Learnings to Kick-Start 2025: Joint Clinical Assessments and Health Technology Assessments

Published January 17, 2025

One trend Trinity Life Sciences has observed and that was reinforced by stakeholders at ISPOR EU is the importance of evidence in healthcare decision-making. This trend was highlighted particularly in the context of joint clinical assessments (JCA) and health technology assessments (HTA). Trinity presented 11 posters at ISPOR EU, 6 of which focused on HTA and/or JCA.

JCA is an EU initiative intended to improve patient access to health technologies in Europe through a harmonized clinical HTA process. Beginning in January 2025, Oncology and advanced therapy medicinal products will be the first to be evaluated through the JCA, which will expand to cover all medicinal products by 2030. As the EU kicks off this process, a few challenges remain, such as capacity available in smaller countries to participate in the process and risk of assessments becoming outdated by the time reimbursement is sought in later launch markets. The introduction of the JCA poses substantial uncertainty for the industry, starting with the scoping (i.e., PICO) process, making it crucial for innovators to prepare for potential impacts on their development and commercialization planning from an evidence-generation and value-demonstration perspective. As an example, developers will need to consider variability around methodological differences between JCA and the local HTA methods (e.g., acceptability of endpoints) in planning next steps.

While all stakeholders are aligned on the importance of bringing medicines to patients quickly, representatives of regulatory and payer bodies at ISPOR noted that it is also essential not to sacrifice evidence quality. Speaking on this point, an HTA agency representative noted that approving reimbursement of a therapy with high uncertainty in benefit can lead to net harm, as it takes away healthcare resources that could be used for other patients or diseases.

With regards to evidence, HTA agency representatives emphasized that goals for regulatory and HTA bodies are different, with the former focused on assessing benefit-risk balance of a product and the latter on comparative benefit vs. existing options. These differences often result in divergent data expectations, which might be further underlined through the parallel regulatory and clinical HTA processes in the next few years.

Real-world evidence (RWE) has become increasingly important in HTA submissions, with most incorporating RWE. Data vendors and consultancies at the conference reported an increased desire amongst industry clients to design EU-based RWE studies to support HTA submissions and inform commercial assessments. Access remains challenging due to more restrictive privacy rules for claims data in Europe, but policymakers and other parties are making strides in streamlining access. RWE must be relevant and applicable to the decision problem and population of interest, and analytical methods that minimize the risk of bias should be used.

One topic that generated considerable interest was the cost-effectiveness of various cancer therapies. Most of these studies relied on data sources that were hard to generalize, highlighting the need for mapping the current ecosystem and organized real-world data throughout the EU5.

The National Institute for Health and Care Excellence (NICE) is an early adopter of AI and sees a “huge playground of use cases where potential risk is covered by having a human looking over the shoulder of AI.” NICE’s ongoing AI work focuses on guidance for tech developers on best practices for AI-based methods to support evidence generation, evaluation of technologies incorporating AI, and using AI to support and improve the efficiency of NICE’s internal processes.

In conclusion, evidence remains critical in healthcare decision-making, and stakeholders must prioritize it over expediency. Real-world data and AI have the potential to improve evidence generation and decision-making, but their applications must be transparent, relevant and applicable to the decision problem. Health technology developers must anticipate methodological differences between EU JCA and local HTA methods and adapt their evidence generation plans accordingly.

Authors: Ismail Ismailoglu, Maxine Diehl, Nandini Hadker, Liz Hamilton, Andreia Ribeiro, Dan Furrer and Mary Fletcher-Louis