EMA launches pilot project on the use of raw data from clinical trials for regulatory decision-making


On 12 July, the European Medicines Agency (EMA) announced the launch of a pilot project to assess whether the analysis of ‘raw data’ from clinical trials by regulatory authorities improves the evaluation of applications for new medicines, or for post-authorisation applications. The pilot will also explore practical aspects of the submission and analysis of such data.

Raw data consists of individual patient data from clinical studies, stored in a structured electronic format that is directly accessible for analysis and visualisation. Examples of raw data include clinical laboratory results, imaging data, and patient medical charts, which are collected during the clinical research process. Currently, raw data is not routinely provided by organisations applying for the authorisation of new medicines, or applying to change the terms of an existing marketing authorisation.

The Human Medicines Committee (CHMP) is responsible for evaluating medicines that are submitted for marketing approval to the EMA. At the moment, during the evaluation process the CHMP receives patient data in an aggregated format, submitted by the applicant after statistical processing as clinical summaries or PDF listings. The CHMP scrutinises these summaries as part of the scientific evaluation of the benefits and risks of new medicines. This process typically results in several rounds of questions, in which the Committee may ask the applicant for methodological clarifications, re-analysis of data, or additional data. Although it is not part of the routine process, raw data have been requested by the CHMP on several occasions in the past when it was considered that it would be helpful in the evaluation of a new medicine.

The raw data project stems from one of the ten priority recommendations issued by the joint Big Data Task Force of EMA and the Heads of Medicines Agencies (HMA) in 2020, which highlighted the need to strengthen the regulatory capability to analyse data collected at individual patient level to better inform regulatory decision making. There are several potential benefits the analysis of raw data might bring, including faster evaluation through fewer questions being put to applicants and a better definition of the target treatment population. Raw data analysis may therefore enable faster and better access to new medicines for patients. To help guide the pilot project, an Advisory Group was established in 2021, consisting of representatives of EMA committees and working parties, including Alzheimer Europe, which is a member of the Patients and Consumers’ Working Party (PCWP).

Pilot participation is open to applicants that are about to submit marketing authorisation applications or post-authorisation applications to EMA. If selected, they will include raw data as part of their submissions. The pilot is expected to last up to two years and will include approximately ten regulatory procedures submitted to EMA from September 2022. The pilot will fully comply with data protection legislation requirements. Upon the completion of the pilot, EMA will organise a workshop with relevant stakeholders to discuss the learnings and will also publish a summary report.

More information on the pilot’s objectives and on the terms of participation is available in the description of the pilot to industry, which can be found here.