Abstract
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What is EU HTA? In 2021, the European Commission passed a new regulation [1] on Health Technology Assessment (HTA) which will require manufacturers to submit clinical evidence as part of a joint clinical assessment (JCA), addressing the needs of all member states in terms of data and analyses. EU HTA is getting closer (applies as of January 2025 for certain oncology medicines) and will have a major impact on the work of statisticians across the industry.
Final versions of the Implementing Act describing the high-level submission process, and methodological guidelines, that provide applicants and assessors guidance on which statistical methods are appropriate in given situations, have recently been published [2]. Further guidelines, in particular related to processes, are yet to be finalized.
The PICO (Patient, Intervention, Comparator, Outcome) framework is a key topic in EU HTA. This framework will be the standard format in which HTA bodies will specify the research questions that define the assessment scope. Meanwhile, estimands are becoming standard for describing "the treatment effect reflecting the clinical question posed by a given clinical trial objective" in clinical studies. While the PICO and estimands frameworks overlap, the latter further defines the population-level summary and intercurrent event strategy.
We review some of the challenges we can foresee, such as the potential need for a number of different analyses to satisfy the needs of various EU member states, as multiple PICOs may require multiple analyses strategies. Statisticians must also be prepared to handle tight timelines in case of label changes as the joint clinical assessment is due before the CHMP opinion. We will also go through the many unknows surrounding this new process.
Reference
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[1] [Regulation on Health Technology Assessment (europa.eu)](https://health.ec.europa.eu/health-technology-assessment/regulation-health-technology-assessment_en#stakeholder-consultation) last accessed 15th August 2024
[2] https://health.ec.europa.eu/health-technology-assessment/key-documents_en?f%5B0%5D=topic_topic%3A225&f%5B1%5D=topic_topic%3A226&f%5B2%5D=topic_topic%3A227&f%5B3%5D=topic_topic%3A228 (last accessed 15th August 2024)
Bio
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Arthur Allignol is currently HTA statistician at Daiichi Sankyo and is involved in the preparation work for the new EU-HTA regulation. Arthur is an active member of the PSI/EFSPI HTA Special Interest Group.
Before joining Daiichi Sankyo, he worked as Real-World Data Scientist at Merck
Healthcare KGaA and in academia, specializing in observational studies and complex event-history analysis. Arthur Allignol obtained his PhD in biostatistics at the university of Freiburg in 2013.