The ATU (Temporary Authorization for Use) was the former French framework (in place until July 2021) that allowed early access to medicines. It has been replaced by the AAP (Early Access Authorization) and AAC (Compassionate Access Authorization), which harmonizes procedures, strengthens evidence requirements, and clarifies pricing rules.
Key Differences ATU vs AAP vs AAC
| Aspect | ATU (until June 2021) | AAP (since July 2021) | AAC (Compassionate Access Authorization) |
|---|---|---|---|
| Legal basis | ATU = Autorisation Temporaire d’Utilisation | AAP = Autorisation d’Accès Précoce | AAC = Autorisation d’Accès Compassionnel |
| Regulator | Exclusively ANSM | ANSM (efficacy & safety) + HAS (final decision & assessment) | Exclusively ANSM |
| Types | Nominative ATU (one patient) and Cohort ATU (group) | Unified single framework (no nominative/cohort distinction) | Always nominative, for individual patients |
| Eligibility | Serious/rare disease, no alternative, positive benefit-risk, ongoing clinical data | Serious/rare/disabling disease, no alternative, treatment cannot be delayed, strong presumption of efficacy & safety, innovation required | Serious/rare disease, no alternative, either no presumption of innovation required, where product is not intended for commercial development or before an AAP |
| Duration | Temporary, renewable | 1 year, renewable | Case-by-case, renewable if no commercialization plan, or 12–18 months when used as a pre-AAP program |
| Evidence obligations | Limited, heterogeneous data collection | Mandatory PUT-RD (protocol for therapeutic use & data collection) → structured RWE | Potentially PUT-SP (Protocol of therapeutic use & patient follow-up), otherwise mainly pharmacovigilance reporting |
| Pricing | Manufacturer set free price, clawback rules unclear | Manufacturer free price; fully reimbursed; clawback mechanism if final negotiated price < AAP price | Manufacturer free price; fully reimbursed; clawback mechanism if final negotiated price < AAP price |
| Process | Initiated by physician (nominative) or company (cohort) | Centralized application via SESAME platform, evaluation ≤90 days | Physician request via e-Saturne portal, authorization by ANSM |
| Transition | Ended July 2021 | Successor framework in force since July 2021 | Still in force (parallel system to AAP) |
Why the Change?
- To simplify and unify early access procedures.
- To align clinical access with European best practices.
- To improve data collection (RWE) for pricing/reimbursement negotiations.
- To provide greater transparency and predictability for manufacturers and healthcare providers.
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