EUnetHTA understands that the relevance and interpretation of clinical outcomes will differ between Member States when measuring a treatments clinical added value at a national level. The guideline sets out two main objectives to combat this. Firstly, it should provide Member States with the ability to define relevant outcomes during the scoping process (EUnetHTA 21 practical guideline D4.2). The outcomes provided during the scoping process will be essential in the results of the JCA, as they will be used to estimate a technologies relative effectiveness. Secondly, at the national level, it should assist by defining all elements needed by Member States in order to show clinical added value during the health technology assessment. Clinical added value can be influenced by the level of validity and reliability of outcomes measurement instruments or the relevance of surrogate outcomes.

Clinical relevance is a major focus of the document. It states that patient centred outcomes should be well defined, decided in close collaboration with patients and healthcare professionals, and be either long-term or final wherever possible. For specific therapeutic areas, there are efforts to standardise outcomes into a defined core outcome set (COS). For the JCA, having a COS in place will provide the benefit of reducing heterogeneity between clinical studies and allowing the easier implementation of evidence synthesis (part of a previous deliverable on direct and indirect comparisons). Health outcomes not included in the COS may be used as complimentary evidence, if deemed relevant by patients, clinical experts or HTA bodies. The use of surrogate outcomes, such as biomarkers and intermediate outcomes, is considered controversial and should only be used if a final outcome is not available. In this case the evidence from the surrogate outcome must show a strong association or correlation of effects on the final outcome.

The document outlines the safety terminology that will be consistent throughout the JCA and provides guidance on overall and specific adverse events. Member States should provide their explicit requirements for safety outcomes during the scoping process. Any specified adverse effects must be reported on. If any adverse effects occur that have not been specified, these will be combined to give an overall safety result as part of the JCA. The guidance goes on to describe the requirements for adverse event reporting including terminology, definition of severity, discontinuation or interruption, and inclusion of all adverse events as an appendix to the JCA report.

The final section of the document relates to validity, reliability and interpretability of outcomes measurement instruments. These require a conceptual framework outlining interrelationships among collected evidence and are used to determine a scale. This can then be used to score specific outcomes. Access to the instrument and/or instructions should be provided by the health technology developer. Studies and statistical analysis will be conducted to determine the validity of and reliability of such instruments. If a specific instrument is requested during the scoping process, the suitability and quality of the instrument is critical. To determine interpretability of an instrument, there should be a classification on whether patients have received a treatment benefit or not. The guidance document outlines the requirements during JCA reporting in order to prove an outcomes measurement instrument is suitable to show validity, reliability and interpretability.

Overall, this gives an example of how the EUnetHTA is beginning to encourage adoption of the JCA at the national level by standardising the relevant outcomes, safety criteria and framework for outcomes measurement instruments between Member States.

Article published 27 January 2023.