Bakgrund: Kostnadsökningen inom hälso- och sjukvården har varit ett stort problem sedan 1980-talet och kommer att fortsätta vara ett problem i framtiden. Det har lett till en mer ansträngd ekonomisk situation som hälso- och sjukvården fått förhålla sig till. Därför är det av stor vikt att de disponibla medel som finns inom hälso- och sjukvården allokeras till de områden där de skapar som mest nytta. Syfte: Syftet med studien är att göra en hälsoekonomisk utvärdering av ett nytt arbetssätt för behandling av knäskador på ortopedkliniken på Länssjukhuset i Kalmar. Metodval: Studien är utformad som en utvärderingsstudie med en abduktiv utgångspunkt. Datainsamlingen har skett utifrån semistrukturerade intervjuer på LSK med personal som är involverad i projektet. Utöver de semistrukturerade intervjuerna har även numeriska data utgjort en del av studiens empiri. Slutsats: Efter genomförd studie kan vi konstatera att fler hälsoekonomiska utvärderingar behövs på icke-nationella nivåer för att stödja verksamheter inom hälso- och sjukvården. Det är viktigt att det aktivt arbetas med effektiviseringsarbeten inom offentliga verksamheter som främst finansieras av skattemedel. Vi kan även konstatera att det nya arbetssättet är mer lönsamt än det gamla både monetärt och icke-monetärt där förändringen bidragit med flera vinningar som exempelvis kortare ledtider, bättre samarbete och utökad kompetens. / Background: The cost increase in healthcare has been a major problem since the 1980s and will continue to be a problem in the future. This has led to a more strained economic situation for the healthcare sector. Therefore, it is of the utmost importance that the available funds in the healthcare sector are allocated to the areas where they are most beneficial.  Purpose: The purpose of this study is to make a health-economic evaluation of a new work procedure for the treatment of knee injuries at the orthopedic clinic at Kalmar’s County Hospital. Method: The study is designed as an evaluation study with an abductive outset. The data collection has been based on semi-structured interviews at LSK with the staff that was involved in the project. In addition to the semi-structured interviews, numerical data has also been part of the study for the empirical data collection. Conclusion: After completion of the study, we can state that more health-economic evaluations are needed at non-national levels to support healthcare organisations. It is important to actively work with efficiency initiatives in public organisations that are primarily funded by tax assets. We can also state that the new work procedure is more profitable than the old one, both monetary and non-monetary, where the change of work procedure has resulted in several non-monetary gains, such as shorter lead times, better cooperation and increased competence.

The diploma thesis focuses on economic situation assessment of the company PULCO, a.s. By the business analysis and financial analysis gauges the health of the company. The analysis is followed by comments and comparison with result for the competitive environment. As a result is suggestions that are applicable in use and may be solutions of the future problems in company.

Rökning är ett utbrett samhällsproblem världen över och sjukdomar relaterade till rökning leder till mer än åtta miljoner dödsfall årligen. Effektiva rökavvänjningsmetoder spelar därför en viktig roll i samhället då de besparar samhället sjukvårdskostnader och främjar folkhälsan. I detta arbete genomförs en hälsoekonomisk utvärdering av ett SMS-baserat stöd för rökavvänjning. Syftet med arbetet är att fylla en kunskapslucka vad det gäller kostnadseffektivitet av digitala rökavvänjningsinsatser och samtidigt ge ett underlag tillbeslutsfattare som ansvarar för att prioritera folkhälsofrämjande insatser. Den hälsoekonomiska utvärderingen använder sig av kostnadseffektivitetsanalys som metod och baseras på en klinisk studie, där långsiktiga kostnader och hälsoeffekter skattas med hjälpav en beslutsanalytisk modell primärt baserad på en Markovmodell. De tre vanligaste följdsjukdomarna av rökning; KOL (kronisk obstruktiv lungsjukdom), lungcancer och hjärt- och kärlsjukdom modelleras mot bakgrund av hur många som slutar röka med hjälp av SMS-stödet. Data från olika källor används i modellen och i de fall data saknas görs antaganden baserade på tidigare studier för att kunna skatta kostnader och hälsoeffekter. Arbetet påvisar att en implementering av ett SMS-baserat stöd skulle generera ökade hälsoeffekter till lägre kostnader i jämförelse med att inte implementera ett sådant stöd. Resultatet från grundscenariot visar att interventionen skulle leda till kostnadsbesparingar på 63 539 kr per individ som tar del av interventionen och 0,30 vunna år i perfekt hälsa, sett över ett livstidsperspektiv. Resultaten bör tolkas med viss försiktighet då de är förknippade med osäkerheter, men kan användas som en del av det underlag beslutsfattare behöver för att fattabeslut om hur hälso- och sjukvårdens resurser ska prioriteras. Trots osäkerheterna i underlagetförefaller ett SMS-baserat stöd för rökavvänjning ha positiva hälsoeffekter och samtidigt spara resurser och därmed framstår metoden som en potentiellt viktig insats i framtida folkhälsoarbete. / Smoking is a globally widespread societal problem and diseases related to smoking account for more than eight million deaths annually. Effective smoking cessation methods therefore play an important role in society as they save society´s healthcare resources and promote public health. In this work, a health economic evaluation of an SMS-based smoking cessation is performed. The purpose of the work is to fill a knowledge gap in terms of cost-effectiveness of digital smoking cessation initiatives and at the same time provide a basis for decision-makers who are responsible for prioritizing public health promotion initiatives. The health economic evaluation uses cost-effectiveness analysis as a method and is based on a clinical study, where long-term costs and health effects are estimated using a decision-analytical model with a Markov structure. The three most common sequelae of smoking; COPD (chronic obstructive pulmonary disease), lung cancer and cardiovascular disease are modeled based on how many people stop smoking with the help of the SMS support. Data from different sources are used in the model and required assumptions based on previous studies are used to be able to estimate costs and health effects. The work shows that the implementation of an SMS-based support would generate increased health effects at lower costs in comparison with not implementing such support. The results from the basic scenario show that the intervention would lead to cost savings of SEK 63,539 per individual who takes part in the intervention and a gain in 0.30 years in full health, seen over a lifetime perspective. The results should be interpreted with some caution as they are associated with uncertainties but can be used as part of the basis on which decision-makers need to make decisions about how health care resources should be prioritized. Despite these uncertainties the SMS-based support for smoking cessation appears to save resources and lead to positive health effects and the method should be considered a potentially important addition in future public health policy.

Ventilator-associated pneumonia (VAP) is the most common healthcare-associated infection in the intensive care unit, resulting in a high burden of illness, mortality and increased cost. The literature around the cost-effectiveness of probiotics in prevention of health-care associated infections has not been previously well-described, and a definitive health economic evaluation alongside a well-designed randomized control trial assessing probiotic prophylaxis has not been previously performed. This thesis consists of 3 separate manuscripts (with 2 published in peer-reviewed journals and 1 pending). The theme of this thesis was to: (1) describe the literature about the cost-effectiveness of probiotics in hospitalized patients in preventing healthcare-associated infections; (2) design a protocol for an economic evaluation alongside a randomized control trial (RCT) examining probiotic prophylaxis of VAP; and then (3) perform and analyze the health economic evaluation presented in the protocol. The first component of this thesis is a systematic review of probiotic prophylaxis of healthcare-associated infections in hospitalized patients. We performed an extensive search including multiple databases which found 7 studies. Probiotics demonstrated favourable cost-effectiveness in 6 of 7 (86%) economic evaluations, with 3 studies being manufacturer-supported, all suggesting cost-effectiveness. Certainty of cost-effectiveness evidence was very low due to risk of bias, imprecision and inconsistency using the GRADE approach. Hence further RCTs with economic evaluations were stated as a solution. The second component of this thesis is a study protocol for an economic evaluation alongside the Probiotics to Prevent Severe Pneumonia and Endotracheal Colonization Trial (PROSPECT), which assessed the efficacy of probiotic prophylaxis in the prevention of healthcare-associated infections (specifically VAP). The third component of this thesis is the cost-effectiveness analysis performed utilizing the individual patient data from PROSPECT to produce the economic evaluation (E-PROSPECT). As of the date of thesis submission, PROSPECT is still pending publication, and hence E-PROSPECT is also pending analysis and publication. However, I have prepared a draft manuscript (along with figures and tables) that will be produced at the conclusion of E-PROSPECT for thesis committee review. / Thesis / Master of Health Sciences (MSc) / Ventilator-associated pneumonia (VAP) is the most common healthcare-associated infection in the intensive care unit, resulting in a high burden of illness, mortality and increased cost. The literature around the cost-effectiveness of probiotics in prevention of health-care associated infections has not been previously well-described, and a definitive health economic evaluation alongside a well-designed randomized control trial assessing probiotic prophylaxis has not been previously performed. This thesis consists of 3 separate manuscripts (with 2 published in peer-reviewed journals and 1 pending). The theme of this thesis was to: (1) describe the literature about the cost-effectiveness of probiotics in hospitalized patients in preventing healthcare-associated infections; (2) design a protocol for an economic evaluation alongside a randomized control trial (RCT) examining probiotic prophylaxis of VAP; and then (3) perform and analyze the health economic evaluation presented in the protocol. The first component of this thesis is a systematic review of probiotic prophylaxis of healthcare-associated infections in hospitalized patients. We performed an extensive search including multiple databases which found 7 studies. Probiotics demonstrated favourable cost-effectiveness in 6 of 7 (86%) economic evaluations, with 3 studies being manufacturer-supported, all suggesting cost-effectiveness. Certainty of cost-effectiveness evidence was very low due to risk of bias, imprecision and inconsistency using the GRADE approach. Hence further RCTs with economic evaluations were stated as a solution. The second component of this thesis is a study protocol for an economic evaluation alongside the Probiotics to Prevent Severe Pneumonia and Endotracheal Colonization Trial (PROSPECT), which assessed the efficacy of probiotic prophylaxis in the prevention of healthcare-associated infections (specifically VAP). The third component of this thesis is the cost-effectiveness analysis performed utilizing the individual patient data from PROSPECT to produce the economic evaluation (E-PROSPECT). As of the date of thesis submission, PROSPECT is still pending publication, and hence E-PROSPECT is also pending analysis and publication. However, I have prepared a draft manuscript (along with figures and tables) that will be produced at the conclusion of E-PROSPECT for thesis committee review.

Esta tese tem por objetivo apresentar limitantes e potencialidades da política de acesso aos medicamentos essenciais, implementada através do Programa Aqui tem Farmácia Popular (PAFP) na perspectiva do desenvolvimento econômico do país. Para alcançá-lo, propôs-se a realização de uma pesquisa, com enfoques particulares, inter-relacionados, dando origem a três artigos. O primeiro artigo analisa a estrutura do Programa Farmácia Popular do Brasil (PFPB), tendo por base as dimensões propostas no modelo da Organização Mundial da Saúde (OMS) em conjunto com a Management Sciences for Health (MSH), discutindo conceitos e abordagens do papel do Estado na perspectiva do desenvolvimento econômico, modelos de acesso a medicamentos e indicadores do setor saúde no contexto do desenvolvimento econômico no Brasil. Ao caracterizar o PFPB, identificaram-se elementos alinhados às dimensões disponibilidade, aceitabilidade, acessibilidade geográfica e poder de compra. A estratégia do PAFP mostrou-se ser econômica, justificando manter-se como estratégia prioritária do Estado. Entretanto, há limitações a serem consideradas para que tenha melhor desempenho no que se propõe, em especial, em relação à dimensão do poder de compra da farmácia privada. Com o intuito de verificar o papel do Estado através de ações no âmbito da assistência farmacêutica, o segundo artigo avalia, qualitativamente, o PAFP, a partir da perspectiva dos usuários. Os dados foram obtidos através de procedimentos metodológicos e de instrumento desenvolvidos para tal. Avaliou-se a ocorrência de migração dos usuários do Programa Farmácia Básica (PFB) do SUS para o PAFP e suas motivações, incluindo a avaliação do atendimento de suas necessidades e o seu grau de satisfação em relação ao PAFP. Os resultados indicam que não há migração significativa (16%) de usuários do PFB do SUS para o PAFP, sugerindo que não há concorrência entre os Programas, sendo complementares. A pesquisa possibilitou reconhecer que houve ampliação da facilidade de acesso a medicamentos, oferta de alternativa positiva para os usuários, ganho financeiro do usuário com a utilização do PAFP, entre outros. Constata-se, entretanto, como limitantes, a questão da inconstitucionalidade do Sistema de Copagamento e a sustentabilidade financeira do Programa. O terceiro artigo apresenta elementos que apontam aspectos de integração, ou não, da dinâmica do Complexo Econômico-Industrial da Saúde (CEIS) aos resultados do PAFP, a partir da pesquisa empírica realizada para identificar e comparar os gastos federais entre o PFB do SUS e o PAFP. A fundamentação teórica abrange as relações do desenvolvimento econômico, inovação tecnológica e o CEIS. Os dados da pesquisa foram obtidos entre 12 municípios que compuseram o estudo piloto, na perspectiva da aplicação da metodologia em todo o país. A análise comparativa dos valores de compra de medicamentos entre as duas redes, pública e privada, evidenciou que o valor de compra pelas farmácias privadas é maior que o praticado pelas farmácias públicas. A análise custo-volume permitiu inferir que a farmácia privada tem compensações entre os medicamentos. Conclui-se que, de fato, não há integração plena do CEIS na promoção do PAFP. Apesar dos limitantes, as potencialidades identificadas no PAFP, desafiam o Estado a assumir o papel de regulador do acesso a medicamentos essenciais, de articulador da complexa rede de atores, públicos e privados e de promotor da equidade das necessidades de assistência farmacêutica, sendo um dos responsáveis pelo desenvolvimento econômico do Brasil, em suas dimensões econômica, política e social. / This thesis aims to present limits and potentialities of access policy to essential medicines, implemented by “Aqui Tem Farmácia Popular” Program (PAFP) from the standpoint of the country economic development. To reach this objective, a research with specific and connected approaches is proposed, giving rise to three papers. The first paper analyses the structure of the “Farmácia Popular do Brasil” Program (PFPB), based on the proposed dimensions of the World Health Organization (OMS) model together with the Management Sciences for Health (MSH), discussing concepts and approaches of the State role on the perspective of the economic development, access to medicines models and health sector indicators as part of economic development in Brazil. While featuring the PFPB, aligned elements have been identified to the dimensions availability, accessibility, acceptability, and affordability. The PAFP strategy showed to be economic, justifying that it is kept as the State priority strategy. Although, there are limits to be considered in order to have a better performance in that it is proposed, specially, related to the private pharmacy affordability dimension. In order to check the State role through actions within the pharmaceutical assistance, the second paper assesses qualitatively the PAFP, from the perspective of the users. All data were obtained through methodological procedure and a survey that has been developed specially for this study. It was evaluated the happening of the users migration from “Farmácia Básica” Program (PFB) of SUS to the PAFP and its motivations, including the evaluation of the attendance of its needs and its level of satisfaction. In relation to PAFP, the results point that there is no significant migration (16%) from PFB of SUS users to the PAFP, suggesting that there is no competition between both Programs, they are complementary. The research allowed recognizing that there was an increase of the access facility to medicines, supply of positive alternative for users, financial gain of user when using the PAFP, among others. It is noted, however, as limits, the question of unconstitutionality of Copayment System and the financial sustainability of the Program. The third paper presents elements that indicate integration aspects, or not, of the Health Economic Industrial Complex (CEIS) dynamic to the PAFP results, from the empiric survey to identify and compare the federal spending’s between the PFB of SUS and PAFP. The theoretical ground covers the relations of the economic development, technological innovation and the CEIS. The research data were achieved amongst 12 municipalities that composed the pilot study, on the perspective of the methodology application in all country. The comparative analysis of purchasing values of medicines between both nets, public and private, highlights that the purchase value in the private pharmacies are higher than the one at the public’s pharmacies. The analysis cost-volume allowed suggesting that the private pharmacy has compensations among medicines. It is concluded that, in fact, there is no full integration from CEIS. Despite the limits, the identified potentialities in PAFP challenge the State to play the role as an access to essential medicines regulator, as the complex public and private actors’ net articulator and as a promoter of the needs equity in the pharmaceutical assistance, being responsible for the Brazil economic development.

Esta tese tem por objetivo apresentar limitantes e potencialidades da política de acesso aos medicamentos essenciais, implementada através do Programa Aqui tem Farmácia Popular (PAFP) na perspectiva do desenvolvimento econômico do país. Para alcançá-lo, propôs-se a realização de uma pesquisa, com enfoques particulares, inter-relacionados, dando origem a três artigos. O primeiro artigo analisa a estrutura do Programa Farmácia Popular do Brasil (PFPB), tendo por base as dimensões propostas no modelo da Organização Mundial da Saúde (OMS) em conjunto com a Management Sciences for Health (MSH), discutindo conceitos e abordagens do papel do Estado na perspectiva do desenvolvimento econômico, modelos de acesso a medicamentos e indicadores do setor saúde no contexto do desenvolvimento econômico no Brasil. Ao caracterizar o PFPB, identificaram-se elementos alinhados às dimensões disponibilidade, aceitabilidade, acessibilidade geográfica e poder de compra. A estratégia do PAFP mostrou-se ser econômica, justificando manter-se como estratégia prioritária do Estado. Entretanto, há limitações a serem consideradas para que tenha melhor desempenho no que se propõe, em especial, em relação à dimensão do poder de compra da farmácia privada. Com o intuito de verificar o papel do Estado através de ações no âmbito da assistência farmacêutica, o segundo artigo avalia, qualitativamente, o PAFP, a partir da perspectiva dos usuários. Os dados foram obtidos através de procedimentos metodológicos e de instrumento desenvolvidos para tal. Avaliou-se a ocorrência de migração dos usuários do Programa Farmácia Básica (PFB) do SUS para o PAFP e suas motivações, incluindo a avaliação do atendimento de suas necessidades e o seu grau de satisfação em relação ao PAFP. Os resultados indicam que não há migração significativa (16%) de usuários do PFB do SUS para o PAFP, sugerindo que não há concorrência entre os Programas, sendo complementares. A pesquisa possibilitou reconhecer que houve ampliação da facilidade de acesso a medicamentos, oferta de alternativa positiva para os usuários, ganho financeiro do usuário com a utilização do PAFP, entre outros. Constata-se, entretanto, como limitantes, a questão da inconstitucionalidade do Sistema de Copagamento e a sustentabilidade financeira do Programa. O terceiro artigo apresenta elementos que apontam aspectos de integração, ou não, da dinâmica do Complexo Econômico-Industrial da Saúde (CEIS) aos resultados do PAFP, a partir da pesquisa empírica realizada para identificar e comparar os gastos federais entre o PFB do SUS e o PAFP. A fundamentação teórica abrange as relações do desenvolvimento econômico, inovação tecnológica e o CEIS. Os dados da pesquisa foram obtidos entre 12 municípios que compuseram o estudo piloto, na perspectiva da aplicação da metodologia em todo o país. A análise comparativa dos valores de compra de medicamentos entre as duas redes, pública e privada, evidenciou que o valor de compra pelas farmácias privadas é maior que o praticado pelas farmácias públicas. A análise custo-volume permitiu inferir que a farmácia privada tem compensações entre os medicamentos. Conclui-se que, de fato, não há integração plena do CEIS na promoção do PAFP. Apesar dos limitantes, as potencialidades identificadas no PAFP, desafiam o Estado a assumir o papel de regulador do acesso a medicamentos essenciais, de articulador da complexa rede de atores, públicos e privados e de promotor da equidade das necessidades de assistência farmacêutica, sendo um dos responsáveis pelo desenvolvimento econômico do Brasil, em suas dimensões econômica, política e social. / This thesis aims to present limits and potentialities of access policy to essential medicines, implemented by “Aqui Tem Farmácia Popular” Program (PAFP) from the standpoint of the country economic development. To reach this objective, a research with specific and connected approaches is proposed, giving rise to three papers. The first paper analyses the structure of the “Farmácia Popular do Brasil” Program (PFPB), based on the proposed dimensions of the World Health Organization (OMS) model together with the Management Sciences for Health (MSH), discussing concepts and approaches of the State role on the perspective of the economic development, access to medicines models and health sector indicators as part of economic development in Brazil. While featuring the PFPB, aligned elements have been identified to the dimensions availability, accessibility, acceptability, and affordability. The PAFP strategy showed to be economic, justifying that it is kept as the State priority strategy. Although, there are limits to be considered in order to have a better performance in that it is proposed, specially, related to the private pharmacy affordability dimension. In order to check the State role through actions within the pharmaceutical assistance, the second paper assesses qualitatively the PAFP, from the perspective of the users. All data were obtained through methodological procedure and a survey that has been developed specially for this study. It was evaluated the happening of the users migration from “Farmácia Básica” Program (PFB) of SUS to the PAFP and its motivations, including the evaluation of the attendance of its needs and its level of satisfaction. In relation to PAFP, the results point that there is no significant migration (16%) from PFB of SUS users to the PAFP, suggesting that there is no competition between both Programs, they are complementary. The research allowed recognizing that there was an increase of the access facility to medicines, supply of positive alternative for users, financial gain of user when using the PAFP, among others. It is noted, however, as limits, the question of unconstitutionality of Copayment System and the financial sustainability of the Program. The third paper presents elements that indicate integration aspects, or not, of the Health Economic Industrial Complex (CEIS) dynamic to the PAFP results, from the empiric survey to identify and compare the federal spending’s between the PFB of SUS and PAFP. The theoretical ground covers the relations of the economic development, technological innovation and the CEIS. The research data were achieved amongst 12 municipalities that composed the pilot study, on the perspective of the methodology application in all country. The comparative analysis of purchasing values of medicines between both nets, public and private, highlights that the purchase value in the private pharmacies are higher than the one at the public’s pharmacies. The analysis cost-volume allowed suggesting that the private pharmacy has compensations among medicines. It is concluded that, in fact, there is no full integration from CEIS. Despite the limits, the identified potentialities in PAFP challenge the State to play the role as an access to essential medicines regulator, as the complex public and private actors’ net articulator and as a promoter of the needs equity in the pharmaceutical assistance, being responsible for the Brazil economic development.

Esta tese tem por objetivo apresentar limitantes e potencialidades da política de acesso aos medicamentos essenciais, implementada através do Programa Aqui tem Farmácia Popular (PAFP) na perspectiva do desenvolvimento econômico do país. Para alcançá-lo, propôs-se a realização de uma pesquisa, com enfoques particulares, inter-relacionados, dando origem a três artigos. O primeiro artigo analisa a estrutura do Programa Farmácia Popular do Brasil (PFPB), tendo por base as dimensões propostas no modelo da Organização Mundial da Saúde (OMS) em conjunto com a Management Sciences for Health (MSH), discutindo conceitos e abordagens do papel do Estado na perspectiva do desenvolvimento econômico, modelos de acesso a medicamentos e indicadores do setor saúde no contexto do desenvolvimento econômico no Brasil. Ao caracterizar o PFPB, identificaram-se elementos alinhados às dimensões disponibilidade, aceitabilidade, acessibilidade geográfica e poder de compra. A estratégia do PAFP mostrou-se ser econômica, justificando manter-se como estratégia prioritária do Estado. Entretanto, há limitações a serem consideradas para que tenha melhor desempenho no que se propõe, em especial, em relação à dimensão do poder de compra da farmácia privada. Com o intuito de verificar o papel do Estado através de ações no âmbito da assistência farmacêutica, o segundo artigo avalia, qualitativamente, o PAFP, a partir da perspectiva dos usuários. Os dados foram obtidos através de procedimentos metodológicos e de instrumento desenvolvidos para tal. Avaliou-se a ocorrência de migração dos usuários do Programa Farmácia Básica (PFB) do SUS para o PAFP e suas motivações, incluindo a avaliação do atendimento de suas necessidades e o seu grau de satisfação em relação ao PAFP. Os resultados indicam que não há migração significativa (16%) de usuários do PFB do SUS para o PAFP, sugerindo que não há concorrência entre os Programas, sendo complementares. A pesquisa possibilitou reconhecer que houve ampliação da facilidade de acesso a medicamentos, oferta de alternativa positiva para os usuários, ganho financeiro do usuário com a utilização do PAFP, entre outros. Constata-se, entretanto, como limitantes, a questão da inconstitucionalidade do Sistema de Copagamento e a sustentabilidade financeira do Programa. O terceiro artigo apresenta elementos que apontam aspectos de integração, ou não, da dinâmica do Complexo Econômico-Industrial da Saúde (CEIS) aos resultados do PAFP, a partir da pesquisa empírica realizada para identificar e comparar os gastos federais entre o PFB do SUS e o PAFP. A fundamentação teórica abrange as relações do desenvolvimento econômico, inovação tecnológica e o CEIS. Os dados da pesquisa foram obtidos entre 12 municípios que compuseram o estudo piloto, na perspectiva da aplicação da metodologia em todo o país. A análise comparativa dos valores de compra de medicamentos entre as duas redes, pública e privada, evidenciou que o valor de compra pelas farmácias privadas é maior que o praticado pelas farmácias públicas. A análise custo-volume permitiu inferir que a farmácia privada tem compensações entre os medicamentos. Conclui-se que, de fato, não há integração plena do CEIS na promoção do PAFP. Apesar dos limitantes, as potencialidades identificadas no PAFP, desafiam o Estado a assumir o papel de regulador do acesso a medicamentos essenciais, de articulador da complexa rede de atores, públicos e privados e de promotor da equidade das necessidades de assistência farmacêutica, sendo um dos responsáveis pelo desenvolvimento econômico do Brasil, em suas dimensões econômica, política e social. / This thesis aims to present limits and potentialities of access policy to essential medicines, implemented by “Aqui Tem Farmácia Popular” Program (PAFP) from the standpoint of the country economic development. To reach this objective, a research with specific and connected approaches is proposed, giving rise to three papers. The first paper analyses the structure of the “Farmácia Popular do Brasil” Program (PFPB), based on the proposed dimensions of the World Health Organization (OMS) model together with the Management Sciences for Health (MSH), discussing concepts and approaches of the State role on the perspective of the economic development, access to medicines models and health sector indicators as part of economic development in Brazil. While featuring the PFPB, aligned elements have been identified to the dimensions availability, accessibility, acceptability, and affordability. The PAFP strategy showed to be economic, justifying that it is kept as the State priority strategy. Although, there are limits to be considered in order to have a better performance in that it is proposed, specially, related to the private pharmacy affordability dimension. In order to check the State role through actions within the pharmaceutical assistance, the second paper assesses qualitatively the PAFP, from the perspective of the users. All data were obtained through methodological procedure and a survey that has been developed specially for this study. It was evaluated the happening of the users migration from “Farmácia Básica” Program (PFB) of SUS to the PAFP and its motivations, including the evaluation of the attendance of its needs and its level of satisfaction. In relation to PAFP, the results point that there is no significant migration (16%) from PFB of SUS users to the PAFP, suggesting that there is no competition between both Programs, they are complementary. The research allowed recognizing that there was an increase of the access facility to medicines, supply of positive alternative for users, financial gain of user when using the PAFP, among others. It is noted, however, as limits, the question of unconstitutionality of Copayment System and the financial sustainability of the Program. The third paper presents elements that indicate integration aspects, or not, of the Health Economic Industrial Complex (CEIS) dynamic to the PAFP results, from the empiric survey to identify and compare the federal spending’s between the PFB of SUS and PAFP. The theoretical ground covers the relations of the economic development, technological innovation and the CEIS. The research data were achieved amongst 12 municipalities that composed the pilot study, on the perspective of the methodology application in all country. The comparative analysis of purchasing values of medicines between both nets, public and private, highlights that the purchase value in the private pharmacies are higher than the one at the public’s pharmacies. The analysis cost-volume allowed suggesting that the private pharmacy has compensations among medicines. It is concluded that, in fact, there is no full integration from CEIS. Despite the limits, the identified potentialities in PAFP challenge the State to play the role as an access to essential medicines regulator, as the complex public and private actors’ net articulator and as a promoter of the needs equity in the pharmaceutical assistance, being responsible for the Brazil economic development.

Ziel: Entwicklung eines gesundheitsökonomischen Evaluationskonzepts zum Nachweis einer Kostenreduktion unter gleichzeitiger Optimierung des medizinischen Nutzens durch pharmakogenetisches Screening bei Erstdiagnose einer Schizophrenie. Finale Zielsetzung ist die Aufnahme der pharmakogenetischen a priori-Diagnostik für die Indikation Schizophrenie in die GKV-Regelversorgung. Methodik: Basierend auf dem aktuellen Stand gendiagnostischer Forschung sowie der evidenzbasierten Schizophrenietherapie wurde eine prospektive, randomisierte und kontrollierte, dreiarmige, offene, multizentrische Pilotstudie im Paralleldesign über 3 Jahre konzeptioniert. Studienpopulation: 300 Patienten (1:1:1) im Alter von 18 bis 65 Jahren mit erstmaliger F20-Diagnose (ICD-10). Interventionen: pharmakogenetisches Screening und integrierte Versorgung; integrierte Versorgung; Standardversorgung. Die Erhebung des medizinischen Nutzens erfolgt durch Messung des klinischen Outcome bzgl. der patientenrelevanten Endpunkte Mortalität, Morbidität, Lebensqualität und Nebenwirkungen zu definierten Zeitpunkten. Perspektivisch relevante Kosten werden im "piggy back"-Verfahren ermittelt. Ergebnisse: Angesichts zurzeit bestehender Limitationen im deutschen Gesundheitssystem (z.B. unzureichendes intersektorales Schnittstellenmanagement bei der Arzneimittelversorgung und Informationsweitergabe) wird die Integrierte Versorgung als geeignete Versorgungsform für den Nutzennachweis eingestuft. Die Integrierte Versorgung stellt jedoch momentan nicht den allgemeinen Standard der psychiatrischen Patientenversorgung dar. Aus GKV-Perspektive wesentliche Kostentreiber der Schizophrenietherapie sind Rückfälle, Krankenhausaufenthalte, Arbeitslosigkeit und vorzeitige Verrentung. Eine Verringerung der Häufigkeit dieser Parameter könnte z.B. zu einer Reduktion der Erstjahres-Behandlungskosten (zurzeit ca. 30% der Gesamtkosten) führen. Die Kosten-Effektivitäts-Analyse erweist sich als Studienform mit der geringsten Anfälligkeit für Bias und Confounder. Trotz einer vergleichsweise hohen externen Validität ist das Studiensetting nicht uneingeschränkt übertragbar auf die Versorgungsrealität des deutschen Gesundheitssystems. Es existiert aktuell keine generelle Empfehlung für den Einsatz der Gendiagnostik zur Steuerung der Arzneimitteltherapie in Psychiatrie. Ebenso hat die integrierte Versorgung bisher keinen umfassenden Einzug in den psychiatrischen Behandlungsalltag gefunden, so dass die beschriebenen Limitationen einen positiven Nutzennachweis erschweren. Dennoch ist das Konzept als praktisch umsetzbar zu bewerten. Schlussfolgerung: Bei dieser Faktenlage ist das Interesse der GKV an der Veranlassung einer gesundheitsökonomischen Evaluation mit dem Ziel einer Erstattungsfähigkeit des a priori durchgeführten pharmakogenetischen Screenings bei Schizophrenie als eher gering einzustufen. Jedoch lassen das Update der S3-Praxisleitlinie mit dem Einbezug der strukturierten u. integrierten Versorgung sowie der Aktionsplan „Individualisierte Medizin“ des Bundesforschungsministeriums auf eine Fokussierung auf diese Fragestellung und veränderte Interessenlage bzgl. der Initiierung der Pilotstudie hoffen. Weitere Forschungstätigkeit sowie die praktische Erprobung neuer gendiagnostischen Verfahren sind, basierend auf versorgungsbezogenen Pilotstudien wie der hier konzeptionierten, fachübergreifend erforderlich, um die Relevanz der Methodik für den psychiatrischen Versorgungsalltag zu belegen. / Purpose: Development of a health-economic investigation method to study whether a cost reduction under concurrent optimisation of the medical use exists by using pharmacogenetic a- priori- screening with first diagnosis of a schizophrenia. Final objective is the reimbursement of pharmacogenetic diagnostics for the indication schizophrenia in the German health statutory insurance (GKV). Methods: A prospective, randomised and controlled, 3-armed, parallel, open, multicentre pilot study with a duration of 3 years was designed based on the actual status of genetic-diagnostic research as well as the evidence-based therapy of schizophrenia. Study population: 300 patients (1:1:1) aged 18 to 65 years with initial F20 diagnosis (ICD-10). Interventions: pharmacogenetic screening and integrated care; integrated care; standard care. For evaluation of the medical benefit the clinical outcome is measured at defined times with regard to the patients' relevant endpoints mortality, morbidity, quality of life and side effects. In perspective relevant costs are determined by "piggy back" procedure. Results: In view of actually existing limitations within the German health system (e.g., insufficient intersectional medication and information management) the integrated care is considered being a suitable setting to demonstrate the advantage of using pharmacogenetic screening. Nevertheless, the integrated care does not show the general standard of the psychiatric patient's care at the moment. From GKV perspective essential cost drivers of schizophrenia therapy are relapses, hospital stays, unemployment and untimely superannuation. Diminishing the rate of these parametres could lead, e.g., to a reduction of the first year medical costs (at the moment approx. 30% of the total expenses). The cost-effectiveness analysis seems to be the study form with the slightest susceptibility to bias and confounding. In spite of a relatively high external validity the study setting is not unconditionally transferable to the German health system. Currently no general recommendation exists for the application of the genetic diagnostics to manage medication therapy in psychiatry. Up to now also the integrated care has not found a comprehensive entry in psychiatric practice, so that the described limitations are complicating a positive use proof. Nevertheless, the investigational concept can be regarded as feasible. Conclusion: Based on the existing situation the GKV's interest in performing a health-economic evaluation, which is focussed on the reimbursement of pharmacogenetic a priori-diagnostics in schizophrenia, is considered to be low. However, the situation may change in view of the expected update of the S3-practise guideline with the focus on structured and integrated care as well as the action plan „individualised medicine“ of the German federal research ministry. Thus, there is hope for changing interests in a pilot study. Based on care-related pilot studies as presented here, further research activities and practical testing of recent gene diagnostic procedures are necessary to demonstrate the relevance of the methodology for psychiatric practice.

Introduction: Quality improvement (QI) encompasses a wide range of healthcare studies and activities with the common goal of improving patient outcomes, healthcare system performance, and professional development. QI is characterized by a diversity of definitions, stakeholders, clinical fields and study designs, which creates challenges for rigorous reporting and evaluation of these studies. Understanding and addressing the methodological issues that arise from conducting QI studies from multiple clinical disciplines is critical for generating good evidence for healthcare improvement to tackle health system challenges. Objectives: This thesis addressed three independent objectives: (i) Determine the quality of reporting of QI studies in neonatology. (ii) Compare different statistical methods that can be used to analyze data from a cluster randomized controlled trial with repeated measures data and examine how the estimate of intervention effects varies between these approaches. (iii) Determine the cost-effectiveness of providing timely surgery or timely rehabilitation for patients with hip fracture. Methods: Objective 1: We conducted a systematic survey of quality improvement studies in neonatology to examine the extent to which these publications adhered to SQUIRE 2.0, the guidelines for reporting studies that sought to improve the quality, safety, and value of healthcare. Using the same set of articles, we examined how various methodological attributes, such as stakeholder engagement, outcome measures, and statistical process controls, are reported in these studies. Objective 2: To compare the differences in the statistical estimates of intervention effects between linear mixed models and Generalized Estimating Equations, for the CP@Clinic Program cluster randomized RCT, which contains routinely collected monthly outcome data aggregated at the cluster level. Objective 3: We constructed a Markov cohort model to estimate the cost-effectiveness of receiving timely surgery within 24 hours of admission to the emergency department, receiving immediate admission to inpatient rehabilitation following acute care discharge, receiving both, or none. Results: Objective 1: In our assessment of reporting quality, we found that adherence to SQUIRE 2.0 guidelines was inadequate and that journals should endorse the SQUIRE 2.0 guideline for improvement publications to alleviate this issue. We found that process measures was the most frequently reported methodological attribute (89%), while stakeholder engagement with leadership (32%) or caregivers (10%) were infrequently reported or conducted. Objective 2: In comparing statistical methods for analyzing a cluster randomized controlled trial with correlated data, we found that it was critical to apply a correction to the variance estimator of generalized estimating equations to produce robust estimates of the intervention effects. Objective 3: In our economic evaluation, both timely surgery alone and the combination of timely surgery and timely rehabilitation yielded cost-effective improvements in the quality-adjusted life-years of patients with hip fracture. However, the combination of receiving timely surgery and timely rehabilitation requires a high willingness-to-pay threshold, above $128,000 per quality-adjusted life-years, to be considered cost-effective. Conclusions: Overall, understanding the state of reporting and the broad spectrum of methods and methodologic issues for evaluating quality improvement initiatives will advance its rigorous research, evaluation, reporting, and contribution towards informed decision-making for tackling pressing healthcare issues. / Thesis / Doctor of Philosophy (PhD) / Quality improvement (QI) is a field of healthcare research that can be defined in many ways, and research in this field is conducted by researchers from various medical disciplines. Consequently, challenges may arise in reporting and evaluating QI interventions. Thus, it is important to examine how QI interventions are reported in academic literature and the methods used to evaluate their effectiveness in improving health. The current thesis aims to address these issues through three independent objectives: (1) examine the details reported in QI studies in neonatology, (2) compare different statistical methods that can be used to analyze data from a community paramedicine cluster randomized controlled trial, and (3) investigate whether providing timely surgery and timely hospital-based rehabilitation is a cost-effective way to improve the quality of life of patients who have experienced hip fracture. The findings of these studies will provide insights into the challenges of reporting and evaluating QI interventions, and suggest ways to improve them.

Pour certaines maladies causées par des agents chimiques, biologiques, radiologiques et nucléaires (CBRN), il n’existe pas de contre-mesures médicales (MedCM) et bon nombre de celles qui existent pourraient ne pas être disponibles en cas de besoin. En cas d’accident CBRN, des efforts inadéquats de financement de la R&D et de mise à disposition par les gouvernements peuvent avoir de graves conséquences économiques nettement supérieures aux coûts d’initiatives préventives. Compte-tenu des contraintes budgétaires auxquelles de nombreux gouvernements sont confrontés, il est nécessaire de définir des priorités. Parallèlement à la mise en place d’indicateurs de décision de santé efficaces qui identifient et mesurent les effets de causalité de l’impact négatif sur la santé, le processus de décision doit également prendre en considération le rapport coût-efficacité pour rendre le financement durable.Cette thèse a pour objectif de définir une voie vers une politique économique de santé publique visant à renforcer la disponibilité des MedCM pour les agents CBRN. Dans la première partie, les causes des défaillances du marché sont identifiées (lorsque les opportunités de profit ne compensent pas l’effort de R&D nécessaire). Dans la deuxième partie, des études de cas illustrent les caractéristiques et les conséquences économiques d’exemples d’accidents CBRN et des scénarios sont analysés afin de mettre en évidence comment la disponibilité de MedCM pourrait potentiellement devenir rentable. Enfin, la troisième partie propose des approches plus complètes pour mesurer et compenser les facteurs contribuant à la défaillance du marché en appliquant des modèles économiques spécifiques. / For some diseases caused by chemical, biological, radiological, and nuclear (CBRN) agents, innovative medical countermeasures (MedCMs) do not exist while many of those that do might not be readily available. In case of a CBRN event, inappropriate medical research and development (R&D) funding and government procurement efforts can result in adverse economic consequences (e.g. lost income) far exceeding the costs of strong and comprehensive preparedness initiatives. Given the budgetary constraints many governments face, priorities must be defined. Parallel to determining effective health decision metrics that identify and weigh the causal effects of negative health impact, decision making must also consider cost-effectiveness to make funding sustainable. Moreover, international cooperation is necessary since the risks increasingly transcend borders due to global travel and the global threat of terrorism. This dissertation ultimately seeks to define a path to public health economic policy to enhance the international availability of CBRN MedCMs. In Part I, the root causes of market failure are identified and depicted (i.e., where rewards for supply do not adequately compensate for the R&D effort). In Part II, case study examples illustrate the characteristics and economic consequences of CBRN incidents. Scenarios for each case are outlined to show where the availability of MedCMs in these situations could potentially be cost-effective. Finally, Part III construes more comprehensive approaches for gauging and offsetting the deterrence factors of market supply and demand by compiling and applying additional economic models and frameworks.