EMA has launched a pilot project to assess whether the analysis of ‘raw data’ from clinical trials by regulatory authorities improves the evaluation of marketing authorisation applications (MAAs) for new medicines as well as post-authorisation applications and to explore the practical aspects of the submission and analysis of such data.

Raw data constitutes individual patient data from clinical studies1 in electronic structured format that is directly accessible for analysis and visualisation. Examples of raw data include records of original observations and measurements of clinical study participants, such as clinical laboratory results, imaging data, and patient medical charts. Currently, the European medicines regulatory system does not routinely require the submission of raw data in the context of a marketing authorisation or post-authorisation application.

EMA’s human medicines committee (CHMP) receives data submitted by the applicant or marketing authorisation holder (MAH) after statistical processing in aggregated format as clinical summaries, as well as in PDF listings. The CHMP scrutinises these summaries as part of the scientific evaluation of the benefits and risks of medicinal products. This process typically results in several rounds of questions in which the Committee may ask the applicants / MAHs for methodological clarifications, re-analysis of data, or additional data. However, according to EU regulation, the CHMP can request the applicant / MAH at any time to provide the raw data to perform further analyses to support the benefit risk assessment of medicines2. 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 medicinal product.

The pilot project is open to applicants or MAHs that are about to submit marketing authorisation applications or post-authorisation applications. If selected, they will include raw data already as part of their submissions. More information on the pilot’s objectives and on the terms of participation is available in the description of the Information about the raw data proof-of-concept pilot for industry. 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.

Applicants and MAHs can contact EMA via rawdatapilot@ema.europa.eu to express their interest in participating in the pilot or to gather more information.

This pilot 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 network’s 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. Thus, raw data analysis may enabler faster and better access to new medicines for patients.

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.

1 Clinical studies include clinical trials as well as non-interventional studies in accordance with the definitions set out in Article 2 of Regulation (EU) No 536/2014.

2 In accordance with Article 7(c) of Regulation (EC) No 726/2004 and Article 16.3 of Regulation (EC) 1234/2008.

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