EMA – Clinical study data proof-of-concept pilot: updates and extension

As set out in the interim report of the pilot:

In 2020, the joint Big Data Task Force of the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) published ten priority recommendations for the European Medicines Regulatory Network (EMRN) to unlock the potential of ‘big data’. A key action involves enhancing the EMRN’s capability to analyse big data including ‘raw data’ – notably individual patient data from clinical studies in structured format from which statistical analyses and clinical insights are derived.

While regulators can already request clinical study data from applicants/marketing authorisation holders (MAHs) as per the current European Union’s pharmaceutical legislation, the European Commission’s proposal for a reform of the legislation foresees a systematic submission of clinical study data at the time of filing of initial marketing authorisation applications. Overseen by the HMA-EMA Big Data Steering Group (BDSG) and the Committee for Medicinal Products for Human Use (CHMP), the EMRN is conducting [from September 2022] a Proof-of-Concept (PoC) pilot (hereinafter “the pilot”) to explore the benefits of visualising and analysing data from clinical studies in support of the scientific assessment of medicinal products. In addition to the regulatory benefit when clinical study data is available at the time of submission (i.e. faster assessment and enhanced decision-making), the pilot aims to determine the EMRN’s capacity and capability requirements, a target operating model and technical prerequisites regarding a systematic submission of clinical study data in the regulatory setting in the future.

Regarding the first results of the pilot, the EMA decided to extend its duration. This was the occasion to publish new and/or updated guidance or information documents for possible applicants:

• Information about the clinical study data proof-of-concept pilot for industry
• Questions and answers about the clinical study data proof-of-concept pilot for industry
• Application of EMA’s transparency principles to the clinical study data proof-of-concept pilot
• Presentation – 3.1 Clinical study data pilot interim results – F Petavy

More information on this pilot and the use of clinical study data in medicine evaluation on the EMA Website.