de Oliveira Verônica Stasiak Bednarczuk, Amaral Marise Basso, Camargo Mariana, Lopes-Pacheco Miquéias
United for Life Institute-Cystic Fibrosis, Rare and Respiratory Diseases, Curitiba 80420-130, PR, Brazil.
Department of Pharmaceutical Sciences, Federal University of Paraná, Curitiba 80210-170, PR, Brazil.
Healthcare (Basel). 2025 Aug 14;13(16):1996. doi: 10.3390/healthcare13161996.
The health technology assessment (HTA) is a multidisciplinary process utilized to determine the clinical, economic, social, and ethical value of new health technologies before they are incorporated into healthcare systems. In the case of rare diseases, such as Cystic Fibrosis (CF), challenges arise due to limited evidence and high treatment costs. Indeed, although CF transmembrane conductance regulator (CFTR) modulators are breakthrough therapies for CF, their incorporation into public health systems has been complex with considerable challenges, especially in low- and middle-income countries. This article presents a descriptive and exploratory case study of the regulatory and policy journey for CFTR modulators to be approved in Brazil. Based on a narrative review and document analysis, we highlight the importance of building lay society knowledge to shape policy decisions and promote equitable access to innovative therapies. In parallel, we critically reflect on the HTA process and highlight efforts in the training, education, and coordination required to enable meaningful public engagement and landmark achievements.
卫生技术评估（HTA）是一个多学科过程，用于在新的卫生技术纳入医疗保健系统之前，确定其临床、经济、社会和伦理价值。在罕见病方面，如囊性纤维化（CF），由于证据有限和治疗成本高昂，会出现各种挑战。事实上，尽管CF跨膜传导调节因子（CFTR）调节剂是CF的突破性疗法，但将它们纳入公共卫生系统一直很复杂，面临着诸多挑战，尤其是在低收入和中等收入国家。本文介绍了巴西批准CFTR调节剂的监管和政策历程的描述性和探索性案例研究。基于叙述性综述和文献分析，我们强调建立普通社会知识以塑造政策决策并促进公平获得创新疗法的重要性。同时，我们批判性地反思了HTA过程，并强调了在培训、教育和协调方面所做的努力，以实现有意义的公众参与和标志性成就。
