PSY176 - SIGNIFICANT BENEFIT AND RELATIVE EFFECTIVENESS ASSESSMENT OF ORPHAN MEDICINAL PRODUCTS IN THE EUROPEAN UNION: A COMPARATIVE ANALYSIS

Abstract

The European Orphan regulation mandates that if there exists a satisfactory method of diagnosis, prevention or treatment, the new medicinal product will have to be of significant benefit (SB) to those affected by that condition. This assessment is carried out by the Committee of Orphan Medicinal Products (COMP) at the time of designation as well as at the market authorization (MA). Health Technology Assessment (HTA) institutions will perform a relative effectiveness assessment (REA) after the MA. In this qualitative study, SB assessments were compared to REAs of 5 HTA institutions in order to describe differences and similarities between the opinions. OMPs which were authorised and assessed for SB at the time of MAbetween 2010-2017 were reviewed. These were matched with available HTA reports. OMPs which were assessed by at least four HTAs were included. The reports were compared based on the objectives according to the PICO method. Finally, 5 exemplary drugs were included in the comparison. A preliminary analysis showed that 75 OMP reports were found for the period 2010-2017, of which 24 were assessed with at least 4 HTAs. The 5 final drugs were included based on the objectives: a EUnetHTA assessment, negative SB, SB based on major contribution to patient care, premature data and different comparators considered. Their detailed analysis is ongoing. As part of the EMA-EUnetHTA work plan 2017-2020, we expect this study will provide more insight into both frameworks. Because this is a qualitative study, it is acknowledged that it will not allow predicting the frequency of these detected differences between both frameworks. Yet this being the first study of such kind, it will contribute to an increased understanding of SB, which ultimately may benefit the health of patients suffering from rare diseases.