EMA Q&A addresses submission of data elements for raw data pilot

The European Medicines Agency (EMA) this week issued a question-and-answer guidance to address sponsors’ questions on its pilot testing the review of raw clinical trial data for marketing authorization applications (MAAs) and post-authorization applications.
 
EMA issued an updated guidance on the pilot in October. (RELATED: EMA to explore reviewing raw data from clinical trials in applications, Regulatory Focus 12 July 2022)
 
The purpose of the pilot is to “investigate the benefits of having access to raw data from regulatory submissions to support the scientific assessment of medicinal products and to identify the associated operational, resource and technological needs.”
 
The guidance addresses general questions, terms for participating, the data package to be submitted and technical questions related to submitting the raw data.
 
Examples of raw data include records of original observations and measurements of clinical study participants, including clinical laboratory results, imaging data and patient medical charts.
 
One question addressed how the raw data analysis results will be used. The answer: “visualsation and analysis of raw data will be used to support the benefit-risk assessment of the regulatory procures. For some procedures the raw data will be used to support GCP [Good Clinical Practice] inspections.”
 
Another question related to which parties are going to analyze the raw data submitted for the proof-of-concept pilot. The answer: “For each regulatory procedures, raw data analysis will be performed by the CHMP rapporteurs teams at the NCs, EMA staff or an EMA contractor.”
 
In response to a question on how long the raw data files will be stored at EMA, the agency said the retention period is up to 30 years after the product has been withdrawn.
 
Another question related to which files are mandatory. The agency states that at a minimum, the package should contain the signed pilot participation letter and data sets from the clinical studies. Data sets should be submitted in either SAS Transport format (XPORT) or other file transport formats such as Extensible Markup Language (XML) or JavaScript Object Notation (JSON).
 
The pilot stems from a joint workplan announced by EMA and the EU’s Heads of Medicines Agencies (HMA) in 2021 to address issues related to big data in healthcare and medicines development. One of the ten priority recommendations that emerged was to launch a pilot allowing sponsors to submit raw data to allow faster evaluation of products with fewer questions. (RELATED: EMA goes all in on big data, Regulatory Focus 17 August 2021)
 
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