Judicialization of Zolgensma in the Ministry of Health: costs and clinical profile of patients
DOI:
https://doi.org/10.11606/s1518-8787.2024058005899Keywords:
Spinal Muscular Atrophy, Onasemnogene Abeparvovec, Health Judicialization, Cost AnalysisAbstract
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.
References
Mendonça RH, Matsui C Jr, Polido GJ, Silva AM, Kulikowski L, Torchio Dias A, et al. Intragenic variants in the SMN1 gene determine the clinical phenotype in 5q spinal muscular atrophy. Neurol Genet. 2020 Sep;6(5):e505. https://doi.org/10.1212/NXG.0000000000000505
Prior TW, Leach ME, Finanger E. Adam MP, Mirzaa GM, Pagon RA. Spinal muscular atrophy. Seattle: University of Washington; 2020.
Costa JO, Costa CCP, Lima NS. Genes de sobrevivência do neurônio motor e a atrofia muscular espinhal 5q. Genética Escola. 2021;16(2):306-15, https://doi.org/10.55838/1980-3540.ge.2021.374
Pera MC, Coratti G, Berti B, D’Amico A, Sframeli M, Albamonte E, et al. Diagnostic journey in Spinal Muscular Atrophy: is it still an odyssey? PLoS One. 2020 Mar;15(3):e0230677. https://doi.org/10.1371/journal.pone.0230677
Araujo APQC, Giuliani A, Bonfim D, Loriato D, Zanotelli E, Braga F, et al. Guia de discussão sobre Atrofia Muscular Espinhal no Brasil: Trabalhando hoje para mudar o amanhã. 2019 [cited 2023 Sep 29]. Available from: https://www.juntospelaame.com.br/content/dam/intl/latam/brazil/sma/patients/hubtogetherinsma/images/articles/Guia_Discussao_AME_Brasil.pdf
Agência Nacional de Vigilância Sanitária. Anvisa concede registro ao medicamento Spinraza. Brasília, DF: Agência Nacional de Vigilância Sanitária; 2017 [cited 2023 Sep 6]. Available from: https://www.gov.br/anvisa/pt-br/assuntos/noticias-anvisa/2017/anvisa-concede-registro-ao-medicamento-spinraza
Agência Nacional de Vigilância Sanitária. EVRYSDI® (risdiplam): novo registro. Brasília, DF: Agência Nacional de Vigilância Sanitária; 2020 [cited 2023 Sep 6]. Available from: https://www.gov.br/anvisa/pt-br/assuntos/medicamentos/novos-medicamentos-e-indicacoes/evrysdi-r-risdiplam-novo-registro
Agência Nacional de Vigilância Sanitária. Aprovado registro de produto de terapia gênica. Brasília, DF: Agência Nacional de Vigilância Sanitária; 2020 [cited 2023 Sep 6Available from: http://antigo.anvisa.gov.br/resultado-de-busca?p_p_id=101&p_p_lifecycle=0&p_p_state=maximized&p_p_mode=view&p_p_col_id=column-1&p_p_col_count=1&_101_struts_action=%2Fasset_publisher%2Fview_content&_101_assetEntryId=5989035&_101_type=content&_101_groupId=219201&_101_urlTitle=aprovado-registro-de-produto-de-terapia-genica&inheritRedirect=true
Ministério da Saúde (BR). Comissão Nacional de Incorporação de Tecnologias no Sistema Único de Saúde. Relatório de Recomendação Nº 449 - Nusinersena para atrofia muscular espinhal 5q. Brasília, DF: Ministério da Saúde; 2019 [cited 2023 Sep 6]. Available from: https://www.gov.br/conitec/pt-br/midias/relatorios/2019/relatorio_nusinersena_ame5q.pdf
Ministério da Saúde (BR). Comissão Nacional de Incorporação de Tecnologias no Sistema Único de Saúde. Relatório de Recomendação Nº 595 - Nusinersena para tratamento da Atrofia Muscular Espinhal 5q tipo II e III (início tardio). Brasília, DF: Ministério da Saúde; 2021 [cited 2023 Sep 6]. Available from: https://www.gov.br/conitec/pt-br/midias/ relatorios/2021/20210602_relatorio_595_nusinersena_ame5q_2e3_p_26.pdf
Ministério da Saúde (BR). Comissão Nacional de Incorporação de Tecnologias no Sistema Único de Saúde. Relatório de Recomendação Nº 710 - Risdiplam para o tratamento de atrofia muscular espinhal (AME) tipo II e III. Brasília, DF: Ministério da Saúde; 2022 [cited 2023 Sep 6]. Available from: https://www.gov.br/conitec/pt-br/midias/relatorios/2022/20220314_relatorio_710_risdiplam_ametipoiieiii.pdf
Ministério da Saúde (BR). Comissão Nacional de Incorporação de Tecnologias no Sistema Único de Saúde. Relatório de Recomendação Nº 709 - Risdiplam para o tratamento de atrofia muscular espinhal (AME) tipo I. Brasília, DF: Ministério da Saúde; 2022 [cited 2023 Sep 6]. Available from: https://www.gov.br/conitec/pt-br/midias/relatorios/2022/20220314_relatorio_709_risdiplam_ametipoi.pdf
Ministério da Saúde (BR). Comissão Nacional de Incorporação de Tecnologias no Sistema Único de Saúde. Relatório de Recomendação Nº 793 - Onasemnogeno abeparvoveque para o tratamento de atrofia muscular espinhal (AME). Brasília, DF: Ministério da Saúde; 2022 [cited 2023 Sep 6]. Available from: https://www.gov.br/conitec/pt-br/midias/relatorios/portaria/2022/20221207_relatorio_zolgensma_ame_tipo_i_793_2022.pdf
Campolina AG, Yuba TY, Soárez PC. Decision criteria for resource allocation: an analysis of CONITEC oncology reports. Cien Saude Colet. 2022 Jul;27(7):2563-72. https://doi.org/10.1590/1413-81232022277.14242021
Guimarães R. Novos desafios na avaliação de tecnologias em saúde (ATS): o caso Zolgensma. Cien Saude Colet. 2023 Jul;28(7):1881-9. https://doi.org/10.1590/1413-81232023287.18122022
Riera R, Bagattini ÂM, Pachito D. Eficácia, segurança e aspectos regulatórios dos medicamentos órfãos para doenças raras: o caso Zolgensma®. Cad Iberoam Direito Sanit. 2019 Sep;8(3):48-59. https://doi.org/10.17566/ciads.v8i3.538
Scottish Medicines Consortium. Decision explained - Medicine: onasemnogene abeparvovec (brand name: Zolgensma®). Amsterdam: European Medicines Agency; 2021.
Canadian Agency for Drugs and Technologies in Health. CADTH Common drug review: onasemnogene abeparvovec. Ottawa: Canadian Agency for Drugs and Technologies in Health; 2021.
National Institute for Health and Care Excellence. Onasemnogene abeparvovec for treating spinal muscular atrophy: highly specialised technologies guidance. Manchester: National Institute for Health and Care Excellence ; 2021.
Chieffi AL, Barata RC. Ações judiciais: estratégia da indústria farmacêutica para introdução de novos medicamentos. Rev Saude Publica. 2010;44(3):421-9. https://doi.org/10.1590/S0034-89102010000300005
Araújo LC, Moita MP, Silva LCS, Mesquita KO, Vasconcelo FJL, Dias MS.A Judicialização do acesso a medicamentos no Brasil: revisão integrativa da literatura. Sanare. 2021;20(1):131-41. https://doi.org/10.36925/sanare.v20i1.1557
Oliveira YM, Braga BS, Pereira SP, Ferreira MA. Braga BSF, Pereira SPD, et al. Judicialização de medicamentos: efetivação de direitos ou ruptura das políticas públicas? Rev Saude Publica. 2020 Dec;54:130. https://doi.org/10.11606/s1518-8787.2020054002301
Vieira FS. Judicialização e direito à saúde no Brasil: uma trajetória de encontros e desencontros. Rev Saude Publica. 2023 Feb;57(1):1. https://doi.org/10.11606/s1518-8787.2023057004579
Ferreira GA, Ferreira CA. O Sistema Único de Saúde (SUS) brasileiro: trajetória e perspectivas. Rev Direito Debate. 2022 Jun 15;32(59):1-12. https://doi.org/10.21527/2176-6622.2023.59.11861
Brasil. Decreto Nº 11.391, de 20 de janeiro de 2023. Altera o Decreto nº 11.358, de 1º de janeiro de 2023, que aprova a Estrutura Regimental e o Quadro Demonstrativo dos Cargos em Comissão e das Funções de Confiança do Ministério da Saúde. Diário Oficial União, Brasília, DF, 2023.
Costa CA. Gastos com a judicialização de tecnologias de saúde: um estudo empírico no executivo federal do Brasil [Master’s degree]. Florianópolis: Universidade Federal de Santa Catarina; 2022.
Ministério da Saúde (BR). Fundação Oswaldo Cruz. Contas de saúde na perspectiva da contabilidade internacional: conta SHA para o Brasil, 2015 a 2019. Brasília, DF: IPEA; 2022.
Silva FS, Rodrigues JMP, Brito RN, Macedo TC, Borgmann AD. Intervenção fisioterapêutica na atrofia muscular espinhal: revisão de literatura. Rev Neurocienc. 2021;29:1-22. https://doi.org/10.34024/rnc.2021.v29.11029
Verhaart IE, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, et al. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review. Orphanet J Rare Dis. 2017 Jul;12(1):124. https://doi.org/10.1186/s13023-017-0671-8
Strauss KA, Farrar MA, Muntoni F, Saito K, Mendell JR, Servais L, et al. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nat Med. 2022 Jul;28(7):1381–9. https://doi.org/10.1038/s41591-022-01866-4
Novartis. Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy (NCT03381729). 2017 [cited 2023 Sep 29]. Available from: https://classic.clinicaltrials.gov/ct2/show/NCT03381729
Novartis. Gene transfer clinical trial for spinal muscular atrophy type 1. Ohio; 2017 [cited 2023 Sep 29]. Available from: https://classic.clinicaltrials.gov/ct2/show/study/NCT02122952
Day JW, Finkel RS, Chiriboga CA, Connolly AM, Crawford TO, Darras BT, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021 Apr;20(4):284-93. https://doi.org/10.1016/S1474-4422(21)00001-6
Mercuri E, Muntoni F, Baranello G, Masson R, Boespflug-Tanguy O, Bruno C, et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021 Oct;20(10):832-41. https://doi.org/10.1016/S1474-4422(21)00251-9
Sales CMP, Soliani FCBG, ACB. Farmacoterapia da atrofia muscular espinhal Pharmacotherapy of spinal muscular atrophy. Volume 40. J Health Sci Inst. 2022;40(2):119-26.
Novartis Gene Therapies. Single-dose gene replacement therapy clinical trial for patients with spinal muscular atrophy type 1 with one or two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion (NCT03837184). 2019 [cited 2023 Sep 29]. Available from: https://clinicaltrials.gov/study/NCT03837184
Institute for Clinical and Economic Review. Spinraza® and Zolgensma® for spinal muscular atrophy: effectiveness and value: final evidence report. 2019 [cited 2023 Sep 29]. Available from: https://icer.org/wp-content/uploads/2020/10/ICER_SMA_Final_Evidence_Report_110220.pdf
Baioni MT, Ambiel CR. Spinal muscular atrophy: diagnosis, treatment and future prospects. J Pediatr (Rio J). 2010;86(4):261-70. https://doi.org/10.2223/JPED.1988
Roche IN. Retrato da AME no Brasil: um mapeamento com 144 respondentes revela o cenário desafiador de pessoas com atrofia muscular espinhal e seus cuidadores, do diagnóstico à assistência. E também aponta caminhos para superar essas barreiras. Veja Saúde. 2022 [cited 2023 Sep 29]. Available from: https://assets.roche.com/f/173846/x/58ecc454a0/book-saude-ame-v2.pdf
Lin CW, Kalb SJ, Yeh WS. Delay in diagnosis of spinal muscular atrophy: a systematic literature review. Pediatr Neurol. 2015 Oct;53(4):293-300. https://doi.org/10.1016/j.pediatrneurol.2015.06.002
Downloads
Published
Issue
Section
License
Copyright (c) 2024 Everton Nunes da Silva, Ana Katheryne Miranda Kretzschmar, Ellen Teixeira, Dayani Galato
This work is licensed under a Creative Commons Attribution 4.0 International License.
