Judicialization of Zolgensma in the Ministry of Health: costs and clinical profile of patients.

IF 2.1 4区 医学 Q3 PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH Revista de saude publica Pub Date : 2024-08-12 eCollection Date: 2024-01-01 DOI:10.11606/s1518-8787.2024058005899
Ana Katheryne Miranda Kretzschmar, Ellen Teixeira, Dayani Galato, Everton Nunes da Silva
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Abstract

Objective: 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®).

Methods: 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.

Results: 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.

Conclusions: 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.

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卫生部对 Zolgensma 的司法化:费用和患者的临床概况。
目的调查因使用onasemnogene abeparvovec(Zolgensma®)治疗脊髓性肌萎缩症(SMA)而向卫生部提起诉讼的患者的费用和概况:这是一项横断面描述性研究,采用普查设计,以2019年1月至2022年9月期间针对卫生部提起的诉讼记录为基础。数据是通过《信息公开法》向卫生部索取的。研究提取了诉讼受益人的流行病学特征信息,以及卫生部在请求获准的情况下所花费的费用:结果:共发现 136 起诉讼,其中 113 起(83%)对患者有利,分析期间共花费 9.448 亿雷亚尔。人口统计学特征(性别和年龄)、临床特征(SMA 亚型、呼吸或营养支持的使用)和诉讼特征(法律服务类型)与诉讼是否获准无关。曾使用药物(奴西那塞纳或利迪普兰)与诉讼被驳回有关。在获准的 113 起有利于患者的诉讼中,只有 6 起(5.3%)符合国家卫生技术整合委员会(Conitec)目前制定的标准(6 个月以下儿童无呼吸和营养支持)。用于向两岁以上儿童供应 Zolgensma 的费用为 1.46 亿雷亚尔,这超出了药品包装说明书中的建议范围:卫生部在佐尔根斯玛(Zolgensma)治疗 SMA 的司法程序上花费巨大,占统一卫生系统药品总支出的 2.45%,其中包括三个行政领域的支出。其中一些诉讼的批准不符合卫生技术评估机构制定的标准和药品制造商的建议。
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来源期刊
Revista de saude publica
Revista de saude publica PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH-
CiteScore
4.60
自引率
3.60%
发文量
93
审稿时长
4-8 weeks
期刊介绍: The Revista de Saúde Pública has the purpose of publishing original scientific contributions on topics of relevance to public health in general.
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