Compassionate access (accès compassionnel)

Compassionate access allows the use of a medicinal product that is not authorised, or not authorised in the indication concerned, for patients suffering from a serious, rare or disabling disease who have no appropriate treatment, where the company is not pursuing commercial development in that indication. It falls within the remit of the ANSM and was introduced by the reform of derogatory access that entered into force on 1 July 2021, which replaced the named-patient temporary authorisation for use and the temporary recommendation for use. It differs from early access, which is reserved for presumptively innovative medicinal products whose development in the indication is ongoing.

The two forms of compassionate access

The compassionate access authorisation is granted on a named-patient basis. The ANSM issues it at the request of the prescribing physician, for a specific patient, where the efficacy and safety of the medicinal product are presumed on the basis of the available data. The compassionate prescription framework is collective. The ANSM establishes it on its own initiative for a medicinal product that already holds a marketing authorisation in other indications, in order to secure widespread off-label prescribing, and it comes with a patient monitoring protocol.

Conditions and regime

Both mechanisms require a serious, rare or disabling disease, the absence of appropriate treatment and a favourable presumption as to the benefit-risk balance. Where the company embarks on commercial development in the indication, it is expected to move to early access within the time limits set by the legislation.

Intellectual property issues

The compassionate prescription framework often concerns a new indication of a known molecule. That indication may be covered by a second medical use patent held by a third party, and the regulatory supervision of prescribing confers no authorisation whatsoever under patent law. In practice, the infringement action is then directed at the operator that manufactures or supplies the medicinal product for the protected use. Nor does compassionate access give rise to any regulatory data protection, since it is not a marketing authorisation.

Key points

Compassionate access meets an individual need or frames an established off-label use, with no development purpose. It should be checked whether the company plans development in the indication, in which case early access is the appropriate route. For the holder of an indication patent, a compassionate prescription framework covering the protected indication calls for close scrutiny of the supplier’s acts. For the supplier, regulatory authorisation never removes the need for a freedom to operate analysis.

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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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