Introduction : La tyrosinémie de type I est une maladie génétique sévère. Elle se caractérise par des manifestations hépatiques, rénales et neurologiques. Depuis 1994, le NTBC représente la thérapie de première ligne. Ce médicament a conduit à une amélioration radicale de la morbidité et du pronostic de la maladie. Objectif : Évaluer les coûts directs des soins de santé reliés au traitement de cette maladie. Cette évaluation économique a été effectuée en trois groupes dont, un groupe historique de patients non traités; un groupe traitement tardif et un groupe traitement précoce par NTBC. Méthode : L’analyse coûts-conséquences inclut les coûts des hospitalisations, des services médicaux et des thérapies associées. Les données proviennent des banques de la RAMQ, de Med-Echo et des dossiers hospitaliers. Résultats : Le NTBC est associé à une réduction significative des hospitalisations, des séjours aux soins intensifs et des greffes hépatiques. Les coûts hospitaliers sont significativement moindres pour les groupes traités (13 979 $, 6 347 $ et 673 $ par année-patient pour les groupes historique, traitement tardif et traitement précoce,respectivement; valeur p < 0,0001). Les coûts des hospitalisations pour greffes par année-patient sont de 4 676$ pour le groupe historique et de 3 567 $ pour le groupe traitement tardif. Les coûts du NTBC par année-patient sont de 66 965 $ et de 51 493 $ pour les groupes traitement tardif et traitement précoce, respectivement. Conclusion : Les résultats démontrent l’impact majeur du NTBC sur la réduction de l’utilisation des ressources de santé, des greffes hépatiques et des coûts associés. / Introduction : Tyrosinemia type I is a severe genetic disorder. Symptoms include hepatic,renal and neurological manifestations. NTBC became the first-line therapy in 1994. This drug has led to a drastic improvement in the prognosis. Objective : To evaluate direct medical costs of healthcare services related to the treatment for this disease. This economic evaluation was conducted among the 3 following groups: an historical group of untreated patients, a late-treatment group and an early-treatment group with NTBC. Methods : The costs-consequences analysis includes costs incurred by hospitalizations, medical services and related treatments. Data are derived from the RAMQ and Med-Echo administrative databases and patients’ hospital charts. Results : NTBC treatment is associated with a significant reduction in hospitalizations, intensive care unit stays and liver transplantations. The cost of hospitalizations is significantly less for both treated groups (13,979 $, 6,347 $ and 673 $ per year-patient for the historical group, the late-treatment and the early-treatment group, respectively; pvalue< 0,0001). The cost of hospitalizations for liver transplantations per year-patient is 4,676 $ for the historical group and 3,567 $ for the late-treatment group. The cost of NTBC per year-patient is 66,965$ and 51,493$ for the late-treatment and the earlytreatment groups, respectively. Conclusion : These results demonstrate that NTBC treatment results in a major reduction in healthcare resources utilization, liver transplantations and associated costs. / Réalisé dans le cadre d'un mandat de l'Unité d'évaluation des technologies et des modes d'intervention en santé (UETMIS) du CHU Sainte-Justine

Made available in DSpace on 2011-05-04T12:36:29Z (GMT). No. of bitstreams: 0 Previous issue date: 2010 / A lei brasileira garante o direito à Assistência Farmacêutica, mas ainda há falhas do Estado na efetivação do acesso dos cidadãos aos medicamentos, assim, as ações judiciais de medicamentos têm tido importante papel como via alternativa ao acesso a medicamentos no SUS. Este tipo de ação judicial vem crescendo anualmente e tem como objeto tanto os medicamentos em falta na rede pública como aqueles ainda não incorporados pelo sistema de saúde. O presente trabalho teve como objetivo principal analisar os medicamentos presentes nas demandas judiciais da comarca da capital encaminhadas a Central de Mandados da Secretaria de Saúde e Defesa Civil do Rio de Janeiro, no período de julho de 2007 a junho de 2008, frente às alternativas terapêuticas presentes nas listas de financiamento público e à luz das evidências científicas. Desta forma, foram analisados os medicamentos cadastrados na Central de Mandados da SESDEC/RJ referentes a 281 pacientes demandantes. Foi observado neste estudo uma diversidade de situações dos indivíduos demandantes e dos medicamentos solicitados. Alguns indivíduos eram demandantes de primeira vez, mas foi também identificada a existência de pacientes que já se encontravam recebendo medicamentos pela Secretaria Estadual de Saúde e que recorreram à via judicial para obtenção do mesmo ou de medicamento adicional. No que tange aos medicamentos solicitados, também houve uma miríade de categorias ao se considerarem as evidências disponíveis e as informações existentes. A maior parte dos medicamentos demandados não era financiada pelo sistema de saúde e entre estes, destaca-se: 1) medicamentos solicitados para indicações registradas no órgão sanitário, com evidências científicas e que apresentavam alternativas terapêuticas financiadas pelo sistema; 2)medicamentos cujas evidências em longo prazo ainda não se encontram bem estabelecidas; 3) medicamentos para indicações não aprovadas pela Anvisa; 4) medicamentos sem registro na Anvisa e; 5) medicamentos cujas evidências inexistem ou não recomendam seu uso. Uma vez que as listas de financiamento da Assistência Farmacêutica e os Protocolos Clínicos e Diretrizes Terapêuticas foram construídos com base em evidências, deve-se priorizar a utilização dos medicamentos constantes nestes. Havendo alternativa terapêutica nas listas oficiais para a indicação proposta na prescrição médica àquela demandada, a decisão deve considerareste fato. / The Brazilian law guarantees the right of Pharmaceutical Care, but is falling short of the state in realization of citizens' access to medicines, so, the medicine lawsuits have been important as an alternative route to access to medicines in the SUS. This type of lawsuit is increasing annually and aims medicines missing from the public health system and those not yet incorporated into this system. This study aimed to examine the medicines in the lawsuits of the District Judicial sent to Central Warrants of the Secretary of Health and Civil Defense of Rio de Janeiro, from July 2007 to June 2008, compared to alternative therapies on the lists of public health system and examine the existence of scientific evidence. Thus, was analyzed the medicines registered in the Central Warrants of SESDEC / RJ referring to 281 patients. Was observed in this study a variety of situations of individuals and the plaintiffs requested medication. Some individuals were the first-time, but also was identified the existence of patients who were already receiving medications for the State Health Department and have appealed to the courts to obtain the same or additional medicine. With respect to medicines requested, there was also a myriad of categories when considering the available evidence and existing information. Most of the medications examined was not financed by the public health system and between them stands out: 1) medicines for indication approved by Anvisa with scientific evidence and having alternative therapies financed by the system, 2) medicines for which evidence on long term are not yet well established, 3) medications for indications not approved by ANVISA, 4) medicines not registered in Anvisa and 5) medicines for which the evidence does not exist or recommend its use. Once the lists of funding for Pharmaceutical Care and Clinical Protocols and Therapeutic Guidelines have been constructed based on evidence, we must prioritize the use of these medications listed. If there is an alternative therapy in the official lists for the proposed indication in the prescription to that defendant, the decision should consider this fact.