Transforming uncertainties into legitimate regulation? Nice and conitec agencies’ decisions on rare diseases

Abstract

As a scientific and technological prac-tice, the evaluation of health technologies (HTA) is, at the same time, a challenge to determine the value of the technologies to be incorporated. This study aimed to explore and compare the results and technical elements of the evaluations issued for rare diseases between the English (NICE) and the Brazilian agency (CONITEC). The first part of the study involved the systematic search for evaluations from 2013 to 2019. In the second stage, the reports were analyzed based on: (i) descriptive narrative review; and (ii) calculation of the absolute and relative frequency according to each domain and component (element) applied in the European HTA network model. Twenty-four medicines were distinctly assessed during the study period. Through 126 questions (elements) distributed among nine domains, the analysis re-vealed that 67 (53.2%) and 44 (35.0%) were described in the reports, 42 (33.3%) and 59 (47.0 %) were only considered partially, and 17 (13.5%) and 23 (18.0%) were not considered in the NICE and CONITEC reports, respectively. We identified a relatively low agreement between the Brazilian agency with the English agency in the reports issued for rare diseases. It remains to be seen whe-ther the agencies are able to capture the various values ​of these medicines, as well as manage uncertainties in the evaluations.