Clinical trial (Regulation (EU) No 536/2014)

A clinical trial is a study conducted in humans to discover or verify the clinical or pharmacological effects of one or more investigational medicinal products, to identify their adverse reactions or to study their absorption, distribution, metabolism and excretion, with the aim of establishing their safety or efficacy. Regulation (EU) No 536/2014 lays down the applicable regime, directly applicable in all Member States since 31 January 2022, replacing Directive 2001/20/EC. It distinguishes the clinical trial, which requires authorisation, from the non-interventional study, which falls outside its scope.

The authorisation procedure

The sponsor submits a single application through the EU portal, the Clinical Trials Information System, designating the Member States concerned. The assessment is divided into two parts. The first, conducted in a coordinated manner by a reporting Member State, covers the scientific aspects, in particular the benefit-risk balance and the quality of the investigational medicinal product. The second falls to each Member State and concerns informed consent, compensation of participants, recruitment and the suitability of trial sites. In France, it involves the ANSM and an ethics committee (comité de protection des personnes). The trial may only begin in a Member State once that State has issued its decision.

Transparency and its impact on novelty

The Regulation lays down a principle of transparency. Information entered in the EU database is in principle public, subject to the protection of personal data and commercially confidential information, and the sponsor must publish a summary of the results within one year of the end of the trial. This publicity is of direct concern to patent law, since a protocol made available to the public forms part of the state of the art and may destroy the novelty of a dosage regimen, a patient population or a combination. Coordinating the timetable for patent filings with that of regulatory publications is therefore a matter of strategy.

Interplay with patent exceptions

Conducting a trial on a protected substance may constitute an act of exploitation for patent purposes. Trials required to obtain a marketing authorisation benefit from the Bolar exemption, and trials relating to the subject matter of the invention for research purposes may fall under the experimental use exception. The boundary between these exceptions and trials conducted for a primarily commercial purpose remains a point of attention, particularly for active ingredient suppliers and subcontractors.

Key points

Clinical trials are governed by a unified European regime, with a single application submitted through a single portal and an assessment shared between a reporting Member State and each Member State concerned. Their increased transparency makes them a potential source of prior art. Patent applications covering dosage regimens, combinations or target populations should be filed before the protocol is published online, and a reasoned request should be made to defer publication of commercially sensitive information.

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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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