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:
    • ANSM assesses presumed efficacy and safety.
    • HAS decides on granting the authorization.
  • 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

  1. HAS – What you need to know about Early Access
  2. HAS – Two-year assessment of the scheme
  3. HAS – Evaluation doctrine
  4. ANSM – Early Access role
  5. ANSM – 2023 Annual Report