Background: Despite proven efficacy, Manual Vacuum Aspiration (MVA) use for incomplete abortions is low in the Swaziland setting, including Raleigh Fitkin Memorial (RFM) Hospital. Uncertainty in the costs implications of introducing MVA to replace Dilatation and Curettage (D&C) is the major hindrance to change. This study aimed to evaluate the cost-effectiveness of introducing MVA as an evacuation method for first trimester incomplete miscarriages as well as assess the implications of the introduction of MVA to the entire post-abortion care budget at RFM Hospital. Methods: The methods comprised cost-effectiveness and budget impact analyses from a healthcare perspective based on a theoretical cohort. Clinical outcomes data for procedures were obtained from relevant literature. Costs were collated from prospective suppliers and then compared for the two treatment modalities. Future numbers of annual evacuations were extrapolated from previous annual figures. First trimester miscarriages were in turn extrapolated from proportions found in previous studies. Total budgets were calculated under the current scenario, as well as if MVA had to be introduced. Results: With initial capital costs of ZAR11 093.00, introduction of MVA for first trimester incomplete abortions will cut post-abortion care costs by 34.7%. MVA would cost ZAR819.86 per procedure while D&C costs ZAR1 255.40 per procedure. An estimated 26 MVA procedures done instead of D&C will compensate for the initial capital investment. Introduction of MVA into the post-abortion care programme will save the hospital about ZAR516 115.30 annually, with at least similar clinical outcomes compared to D&C. Conclusions: MVA should be considered as the first option in first trimester post abortion care. / Dissertation (MSc)--University of Pretoria, 2013. / gm2014 / Clinical Epidemiology / unrestricted

O estudo tem como objetivo geral avaliar a razão de custo-utilidade do tratamento da infecção pelo vírus da hepatite C (VHC) em pacientes dialisados, candidatos a transplante renal, tendo como esquemas terapêuticos alternativos o interferon-_ em monoterapia; o interferon peguilado em monoterapia; o interferon-_ em terapia combinada com ribavirina e o interferon peguilado em terapia combinada com ribavirina, comparando-os com o nãotratamento. A perspectiva do estudo foi a do Sistema Único de Saúde(SUS), que também serviu de base para estimar o impacto orçamentário da estratégia de tratamento mais custo efetiva. Para o alcance dos objetivos, foi construído um modelo de Makov para simulação de custos e resultados de cada estratégia avaliada. Para subsidiar o modelo, foi realizada uma revisão de literatura, a fim de definir os estados de saúde relacionados à infecção pelo vírus da hepatite C em transplantados e a probabilidade de transição entre os estados. Medidas de utilidade foram derivadas de consultas a especialistas. Os custos foram derivados da tabela de procedimentos do SUS. Os resultados do estudo demonstraram que o tratamento da infecção pelo VHC antes do transplante renal é mais custo-efetivo que o não tratamento, apontando o interferon-a como a melhor opção. O impacto orçamentário para adoção dessa estratégia pelo SUS corresponde a 0,3% do valor despendido pelo SUS com terapia renal substitutiva ao longo do ano de 2007. / The objective of the study was to analyze the cost-utility of the treatment of hepatitis C in patients on dialysis awaiting renal transplantation. The aim was to compare among distinct treatments: monotherapy with interferon-_, monotherapy with pegylated interferon, interferon-_ associated with ribavirin, and pegylated interferon with ribavirin, and with those without treatment. The perspective used was the Brazilian Public health system (SUS), which was also the base for calculating the costs of the treatment more cost effective. To analyze the data the Markov model was used for simulation of the costs and results obtained for each treatment used. Data from literature were used to define the health states of the transplanted patients infected by hepatitis C, and the probability of transition among the states. Utility measures derived from specialists evaluation. The costs derived from the procedures list of the SUS. The results show that treatment of hepatitis C before renal transplantation is more cost-effective compared with no treatment, and interferon-a is the best option. The budget impact of this strategy corresponds to 0.3% of the total amount used by the SUS with renal replacement therapy in the year of 2007.

O estudo tem como objetivo geral avaliar a razão de custo-utilidade do tratamento da infecção pelo vírus da hepatite C (VHC) em pacientes dialisados, candidatos a transplante renal, tendo como esquemas terapêuticos alternativos o interferon-_ em monoterapia; o interferon peguilado em monoterapia; o interferon-_ em terapia combinada com ribavirina e o interferon peguilado em terapia combinada com ribavirina, comparando-os com o nãotratamento. A perspectiva do estudo foi a do Sistema Único de Saúde(SUS), que também serviu de base para estimar o impacto orçamentário da estratégia de tratamento mais custo efetiva. Para o alcance dos objetivos, foi construído um modelo de Makov para simulação de custos e resultados de cada estratégia avaliada. Para subsidiar o modelo, foi realizada uma revisão de literatura, a fim de definir os estados de saúde relacionados à infecção pelo vírus da hepatite C em transplantados e a probabilidade de transição entre os estados. Medidas de utilidade foram derivadas de consultas a especialistas. Os custos foram derivados da tabela de procedimentos do SUS. Os resultados do estudo demonstraram que o tratamento da infecção pelo VHC antes do transplante renal é mais custo-efetivo que o não tratamento, apontando o interferon-a como a melhor opção. O impacto orçamentário para adoção dessa estratégia pelo SUS corresponde a 0,3% do valor despendido pelo SUS com terapia renal substitutiva ao longo do ano de 2007. / The objective of the study was to analyze the cost-utility of the treatment of hepatitis C in patients on dialysis awaiting renal transplantation. The aim was to compare among distinct treatments: monotherapy with interferon-_, monotherapy with pegylated interferon, interferon-_ associated with ribavirin, and pegylated interferon with ribavirin, and with those without treatment. The perspective used was the Brazilian Public health system (SUS), which was also the base for calculating the costs of the treatment more cost effective. To analyze the data the Markov model was used for simulation of the costs and results obtained for each treatment used. Data from literature were used to define the health states of the transplanted patients infected by hepatitis C, and the probability of transition among the states. Utility measures derived from specialists evaluation. The costs derived from the procedures list of the SUS. The results show that treatment of hepatitis C before renal transplantation is more cost-effective compared with no treatment, and interferon-a is the best option. The budget impact of this strategy corresponds to 0.3% of the total amount used by the SUS with renal replacement therapy in the year of 2007.

INTRODUÇÃO: As diretrizes para Análise de Impacto Orçamentário (AIO) têm a finalidade de padronizar os estudos, tornando-os úteis e compreensíveis para os tomadores de decisão. Avaliar a concordância dessas diretrizes às recomendações de boas práticas para elaboração de AIO da Internacional Society for Pharmacoeconomic and Outcomes Research (ISPOR) e conhececer a metodologia do desenvolvimento por meio do instrumento Appraisal of Guidelines for Research & Evaluation Instrument (AGREE II) podem contribuir para um conhecimento dos itens de maior relevância para a promoção de padronização e de transparência dos estudos de AIO. OBJETIVO: Revisar sistematicamente as diretrizes para a elaboração de AIO. MÉTODOS: Foram utilizadas as bases de dados Medical Literature Analysis and Retrieval System Online (MEDLINE) (via PubMed), Excerpta Medica dataBASE (EMBASE) (via Elsevier), Cochrane Library, NHS Economic Evaluation Database (NHS EED), HTA Database (via Centre for Reviews and Dissemination - CRD) e Latin American and Caribbean Health Sciences Literature (LILACS); websites de organizações de avaliações de tecnologias em saúde e Ministérios da Saúde dos países membros da Organização das Nações Unidas. O critério de inclusão foi tratar-se de diretriz metodológica com recomendações para a elaboração de AIO. O processo de seleção foi realizado por dois revisores de forma independente e, no caso de discordância, por um terceiro revisor. Os instrumentos utilizados foram recomendações de boas práticas para a elaboração de AIO da ISPOR e o AGREE II. Esta revisão foi relatada de acordo com o Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (PRISMA). RESULTADOS: Foram incluídas 17 diretrizes. O período de publicação ocorreu de 2006 a 2018. Das diretrizes incluídas, dez eram atualizações. Mais da metade (10 de 17) estava incluída em documentos para outros tipos de avaliação econômica em saúde e sete eram diretrizes específicas para AIO. Essas apresentaram concordância com as recomendações da ISPOR de 30% a 92% e as diretrizes incluídas em documentos para outros tipos de avaliação econômica em saúde apresentaram concordância de 19% a 70%. A avaliação metodológica por meio do instrumento AGREE II apresentou uma pontuação de 18% (DP ±12%) no domínio rigor metodológico e de 15% (DP ±23%) no domínio independência; para as diretrizes específicas para AIO, a pontuação foi de 26% (DP ±32%) e para as diretrizes com mais de um tipo de avaliação econômica em saúde, de 8% (DP ±13%). CONCLUSÃO: A concordância das diretrizes para a elaboração de AIO foi maior nas diretrizes específicas para AIO. Quanto à análise da metodologia, 15 diretrizes apresentaram baixa qualidade metodológica no domínio rigor metodológico, sendo recomendável explicitar o processo do desenvolvimento metodológico / INTRODUCTION: The guidelines for Budgetary Impact Analysis (BIA) aim to standardize studies, making them useful and understandable to decision makers. To evaluate the agreement of these guidelines to the recommendations of good practices for the preparation of BIA of the International Society for Pharmacoeconomic and Outcomes Research (ISPOR) and to know the development methodology through the Appraisal of Guidelines for Research and Evaluation Instrument (AGREE II) can contribute to a knowledge of the most relevant items to promote a standardization and transparency of BIA studies. OBJECTIVE: To systematically review the guidelines for elaborating budget impact analysis. METHODS: We used the Medical Literature Analysis and Retrieval System Online (MEDLINE), Excerpta Medica dataBASE (EMBASE) (via Elsevier), Cochrane Library, NHS Economic Evaluation Database (NHS EED) and HTA Database (via Center for Reviews and Dissemination (CRD) and Latin American and Caribbean Health Sciences Literature (LILACS); on the websites of the following organizations health technology assessment and of the Ministries of Health of the member countries of the United Nations. The inclusion criterion was to be methodological guidelines with recommendations for the elaboration of BIA. The selection process was carried out by two reviewers independently and in the case of disagreement, a third reviewer. The instruments used were those of ISPOR and AGREE II. This review has been reported according to the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (PRISMA). RESULTS: 17 guidelines were included. The publication period occurred from 2006 to 2018. Of the guidelines included, ten were updates. More than half (10 out of 17) were included in documents for other types of economic health assessment and only seven guidelines were BIA-specific documents. The specific BIA guidelines were in agreement with the ISPOR recommendations from 30% to 92% and the guidelines for BIA included in documents for other types of health economic assessment presented agreement from 19% to 70%. The methodological evaluation using the AGREE II instrument presented a score of 18% (SD ±12%) in the methodological rigor domain and 15% (DP ±23%) in the independence domain, for the specific guidelines for AIO was 26% (SD ± 32%) and guidelines with more than one type of health economic assessment, 8% (SD ± 13%). CONCLUSION: A greater adherence to the recommendations of ISPOR was observed in the BIA-specific guidelines. As regards the methodology analysis, 15 guidelines presented low quality in the methodological rigor domain, and it is advisable to explain the process of methodological development

Depuis le début des années 2000, les traitements anticancéreux oraux (TAO) sont en développement croissant, notamment dans le cancer du sein. Ils permettent aux patients la prise de comprimés par voie orale à domicile améliorant la qualité de vie. On estime que 10 à 34% des tumeurs du sein sur-expriment la protéine HER2 (HER2+) qui augmente le risque de métastases. Deux thérapies ciblées anti-HER2 sont actuellement disponibles : le trastuzumab, anticorps monoclonal administré par voie intraveineuse et le lapatinib, inhibiteur de tyrosine kinase administré par voie orale. D’après les recommandations de l’Agence Nationale d’Accréditation et d’Evaluation en Santé (ANAES, 2003), les TAO devraient être privilégiées par rapport aux formes intraveineuses prises à l’hôpital lorsque leur efficacité est équivalente. Dans la pratique courante des oncologues, les TAO semblent néanmoins sous-utilisées dans certains cas. En plus des freins médicaux connus (adhésion thérapeutique, gestion des effets indésirables), les TAO induisent une consommation de ressources hopitalières supplémentaires qui n’est pas valorisée dans le modèle de tarification à l’activité (T2A) des établissements hospitaliers, lequel repose sur la nature et la quantité d’activité médicale réalisée. Nous supposons que le modèle de T2A représente un frein économique à l’utilisation des TAO entrainant une disparité d’accès entre les traitements anticancéreux oraux et intraveineux. L’objectif de ce travail de thèse était donc de déterminer le rôle de la T2A dans l’accès des patients aux TAO au moyen d’une évaluation médico- économique de l’impact économique et organisationnel des TAO sur le système de soins. Une revue de littérature a permis de mettre en évidence le rôle du mode de financement des soins sur l’accès aux TAO en France et aux Etats-Unis. L’analyse des bases de données nationales hospitalières du Programme Médicalisé des Systèmes d’Information (PMSI) a permis de quantifier l’enjeu économique de la chimiothérapie pour les établissements de soins et qui représente la deuxième activité des hôpitaux en volume après l’hémodialyse. Le bénéfice lié aux séances de chimiothérapie a ainsi été estimé à 108 millions d’Euros en 2010 pour l’ensemble des établissements publics et privés en France. L’analyse a également permis de simuler le transfert d’allocation de ressources de l’hôpital vers les soins de ville induit par la substitution des séances de chimiothérapie par l’utilisation des TAO. Un modèle comparant le coût du traitement intraveineux (trastuzumab) aux TAO (lapatinib et capécitabine) dans le cancer du sein métastatique HER2+ a confirmé des coûts moindres pour les TAO (17 165€ versus 36 077€ par an et par patient) liés à une économie sur les transports médicalisés mais surtout sur les consommations hospitalières et ce, malgré un coût d’acquisition plus élevé des TAO. Malgré cet impact budgétaire négatif pour les hôpitaux, une étude de préférences (Méthode des Choix Discrets) conduite auprès d’un échantillon de 203 médecins hospitaliers et libéraux a montré que l’efficacité d’un traitement anticancéreux restait le déterminant principal de la décision thérapeutique (β=2,214, p<0,0001). L’étude a toutefois révélé que, aux stades avancés du cancer, la voie d’administration et son coût étaient également associés au choix du traitement (β=0,612, p=0,035 ;β=0,506, p<0,0001). Les résultats montrent donc que le modèle de financement de l’activité hospitalière influence le choix des modalités de traitement même si les critères d’ordre médicaux et environnementaux du patient demeurent des déterminants essentiels dans le choix du recours aux TAO (profil clinique du patient, adhésion thérapeutique, préférences du patient, environnement familial et socio-économique, conditions d’accès à l’offre de soins) / Since the early 2000’s, oral anticancer drugs (OADs) are increasingly available especially for the treatment of breast cancer. This route of chemotherapy administration allows patients to take oral tablets at home improving their quality-of-life. We estimate that 10 to 34% of breast tumors over-express the HER2 protein (HER2+) that increases the risk of developing metastasis. Two anti-HER2 targeted therapies are currently available: trastuzumab, a monoclonal antibody administered intravenously and lapatinib, a tyrosine kinase inhibitor administered orally. According to the recommendations of the National Agency for Accreditation and Evaluation in Health (ANAES, 2003), OADs should be administered when their efficacy is equivalent to the one of intravenous forms taken at hospital. In the current practice of oncologists, OADs seem to be underused in some cases. The medical brakes to the use of OADs (i.e. adherence, management of side effects) are well known. Nonetheless, OADs induce additional hospital healthcare resources which are not taken into account in the hospital payment system that is based on the nature and the quantity of medical activities performed (i.e. per-case payment system (PPS)). We assume that the current model of PPS represents an economic barrier to the use of OADs and which induces a disparity of access between oral and intravenous cancer treatments. The objective of this thesis was to determine the role of the PPS on the patient access to OADs based on a medico-economic evaluation of the economical and organizational impacts of OADs on the health care system. A literature review has highlighted the role of the funding of care on the access to OADs in the French and US healthcare systems. From an analysis of the national hospital database (PMSI database), we have quantified the economic implications of chemotherapy administration that is the second hospital activity in volume after hemodialysis. Earnings associated with chemotherapy sessions have been estimated at 108 million Euros in 2010 for all private and public institutions in France. This analysis also allowed us to simulate the transfer of resources allocation from hospital to community setting induced by the substitution of chemotherapy sessions by the use of OADs. A model comparing the cost of intravenous anticancer drug (trastuzumab) to OADs (lapatinib and capecitabine) in the treatment of HER2+ metastatic breast cancer confirmed the lower costs for OADs (€ 17,165 versus € 36,077 per year per patient). The higher acquisition cost of OADs was offset by the cost savings in terms of medical transportation and hospital resources. Despite this negative budget impact for hospitals, a preference study (Discrete Choice Experiment) conducted among 203 physicians showed that the efficacy of cancer treatment remained the main determinant of the therapeutic decision (β=2.214, p<0.0001). The study has also revealed that, in the advanced stages of cancer, the route of administration and its associated cost was also associated with the treatment choice (β=0.612, p= 0.035; =β0.506, p<0.0001). Overall, the results show that the hospital payment system influences the choice of treatment modalities. Nonetheless, medical criterions related to the patient remain essential in the choice of using OADs (clinical profile of the patient, adherence, patient preferences, familial and socio-economic environment, and conditions of access to health care)