Euro Roundup: EMA answers questions about raw data pilot

The European Medicines Agency (EMA) has published a question-and-answer document about its raw data proof-of-concept pilot, providing the industry with insights into the selection process, the terms of participation and more.

EMA is running the pilot 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,” according to the document. Raw data, also called standardized study data, refers to “individual patient data from clinical studies in [a] structured format.”

The first section of the Q&A covers general questions about the pilot, such as its duration, the regulatory procedures that are in scope and how EMA will use the raw data analysis results. EMA also answered a question about whether it plans to ask for raw data in all applications after the pilot.

“No decision has been taken on the future scope of requests for raw data by the agency. The objective of the pilot is to gather information and provide recommendations to the European Medicines Regulatory Network on the place of access to raw data in future regulatory submissions. Therefore, this is one of the key questions that the PoC pilot will help to answer,” the agency wrote.

In the second section, EMA responds to questions about the terms of participation, such as how and when applicants can confirm their intention to join the pilot and how they will communicate with the agency. The agency plans to use existing regulatory channels to discuss questions related to the pilot but “will follow a flexible approach to allow for sufficient dialogue” and “additional interactions are planned or may be facilitated as needed.”

EMA’s answers in that section confirm that participation in the pilot will not delay the adoption of a scientific opinion on whether to approve a medicine, and that an applicant cannot withdraw once it has confirmed its intention to participate.

The other two sections address the submission of data. Packages prepared for other regulators, such as the US Food and Drug Administration, may be submitted to EMA if they meet the mandatory criteria. EMA lists the files included in the mandatory data package in the response to another question. Another response lists optional files.

EMA Q&A

Access Consortium creates Promise Pilot Pathway, an aligned process for priority review

The Access Consortium working group for new active substances has created an aligned process for priority review that includes the decision on priority status and common timelines for handling requests.

Through the Consortium, agencies in Australia, Canada, Singapore, Switzerland and the UK collaborate on regulatory activities including the review of new active substances. This week, the five agencies shared details of a new work-sharing pathway for priority medicines for serious, life-threatening or severely debilitating conditions that lack approved treatment options.

The Consortium shared details of the Promise Pilot Pathway in an update to its operational procedures for new active substances. Applicants should submit an expression of interest at least six months before filing for marketing authorization. The Consortium is advising applicants that are unsure if their filing will be eligible to discuss the timelines with the agencies or express an interest six months out and withdraw the request if clinical data subsequently fails to support priority review.

One agency will lead the assessment of whether there is evidence of significant therapeutic benefit, and by extension justification for priority review, and “peer review will take place by all agencies, with work being shared between the agencies.” The agencies will try to reach a consensus on the priority review status but “in principle, an agency may still consider its national criteria” as part of its decision. Failure to reach a consensus may lead to the Promise Pilot Pathway only being used for filings with some agencies.

The timeline calls for the submission of applications for screening 25 days before the start of the review. Once the review starts, the agencies have 180 days to reach separate sovereign decisions, in which time they will work together to issue rolling questions and conclude formal work sharing.

Swissmedic Notice

EMA shares guidance on incorporating companion diagnostics into drug development

EMA has responded to frequently asked questions about the use of companion diagnostics in drug development. The document is informed by the implementation of the In Vitro Diagnostic Regulation (IVDR).

In the text, EMA responds to seven questions about the specifics of using companion diagnostics in clinical trials and making drug regulatory submissions based on the resulting data. The first question covers what to consider when predictive biomarker assays are used to define eligibility for treatment. Biomarker selection should use sound scientific rationale, valid scientific criteria and reliable evidence.

EMA also addresses the elements to present in regulatory submissions when predictive biomarker assays are used to select patients, what to consider when multiple assays are used to measure one biomarker and how to present information in the summary of product characteristics.

The agency recommends reading the answers in conjunction with IVDR and links out to multiple other guidance documents related to companion diagnostics in the Q&A.

EMA Q&A

European politicians reach provisional agreement on AI Act

Negotiators for the European Parliament and Council have reached a provisional agreement on the Artificial Intelligence (AI) Act, a wide-ranging regulation with implications for healthcare and medtech.

Meetings to try to reach an agreement took place in June, July, September and October but failed to yield a breakthrough. That changed late last week when negotiators reached a political deal that features safeguards on general purpose AI, regulatory sandboxes for testing technologies before they come to market and fines of up to 7% of global turnover for noncompliance.

“The world’s first horizontal legislation on artificial intelligence will keep the European promise, ensuring that rights and freedoms are at the center of the development of this ground-breaking technology. Correct implementation will be key,” co-rapporteur Brando Benifei said in a statement.

Citizens will have the right to receive explanations about decisions based on high-risk AI systems that impact their rights and national authorities will establish regulatory sandboxes to develop and train innovative AI before it is put on the market.

Press Release

European Commission, WHO sign €12M deal to improve health information systems

The European Commission and World Health Organization (WHO) have inked a €12 million ($13 million), four-year agreement to strengthen health information systems, governance and interoperability.

Through the alliance, the partners aim to support healthcare services for almost 1 billion people living in 53 countries across the WHO European Region. The goal is to improve the use and reuse of health data by care providers, policymakers and patients, and to enhance the quality and interoperability of health information systems.

To achieve those ambitions, the Commission and WHO Regional Office for Europe plan to help build capacity by providing activities and assistance intended to address “gaps, needs and areas of expertise within supported countries’ health information systems and health data governance and capabilities.” The partners may expand successful practices at country or regional levels.

Press Release

Euro Roundup: EMA answers questions about raw data pilot

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