Quinze Anos de Avaliação Económica de Medicamentos em Portugal: uma Revisão

Abstract

Objectives: The aim of this review is to quantify and analyze, under Portuguese methodological guidelines, studies of economic evaluation of medicines presented and published in the last fifteen years. Additionally, based on empirical evidence from this revision and the experience of the authors, it is our purpose to launch the discussion for a future review of methodological guidelines. Methods: A comprehensive literature review identified the studies published between January 1999 and December 2013 was performed for Portugal. The literature search method is based on the iterative technique of growing pearl using capture-recapture methods. The search was supplemented with a request for studies through the list of associated APIFARMA and APES, which enabled the integration of grey literature. Results: This review identified and analyzed a total of 99 studies presented in the last fifteen years being the majority of studies presented as a poster/oral communication (64%) and 33 (33%) published as scientific articles. There was observed an increasing trend in the number of studies trough the years, reflecting the need and the importance of these studies to support decision-making on public funding of medicines in Portugal. Most studies have focused on cancer or biological medicines (32%). Most studies (36%) reported dominance conclusions and only 14% of studies reported cost effectiveness ratios over 30 000 €, which may indicate a selection presentation bias, towards studies with more positive results. Regarding Portuguese methodological guidelines, some aspects related to social perspective, indirect costs, discount rates, estimative of costs, health care resources consumption and estimates of treatment effect in the case of indirect comparisons would benefit from an extended discussion in Portuguese guidelines. Conclusions: There was an increasing trend in the number of publications trough the years, reflecting the need to execute studies of economic evaluation for submission of requests for reimbursement of medicines in Portugal. There seems to be some evidence of publication bias towards positive results. Despite the huge gains that methodological guidelines provided to the execution and standardization of economic evaluation studies, and therefore to the decision making policy in Portugal, the last fifteen years show that our guidelines did incorporated a set of principles whose validity remained steady over time but there are areas to improve and a thorough revision is necessary.

Keywords

• Economic evaluation
• medicines
• cost-effectiveness
• cost-utility
• Portugal