Clinical outcome assessments (COAs), used in clinical studies (whether they are interventional (also called experimental), or observational), are a key component of health technology assessment (HTA). They provide the estimate of the effectiveness of the targeted treatment on how patients feel, function, or survive, and the estimate of treatment safety (1). In the context of joint clinical assessment (JCA), outcomes are
relevant in two different steps. The first step is during the scoping process (as described in the Health Technology Assessment Regulation (HTAR) guidance on the scoping process), in which Member States (MSs) are expected to request their needs in terms of health outcomes (HTA Regulation (EU) 2021/2282, Article 8(6)) when defining the assessment scope regarding the Population(s), Intervention, Comparator(s), and Outcome(s) (PICO). Defining relevant outcomes is an important component of this process. The second step is the production of the JCA report by assessors and co- assessors based on the dossier submitted by the health technology developer (HTD) and the assessment scope previously defined for a given health technology. While MSs are required to give due consideration to the JCA reports published (Article 13(1)), the clinical relevance or interpretation of the estimate of relative effectiveness and safety may differ between MSs when drawing conclusions regarding the clinical added value of a treatment at a national level. As a JCA report must not contain any judgment on the clinical added value of a health technology,
appropriate factual reporting of the methodological and statistical elements and results
of the analyses of the outcomes requested is essential to allow MSs to perform the
appraisal they deem appropriate according to their national decision-making process
(Article 9(1))…
