How can Cevidra manage early access for your medicinal products to French market before their commercial launch?

#PreApprovalPrograms #CompassionateUse #EarlyAccess

Cevidra offers services for compassionate use and early access programs for your medicinal products in France before the commercial launch.

Compassionate Use
Early Access Programs

Compassionate Use

What is a compassionate use?

Previously named nATU (nominative Temporary Authorization), compassionate use now replaces the former scheme and is applicable to medicinal products not intended to be marketed with a Marketing Authorization in the concerned indication although the product meets a therapeutic need.

Compassionate use authorisations (nominative ATUs) are subject to access criteria. Treatment initiation is at the initiative of Health Care Professionals and is dedicated to a given patient (named patient medicine). Since the reform in July 2021, manufacturers must undertake to apply for early access if they are considering clinical and commercial development in a given indication.

Access to these medicines is under the ANSM’s responsibility. If access is granted, products are reimbursed by the Social Security at no cost for the patient or the hospital.

How to provide products on a named patient basis?

Cevidra acts as local representative in France for companies located outside the country. In this context, Cevidra offers these companies the supply chain needed to deliver products under compassionate access to French patients.

After signing a contract with product “owner”, Cevidra’s role is to facilitate access to these products for hospitals, offering:

  • French speakers

  • Importation of products into France

  • Qualified storage room (ambient and cold room)

  • Dematerialized ordering process

  • Product delivery in 24/48 hours

  • Invoicing & cash collection

What are the obligations before delivering products under compassionate use?

As the access to these treatments is regulated by the ANSM, delivery to hospitals is subject to a pharmaceutical control of the product access form. This assessment is based on:

  • Verification of the validity period

  • Patient anonymisation

  • Product identification

  • Approval of the request by the French Health Agency (ANSM)

Cevidra has all the processes and the operational procedures in place to ensure the full compliance of these activities with regulations in force.

How to manage Medical Information requests for these products?

For products under compassionate use, medical information requests can raised by hospitals. Cevidra ensures the reception and treatment of all MedInfo requests and liaises with the Marketing Authorization Holder (MAH) to provide the adequate answer to the hospital in a timely manner.

Are the products subject to public tenders?

Products under compassionate use are delivered to hospitals only. Some of these products can be included in public tenders for the hospital reserve. They are usually part of a specific catalogue which groups together all the products with compassionate use status. Demands for public tenders are periodically published and renewed, which requires continuous monitoring to avoid missing an offer.

Cevidra has a strong expertise in hospital reserve that are subject to public tenders. Cevidra closely monitors all the tender publications related to products on its portfolio and liaises with the MAH to validate the participation to each tender.

Early Access Program (EAP)

What is an early access?

Early access (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 scheme 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.

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 scheme 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