Interim-Report on EMA's Clinical Study Data Proof-of-Concept Pilot

The European Medicines Agency (EMA), together with the Heads of Medicines Agencies (HMA) and the Big Data Steering Group (BDSG), has been conducting a pilot study to explore the benefits of visualizing and analyzing data from clinical studies in support of the scientific assessment of medicinal products. The pilot has initially been designed with an estimated duration of two to three years (from September 2022). An interim report is now available analyzing patient-level clinical study data submitted voluntarily by marketing authorization holders (MAHs).

Submission of Structured Clinical Trial Data to EMA

The EMA launched the pilot project in 2022, which was originally referred to as the ‘Raw Data’ pilot project. As part of the Proof-of-Concept (PoC) pilot, selected applicants can submit structured clinical trial data to the EMA as part of their initial applications and post-authorization procedures. Clinical trial data refers to individual patient data from clinical trials. This includes

• Clinical laboratory results
• Images
• Medical records

Currently, applicants submit data in an aggregated format as clinical summaries or as individual patient data in PDF lists. This can complicate data analysis and slow down the evaluation process. In contrast, the clinical trial data in the pilot is stored in an electronically structured format. This enables the responsible authorities to visualize and analyse the data more easily. The aim of the pilot project is to obtain information on whether the use of structured clinical trial data can help to speed up and improve the drug assessment process. The aim is also to ensure that patients have faster and better informed access to innovative medicines. Based on the interim analysis of the pilot project, the EMA has published the following additional documents on the project website:

• Information about the clinical study data proof-of-concept pilot
• Application of EMA's transparency principles
• A questions and answers document
• A participation letter to confirm pilot participation for a specific regulatory procedure
• A cover letter for pilot participants to attach to their data packages
• EMA's data protection notice
• Records of data processing activity

The report covers the time period from the inclusion of the first regulatory procedure in the pilot (September 2022) up to December 2023. However, EMA and HMA decided to extend the duration of this pilot based on the lessons learnt so far. As a result, interested companies can still apply to the EMA to participate in the project.

More information is available at EMA's Use of clinical study data in medicine evaluation website.