Use of clinical study data in medicine evaluation

Clinical study data refer to individual patient data from clinical studies. The European Medicines Agency (EMA) aims to assess whether using clinical study data can help speed up and improve the medicine-evaluation process.

For more information on how EMA works with big data, see:

• Data in regulation: Big data and other sources

Pilot on using clinical study data in medicine evaluation

Through a proof-of-concept pilot, selected applicants can submit clinical study data to EMA as part of their initial and post-authorisation marketing authorisation applications.

Clinical study data refer to individual patient data from clinical trials. These include:

• Clinical laboratory results
• Imaging data
• Patient medical charts

Currently, applicants are submitting data in an aggregated format as clinical summaries or as individual patient data in PDF listings. This can hinder data analysis and slow down the evaluation process.

In contrast, clinical study data are stored in electronic structured format. This enables regulators to more easily visualise and analyse the data if needed.

The pilot aims to assess whether using clinical study data can help speed up and improve the medicine-evaluation process. The goal of this is to allow patients faster and better informed access to innovative medicines.

EMA launched the pilot in July 2022. It was initially referred to as the 'raw data' pilot.

The pilot is a key activity under the priority recommendations of the former HMA-EMA Big Data Task Force, which has been replaced by the HMA-EMA Big Data Steering Group. It refers to the priority of building network capability to analyse data.

For any queries and to apply to take part in the pilot, write to [e-mail].

Pilot extension

EMA and HMA decided to extend the duration of this pilot based on the insights it generated so far. 

As a result, expressions of interest will continue to be accepted until further notice.

For more information about the pilot, see:

Information about the clinical study data proof-of-concept pilot for industry

Reference Number: EMA/174598/2022

Application of EMA’s transparency principles to the clinical study data proof-of-concept pilot

Reference Number: EMA/949891/2022

Presentation - 3.1 Clinical study data pilot interim results - F Petavy

Support for pharmaceutical companies

Further information is available to support pharmaceutical companies with their participation in EMA's clinical study data pilot.

The documents include:

• a questionsandanswers document on the clinical study data pilot;
• a participation letter to confirm pilot participation for a specific regulatory procedure;
• a cover letterfor pilot participants to attach to their data packages.

Questions and answers about the clinical study data proof-of-concept pilot for industry

Reference Number: EMA/658116/2022

Pilot participation letter: Clinical study data proof-of-concept

Reference Number: EMA/659352/2022

Data submission cover letter template

Reference Number: EMA/658203/2022

Interim report on using clinical study data in medicine evaluation

An interim report is available on the European medicines regulatory network's experience with analysing patient-level clinical study data submitted voluntarily by marketing authorisation applicants and holders.

This report: 

• offers preliminary insights into the regulatory benefits of including clinical study data in the marketing authorisation application dossier, potentially leading to earlier opinion and authorisation of medicines;
• examines the operational and technical practicalities for future systematic submission of clinical study data as part of the marketing authorisation dossier.

It includes experience gained with submission and analysis of patient-level data from clinical studies conducted between September 2022 and December 2023.

Proof-of-concept pilot on using data from clinical studies in medicines evaluation: Interim report

Data protection

For information on data protection in the clinical study data proof-of-concept pilot, see:

European Medicines Agency’s data protection notice for the clinical study data proof-of-concept pilot

Reference Number: EMA/716436/2022

Records of data processing activity for the clinical study data proof-of-concept pilot

Reference Number: EMA/716437/2022