Universidade de Brasília. Faculdade de Ceilândia. Programa de Pós-Graduação em Ciências e Tecnologias da Saúde. Brasília, DF, Brasil.
Universidade de Brasília. Faculdade de Ceilândia. Curso de Farmácia. Brasília, DF, Brasil.
Rev Saude Publica. 2024 Aug 12;58:36. doi: 10.11606/s1518-8787.2024058005899. eCollection 2024.
To investigate the costs and profile of patients who have filed a lawsuit against the Ministry of Health for the treatment of spinal muscular atrophy (SMA) with the onasemnogene abeparvovec (Zolgensma®).
This is a cross-sectional, descriptive study with a census design, based on records of lawsuits filed against the Ministry of Health between January 2019 and September 2022. Data was requested from the Ministry of Health via the Access to Information Act. Information was extracted on the epidemiological profile of the beneficiaries of the lawsuits, as well as the expenses spent by the Ministry of Health in cases where the requests were granted.
136 lawsuits were identified, of which 113 (83%) were favorable to patients at a cost of R$ 944.8 million in the period analyzed. Demographic (gender and age), clinical (SMA subtypes, use of ventilatory or nutritional support), and lawsuit (type of legal service) characteristics were not associated with the granting of lawsuits. Prior use of medication (nusinersena or ridisplam) was associated with the dismissal of lawsuits. Of the 113 lawsuits granted in favor of patients, only six (5.3%) would meet the criteria currently established by the National Committee for Health Technology Incorporation - Conitec (children up to six months without ventilatory and nutritional support). R$ 146 million was spent on supplying Zolgensma to children over the age of two, which is outside the recommendation contained in the drug's package leaflet.
The Ministry of Health incurs a high cost with the judicialization of Zolgensma for SMA, representing 2.45% of total spending on medicines in the Unified Health System, including spending by the three administrative spheres. Some of the lawsuits have been granted in disagreement with the criteria established by health technology assessment agencies and the drug manufacturer's recommendations.
调查针对卫生部治疗脊髓性肌萎缩症（SMA）而对卫生部提起诉讼的患者的费用和特征，这些患者使用的是onasemnogene abeparvovec（Zolgensma®）。
这是一项基于 2019 年 1 月至 2022 年 9 月期间对卫生部提起的诉讼记录的横截面、描述性研究，采用普查设计。通过信息自由法向卫生部请求数据。提取了诉讼受益人的流行病学特征以及卫生部在批准请求的情况下支出的费用。
共确定了 136 起诉讼，其中 113 起（83%）对患者有利，在此期间花费了 9.448 亿雷亚尔。人口统计学（性别和年龄）、临床（SMA 亚型、使用呼吸机或营养支持）和诉讼（法律服务类型）特征与诉讼的批准无关。先前使用药物（nusinersena 或 ridisplam）与诉讼的驳回有关。在批准的 113 起有利于患者的诉讼中，只有 6 起（5.3%）将符合目前国家卫生技术纳入委员会 - Conitec （无呼吸机和营养支持的六个月以下儿童）确立的标准。为 2 岁以上儿童供应 Zolgensma 花费了 1.46 亿雷亚尔，这超出了药物说明书中建议的范围。
卫生部因 Zolgensma 治疗 SMA 而面临高额的司法费用，占统一卫生系统药品总支出的 2.45%，包括三个行政领域的支出。一些诉讼的裁决与卫生技术评估机构和药品制造商的建议确立的标准不一致。
