How Do We Unleash the Ambition of the EU HTA Regulation Through Practical Methodological Solutions?

ISSUE: Wide variations in clinical practice and the availability of treatments across the EU will likely result in many PICOs being identified when scoping Joint Clinical Assessments (JCAs). This has been confirmed in multiple PICO simulation exercises including the ones undertaken in the EFPIA and Evidera report. Whilst good-quality direct comparative evidence is optimal, the likely number of PICOs mean this will not fully address the scope of many JCAs underscoring the need to understand the experience and current use of state-of-the-art evidence synthesis methods, and their strengths and limitations to facilitate decision-making in the context of JCAs.

OVERVIEW: EFPIA and Evidera conducted a simulation to explore the impact of the proposed EUnetHTA21 methodologies on several widely reimbursed oncology treatments. The simulation of the scoping process resulted in many potential PICOs for each product, ranging from 16 up to 57 depending on the indication and line of treatment.

Context is key to the use of different evidence synthesis methodologies, the simulation demonstrated the central role indirect treatment comparisons, real-world evidence, single-arm studies, and oncology-relevant endpoints play in country appraisals to help address uncertainty. The simulation found that a wide range of evidence synthesis approaches will be essential to conduct comprehensive JCAs and underlined the importance of considering the totality of evidence or endpoints available at the time of submission. With the publication of methodological guidelines over the next few months, it will be important for assessors and technology developers to understand how best to use state-of-the-art methodologies in JCAs and apply learnings from these previous experiences.

The issue panel will start with a short presentation highlighting the issues identified followed by a structured panel discussion with recommendations and audience questions. This panel will be beneficial to stakeholders involved in evidence generation, HTA, and JCA processes.