Análise crítica do processo de incorporação de novas tecnologias em saúde para doenças raras pelo Sistema Único de Saúde e propostas de otimização

Resumo

The advances in medicine and technology have led to the development of therapies for the treatment of rare diseases that, until recently, did not have therapeutic options available. This fact imposes a great challenge to the healthcare systems due to the high cost and high budget impact caused by the acquisition of those technologies. In this context, aiming at the sustainability of the systems and more efficient resource allocation, the Health Technology Assessment (HTA) process provides methodologies for the definition around effectiveness and the feasibility of reimbursement of new therapies, medicines and procedures. In Brazil, the National Commission for the Incorporation of Healthcare Technologies (CONITEC) has been performing this role for a decade within the SUS, dealing more and more frequently with the challenge of HTA for rare diseases therapies. The current work aims at performing a critical analysis of the HTA process led by CONITEC for rare diseases therapies, more specifically for the products indicated for genetic rare disorders. 46 reports have been evaluated, referring to 21 genetic rare conditions. Criteria such as time for assessment, cost of treatment, costeffectiveness ratio, budget impact analysis, available evidence, social participation, and the recommendation from other HTA agencies have been evaluated. Overall, it has been identified a lack of cohesion among the presented justification for the final recommendations, with no clear criteria seen as determinant for the decision on the reimbursement of the technologies. It has been also noted that the HTA process conducted for rare diseases is the same used for the evaluation of therapies for more prevalent conditions, what has been proved to be insufficient. Although the HTA process for rare diseases has evolved in the last decade, there is still room for improvement. Among the proposals of this project are the establishment of specific processes for rare and ultrarare diseases, the utilization of broader models rather than classic cost-effectiveness analysis, the intensification of the collaboration with other international HTA agencies and the revaluation of the feasibility of implementing alternative reimbursement models and agreements to increase access to those therapies.

Ficha do documento

Tipo

Dissertação

Ano

2022

Instituição

Fundação Getulio Vargas

Fonte

Repositório da FGV

Idioma

Português

Acesso

Acesso aberto

Identificador

oai:repositorio.fgv.br:10438/33013

Temas

Administração PúblicaTecnologiaSaúde

Palavras-chave

Rare diseasesHealth technology assessmentUnified Healthcare SystemPublic healthDoenças rarasAvaliação de tecnologias em saúdeSistema Único de SaúdeSaúde públicaCONITECAdministração públicaSistema Único de Saúde (Brasil)Saúde pública - Inovações tecnológicasInovações médicas