses unmet medical needs when no satisfactory alternative exists.
Key takeaways
- Regulators:
- Eligibility Criteria:
- Serious, rare, or disabling condition.
- No appropriate alternative.
- Treatment cannot be delayed.
- Strong presumption of efficacy/safety.
- Demonstrated innovation.
- Duration & Obligations:
- Granted for 1 year, renewable.
- Mandatory Protocol of Therapeutic Use (PUT-RD) to collect real-world data.
- Market & Access Impact:
- Manufacturers set a free price during AAP.
- A clawback mechanism applies after reimbursement negotiations including yearly provisions.
- Provides early patient access and generates real-world evidence (RWE) for pricing and reimbursement dossiers.
- Performance:
- Since July 2021: >100,000 patients treated.
- Approval rate around 78%.
- Median evaluation time: ~78 days (below the 90-day limit).
Official Sources
- HAS – What you need to know about Early Access
- HAS – Two-year assessment of the scheme
- HAS – Evaluation doctrine
- ANSM – Early Access role
- ANSM – 2023 Annual Report
The main regulatory authority for pharmaceuticals in France is the ANSM (Agence nationale de sécurité du médicament et des produits de santé). It oversees drug approval, safety, pharmacovigilance, and market supervision. For health technology assessment and reimbursement decisions, the competent body is the HAS (Haute Autorité de Santé).
Pricing negotiations are handled by the CEPS (Comité économique des produits de santé).
The URSSAF oversees social security contributions and employer obligations, but it is not directly involved in the regulatory pathway of a medicinal product.
Sources
Role of an “Exploitant” in France: In the French pharmaceutical system, the “exploitant” is the legal entity responsible for the commercialization and distribution of a medicinal product once it is authorized.
Key Points
- Regulatory role:
- Holds the responsibility vis-à-vis ANSM (French Medicines Agency) for the product placed on the French market.
- Ensures compliance with pharmacovigilance, distribution, sales, advertising rules, financial and regulatory obligations.
- Operational role:
- May differ from the MAH (Marketing Authorization Holder) if the MAH is not established in France.
- The exploitant is the local interface between the manufacturer/MAH, the French authorities and administrative bodies.
- Legal basis:
- Defined under French Public Health Code (CSP, Article R.5124-2).
Sources
- ANSM – Responsibilities of the Exploitant
https://ansm.sante.fr/activites/medicaments/exploitants-et-distributeurs - Code de la Santé Publique – Article R.5124-2
https://www.legifrance.gouv.fr/codes/article_lc/LEGIARTI000006690421 - Leem – The role of the Exploitant in France
https://www.leem.org/
HAS assesses and grants early access authorizations for innovative medicines, ensuring timely patient access. (Haute Autorité de Santé)
The ANSM (French National Agency for Medicines and Health Products Safety) is responsible for compassionate use authorizations. (Haute Autorité de Santé)
