Pilot scheme to analyse raw trial data launched by EMA

The pilot will assess whether analysing raw clinical trial data is beneficial to regulatory decision making on marketing authorisation and post-authorisation applications.

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A new pilot project by the European Medicines Agency (EMA) will assess whether the analysis of ‘raw data’ from clinical trials improves the evaluation of marketing authorisation applications (MAAs) for new medicines as well as post-authorisation applications. The project will also evaluate the practicality of such submissions and analyses.

Raw data constitutes individual patient data from clinical studies 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. Instead, the Committee for Medicinal Products for Human Use (CHMP) receives statistically processed data in aggregated format as clinical summaries or PDF listings to use in its evaluation of the benefits and risks of medicinal products.

According to EU regulation, the CHMP can request the applicant or marketing authorisation holder (MAH) provide the raw data to perform further analyses to support the benefit risk assessment of medicines at any time. 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 as part of their submissions. More information on the pilot’s objectives and on the terms of participation is available here. 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 also contact EMA via [email protected] 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.