The value of medicinal products: from the EMA to HTA bodies

There can be diverse conceptions and perspectives regarding the value of a product, which may be perceived by the various stakeholders in different ways. The value of a medicinal product can be closely related to therapeutic and clinical aspects (such as efficacy and safety), as well as to its broader societal effects on the community; it can also be defined in terms of the economic advantage it offers, or its cost-utility and cost-effectiveness. For many years now, regulatory agencies have been responsible for the demanding task of assessing the value of a therapy. The assessments conducted by the EMA, the FDA and the singular HTA authorities to establish the value of a therapy can differ. The potential concordance between the agencies’ evaluations is thus an important consideration with direct implications for patient access. Whether they are the result of temporal factors or evaluation methodologies, discrepancies in the assessment of pharmaceutical dossiers can lead to heterogeneous situations regarding the availability of products across regions. In this analysis, we aimed to elucidate regulatory and access discrepancies in the assessment of medicinals value', taking into consideration the impact of clinical uncertainties in the overall benefit/risk evaluation and cost-effectiveness assessments.