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).
early-access-programs-schema

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.

Distribution
Sales-process

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