The European Medicines Agency (EMA) has answered a multitude of questions from sponsors on its raw data pilot, a program aimed at understanding if submitting and assessing raw data will improve the agency’s review of drug applications.

Currently, the agency’s review body, the Committee for Medicinal Products for Human Use (CHMP), mainly assesses data in clinical summaries and information in trial reports, which come in a format that do not directly allow disaggregation of data or any other form of further analysis. The EMA believes that it may help to have access to raw data — individual electronic participant data that are directly available for reanalysis, additional analysis and visualization during the committee’s reviews.

The Q&A document, which spans 34 questions in total, is split into four sections:

• General questions (such as the reasons behind the pilot, its start and end date, its scope and how findings will be shared with the public);
• Terms of participation (such as how to submit inquiries about the pilot, how to express interest, withdrawal policy and how to communicate during participation);
• The data package to be submitted (such as whether data packages with other regulators can be used, the scope of data packages, mandatory and optional files to include, and the impact of issues with data standards/formats and missing files); and
• Technical questions on submitting raw data (such as how to submit datasets to EMA, the maximum size of the data package allowed and how to submit additional data or data from multiple trials).

Data packages should, at a minimum, contain: the signed participation letter; deidentified datasets from the relevant trials; data definition files in CDISC Define-XML format; the analysis data reviewer’s guide and study data reviewer’s guide; software programs for the creation of Analysis Data Model sets; and software programs related to the primary and secondary efficacy analyses, including programs used to generate figures and tables.

The EMA says it will accept data packages prepared for other regulators, including the FDA and Health Canada, as long as they contain the required contents.

Access the Q&A document here: https://bit.ly/3Wss2FR.