Early access (accès précoce)

Early access is the mechanism that allows, on an exceptional and temporary basis, the use and reimbursement by the national health insurance system of a presumptively innovative medicinal product intended to treat a serious, rare or disabling disease, before its marketing authorisation in the indication concerned or before its inclusion on the reimbursement list. Introduced by the Social Security Financing Act for 2021, it entered into force on 1 July 2021 and, together with compassionate access, replaced the regime of temporary authorisations for use. The authorisation is granted by the French National Authority for Health, at the request of the pharmaceutical company, after a binding opinion from the ANSM where the medicinal product is not yet authorised in the indication.

Conditions for authorisation

The medicinal product must meet several cumulative conditions, assessed indication by indication.

• the disease is serious, rare or disabling
• no appropriate treatment exists
• initiation of treatment cannot be postponed
• the efficacy and safety of the medicinal product are strongly presumed on the basis of clinical trial results
• the medicinal product is presumed to be innovative, in particular in comparison with a clinically relevant comparator.

The company further undertakes to file, within a set period, an application for marketing authorisation where early access precedes authorisation, or an application for inclusion on the reimbursement list where the product is already authorised. The authorisation is granted for a limited, renewable period and comes with a therapeutic use and data collection protocol.

Financial regime

During early access, the company freely sets the amount of the indemnity it receives, which is paid by the national health insurance system. That amount remains provisional. Once the price or tariff is set, the company pays back, in the form of a rebate, the difference between the indemnity received and the price finally adopted, and rebates based on turnover apply during the period.

Interplay with patents and regulatory protection

Early access is not a marketing authorisation. It therefore does not start the period of regulatory data protection, which runs from the first authorisation in the Union, and it is not the authorisation required by Regulation (EC) No 469/2009 for the grant of a supplementary protection certificate. Supplying the medicinal product for remuneration under this scheme is, by contrast, an act of commercial exploitation. It falls outside the Bolar exemption, which is limited to the studies and trials needed for authorisation, and may therefore amount to infringement where a third-party patent covers the product.

Key points

Early access opens a market before authorisation or before reimbursement, under the supervision of the French National Authority for Health and the ANSM. Its conditions should be checked indication by indication and the price negotiation prepared in parallel, since the indemnity received will be subject to repayment. For the company, the early access application is also the moment to check its freedom to operate, because the Bolar exemption ceases to protect it as soon as the product is supplied to patients.

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This glossary entry is general in scope and does not constitute legal advice.

Dhenne Avocats acts in pharmaceutical patent disputes and on the medicines regulatory questions that accompany them, from the marketing authorisation to the supplementary protection certificate.

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