Mapping the Alignment Between the Portuguese Pharmacotherapeutic Assessment Methodology and EU Joint Clinical...

Regulation (EU) 2021/2282 on health technology assessment (HTA) entered into force on January 11, 2022 and became applicable on January 12, 2025, introducing Joint Clinical Assessments (JCAs) focused on relative clinical effectiveness and safety to support national HTA processes. This study assessed the alignment of Portugal's pharmacotherapeutic assessment methodology (version 3.0, 2022) in force within the National HTA System (SiNATS) with EU JCA guidance.

Five domains were analyzed: quantitative evidence synthesis; multiple hypothesis testing and complementary analyses; outcome measures; clinical study validity; and scoping. Methodological topics and subtopics were extracted from both sources and compared item by item to determine whether they were present in both methodologies (concordant or divergent) or in only one. Descriptive statistics were used to summarize findings.

Most differences reflected information gaps in Portugal's methodology rather than conflicting methodological principles. Across domains, subtopics present in only one methodology accounted for 43.1% of items in quantitative evidence synthesis, 56.2% in multiple hypothesis testing and complementary analyses, 72.2% in outcome measures, 61.1% in clinical study validity, and 58.3% in scoping; most of these were addressed only in EU JCA guidance. Where subtopics overlapped, agreement was high (71.4%-100%), and remaining divergences mainly concerned terminology and approaches to complementary analyses.

Portugal's guidance is broadly consistent with the EU JCA framework where content is shared, but targeted updates are needed, particularly for outcomes, scoping, evidence validity appraisal, and multiplicity reporting, to maximize the direct usability of JCA reports within SiNATS as the Regulation is progressively implemented.