As of April 2025, France’s Reimbursed Early Access Program (AAP, formerly ATU) continues to stand as one of Europe’s most advanced pathways for granting early, publicly funded access to life-saving therapies, even before full marketing authorisation or formal reimbursement.
For biopharmaceutical companies, particularly in oncology, rare disease and gene therapy, the system offers not just patient access, but a structured economic and scientific bridge to long-term reimbursement.
Historically referred to as the ATU (Autorisation Temporaire d’Utilisation) system, France revised its approach in 2021 under the Social Security Financing Law to form two new routes:
- AAP (Accès Précoce) – Early Access, reimbursed, HAS-evaluated access for medicines prior to marketing authorisation (MA) or in an unapproved indication.
- AAPC (Accès Compassionnel) – Compassionate Access, for products not intended for commercialisation or broader use.
These programs are coordinated by:
- HAS (Haute Autorité de Santé) for clinical and medico-economic evaluation
- ANSM (Agence nationale de sécurité du médicament) for safety oversight
- CNAM (Caisse Nationale d’Assurance Maladie) for temporary reimbursement agreement and price
Once accepted into the AAP, the product is:
- Reimbursed at 100% under French national insurance;
- Priced via maximum compensation ceilings (montants de remboursement maximaux), published and updated semi-annually;
- Subject to data collection and RWE obligations to inform future CEPS pricing negotiations.
As of April 2025, 366 products and 236 unique active substances (INNs) have received early reimbursement—a 10% increase since September 2024. These include cutting-edge gene therapies, immuno-oncology agents, and orphan drugs addressing ultra-rare diseases.
Notable new entries include:
| Product | INN | Indication (Simplified) |
| Vyjuvek | beremagene geperpavec | Dystrophic epidermolysis bullosa |
| Elrexfio | elranatamab | Refractory multiple myeloma |
| Livmarli | maralixibat | Progressive intrahepatic cholestasis |
| Jakavi | ruxolitinib | Graft-versus-host disease |
| Elahere | mirvetuximab soravtansine | Ovarian and fallopian tube cancers |
| Winrevair | sotatercept | Pulmonary arterial hypertension |
| Vylov | zolbetuximab | HER2-negative advanced gastric cancer |
These additions underscore the scientific and economic openness of the French system toward:
- First-in-class mechanisms
- Ultra-orphan populations
- Real-world validated endpoints
- Drugs priced in the millions, such as PTC Therapeutics’ Upstaza (€3.5 million)
Unlike some EU systems where early access is limited to nominal pricing or unfunded“named patient use” France enables full public reimbursement with transparent upper-limit pricing, often in line with expected future list prices.
Key principles include:
- Temporary, capped price (montant maximal de remboursement) does not lock in CEPS pricing.
- Companies are obliged to submit a final dossier for CEPS price negotiation and Transparency Committee (CT) assessment after early access ends.
- Volume and budget impact are reviewed retrospectively, and overcompensation may lead to paybacks (remboursements à posteriori) to CNAM.
According to the French Cour des Comptes (Court of Audit), in 2023 alone, over €1.4 billion was spent through early access mechanisms, but offset by reduced hospitalisations and emergency interventions, especially in oncology and CAR-T applications. (Ref: Cour des Comptes, Rapport sur les comptes de la sécurité sociale 2024).
Entry into AAP is not merely a shortcut to market. It demands a scientific and real-world commitment:
- Post-entry, manufacturers must submit data on safety, usage, early efficacy signals and economic value.
- HAS uses this data to assess service médical rendu (SMR) and amélioration du service médical rendu (ASMR) – key criteria for permanent reimbursement.
- Hospitals and prescribers are obliged to report treatment outcomes, contributing to a national RWE evidence base.
This aligns with France’s 2023–2027 National Health Data Strategy, which emphasises using health data (via SNDS and EDS) to power both HTA and real-time clinical improvement.
While other EU countries such as Germany (via NUB), Italy (Law 648/96) and the UK (NHS EAMS) have early access frameworks, France’s AAP is currently the most transparent, high-value and structurally integrated into HTA and payer planning.
For biotechs with gene therapies, ultra-rare indications or post-Phase II oncology products, this creates:
- A risk-sharing pre-launch model
- A pricing data point for other EU negotiations
- A de-risking tool for investors
France’s Early Access Program is no longer a niche or exceptional route. It has become a core element of the French reimbursement ecosystem, particularly for advanced biologics and cell/gene therapies. With 366 products covered as of April 2025 and millions allocated to rare and complex treatments, the message is clear: If you have an innovative, high-impact therapy, France offers a real-world, reimbursed, and scientifically aligned route to patients and payers.
🧾 Further Reading:
- HAS Early Access Portal
- ANSM ATU/AAP Procedures
- Cour des Comptes Social Security Expenditure Reports
- French Social Security Code: Articles L5121-12 & R5121-70:https://www.legifrance.gouv.fr/codes/article_lc/LEGIARTI000045166651https://www.legifrance.gouv.fr/codes/article_lc/LEGIARTI000048703237