Early Access Programs in France (ex ATU)

#PreApprovalPrograms #CompassionateUse #EarlyAccess

Cevidra offers services for Early Access Programs in France for your medicinal products in France before the commercial launch.

What is an early access program?

Early access programs (previously named Cohort ATU) is a scheme that allows patients who have reached a therapeutic impasse to benefit, on an exceptional and temporary basis, from certain medicinal products that are not authorised in a specific therapeutic indication. It is a Temporary Authorization for Use.

The early access authorisation (also called expanded access) is granted by the HAS on a derogatory basis and for a renewable period of one year. It applies either to a medicinal product:

Which does not yet have a Marketing Authorisation for the specific indication but for which the company undertakes to submit it within 2 years, called pre-MA early access (AP1);
Or to a medicinal product which already has a Marketing Authorisation in the indication but has not yet been accepted for reimbursement by Social Security, called post-MA early access (AP2).

The early access program is intended for innovative products and access is subject to various criteria, as defined in Article L.5121-12 of the Public Health Code:

The medicinal product must be intended for the treatment of serious, rare or debilitating diseases
There is no suitable treatment available
The implementation of the treatment cannot be delayed
The medicinal product is presumed to be innovative, in particular with regard to a possible clinically relevant comparator

How to apply for an early access?

Early Access (EA) applications are made to the HAS through a dematerialized procedure, at the initiative of the pharmaceutical company. The evaluation period can last 3 months after the validation of the administrative application. At the time of the request, the pharmaceutical company has to appoint a local representative, named “exploitant”.

Cevidra has a strong expertise in early access programs and can support you in the following steps:

Clinical Data due diligence to assess eligibility to an EA
Definition of the EA strategy and timelines
Review of the Protocol of Therapeutic Use (PTU)
Labelling preparation
Support in early access request: Application preparation, Pre-submission meeting preparation, Submission of the application through the e-platform (Sesame), Follow-up on the exchanges with the HAS

How to manage an early access?

The management of early access starts when the EA is granted by the HAS, and requires the involvement of the exploitant at different levels.

Product Compliance

Products must be in compliance with the EA dossier and more specifically with the approved labelling. Batch release steps must ensure this compliance.

Data Collection

Data are collected throughout early access, through paper forms or dematerialized tools to facilitate data collection from doctors and patients.

Medical Information

MedInfo requests from Health Care Profesionnals or patients are followed-up by the exploitant in collaboration with the MAH.

Pharmacovigilance

A local Responsible Person for PV is appointed to ensure the reception of adverse event declaration/treatment also the preparation and submission of periodic safety reports to the Health Authority.

How to distribute products under early access?

Once early access is granted, products should be made available within 2 months. Products can be delivered to hospitals when the treatment access form has been fully validated since certain inclusion criteria must be fulfilled, as defined in the PTU.

Early Access products are for hospital use only. The ordering process may require the signature of a price offer or a specific contract. This step is a prerequisite to the reception of orders.

Cevidra has its own logistics platform to offer product storage, stock monitoring, delivery from its warehouse, order management, invoicing and cash collection.

How is the early access program funded ?

The granting of an early access by the HAS opens the rights to health insurance coverage. This means that a selling price is freely defined by the company (MAH), and that this price is fully reimbursed to hospitals.

Following the reform of Early Access Programs in July 2021, two systems of rebates have been implemented and correspond to fees paid by the company (MAH):

An annual rebate based on the turnover with a progressive rate
A rebate paid retrospectively at the end of the scheme which corresponds to a payback if the final price of the commercial product is below the price defined during the EA (overpayments are refunded)

Key takeaways

Early Access Programs

Early Access in France is very attractive but complex, it allows early earning even if rebates must be taken into consideration

Exploitant

The appointment of an exploitant (local partner) is mandatory in France

How Cevidra can help you?

Cevidra is a high added value partner to run your early access program

FAQ

What is the role of HAS in early access programs in France?2025-10-29T17:13:31+02:00

What is the role of HAS in early access programs in France?

HAS assesses and grants early access authorizations for innovative medicines, ensuring timely patient access. (Haute Autorité de Santé)

What is the Early Access Authorization (AAP) in France?2025-10-30T15:58:06+02:00

What is the Early Access Authorization (AAP) in France?

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.

How do EAPs benefit pharmaceutical companies?2025-10-29T12:57:53+02:00

How do EAPs benefit pharmaceutical companies?

EAPs can provide real-world data, expose physicians to new drugs, and assist in regulatory approval processes.

What is the difference between the ATU and AAP programs in France?2026-02-09T19:27:13+02:00

What is the difference between the ATU and AAP programs in France?

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.

Sources

HAS – Reform of Early Access & Compassionate Use (AAP replacing ATU, July 2021)
HAS – AAP Evaluation Doctrine
HAS – Two-year assessment of AAP
ANSM – Référentiel des spécialités en accès dérogatoire

What is the Early Access Program (AAP) in France?2025-10-29T18:07:07+02:00

What is the Early Access Program (AAP) in France?

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

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