Caetano Rosângela, Hauegen Renata Curi, Osorio-de-Castro Claudia Garcia Serpa
Instituto de Medicina Social, Universidade do Estado do Rio de Janeiro, Rio de Janeiro, Brasil.
Centro de Desenvolvimento Tecnológico em Saúde, Fundação Oswaldo Cruz, Rio de Janeiro, Brasil.
Cad Saude Publica. 2019 Aug 29;35(8):e00099619. doi: 10.1590/0102-311X00099619.
In April 2019, a ruling was signed for the incorporation of the drug nusinersen by the Brazilian Unified National Health System (SUS). Nusinersen is the most expensive drug ever incorporated by the SUS and is used to treat type I 5q spinal muscular atrophy. The incorporation has been described as a milestone in decision-making on new technologies in the SUS, enabled through a risk-sharing agreement. The article discusses the process involved in the incorporation of nusinersen, highlighting the context, timing, and technical issues, in addition to possible consequences for the institutionalization of health technology assessment (HTA) in the SUS. The study used an exploratory method, reviewing public information produced by the Commission for Incorporation of Technologies in the SUS (CONITEC) and searches in government databanks on prices and purchases. A timeline was produced, describing the key points in the process of incorporation. There were two formal requests for the drug's incorporation. The first was submitted by the Division of Science, Technology, and Strategic Inputs (SCTIE) of the Brazilian Ministry of Health and was turned down unanimously in November 2018. This was followed by a petition by the head of the SCTIE to the Attorney General's Office (AGU) to overrule the recommendation by the CONITEC plenary. The AGU recommended a new submission, made by the drug's manufacturing company, which was approved unanimously in March 2019. The was no addition of new evidence or a price reduction to justify the change of decision. No elements were identified in the risk-sharing agreement. This suggests problems of transparency and accountability, as well as risks in the process of institutionalization of HTA that had been underway in the SUS.
2019年4月，一项关于巴西统一国家卫生系统（SUS）纳入药物诺西那生钠的裁决签署。诺西那生钠是SUS纳入的最昂贵药物，用于治疗I型5q脊髓性肌萎缩症。此次纳入被描述为SUS新技术决策中的一个里程碑，这是通过风险分担协议实现的。本文讨论了诺西那生钠纳入过程中涉及的程序，除了对SUS卫生技术评估（HTA）制度化可能产生的影响外，还强调了背景、时机和技术问题。该研究采用探索性方法，审查了SUS技术纳入委员会（CONITEC）产生的公共信息，并在政府数据库中搜索价格和采购信息。制作了一个时间表，描述了纳入过程中的关键点。有两次正式的药物纳入申请。第一次由巴西卫生部科学、技术和战略投入司（SCTIE）提交，于2018年11月被一致否决。随后，SCTIE负责人向总检察长办公室（AGU）请愿，要求推翻CONITEC全会的建议。AGU建议由药品制造公司重新提交申请，该申请于2019年3月获得一致批准。没有增加新的证据或降低价格来证明决策的改变是合理的。在风险分担协议中未发现任何要素。这表明存在透明度和问责制问题，以及SUS正在进行的HTA制度化过程中的风险。
