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OBJECTIVE: Medication non-adherence in mental health problems has social and economic costs. The objective of the study was to review the cost-effectiveness of interventions to enhance medication adherence in patients with mental health problems. METHODS: The update of a previous systematic review was performed. Databases were searched in June 2019: MEDLINE, PSYCINFO, EMBASE, CINAHL, CRD, WOS. Cost-effectiveness studies comparing an intervention to improve the medication adherence with other interventions/usual care in adults with mental health problems were included. Data were extracted, methodological quality of the studies was assessed and a narrative synthesis was performed. RESULTS: Nine studies were included in the review. The interventions that showed medication adherence increase were: a financial incentive when depot injection was taken by patients with psychotic disorders, a value-based benefit design policy including copayment and counselling in a company setting, and a medication treatment decision supported by a pharmacogenetic test. The other studies (coaching by pharmacists; a psychological and educational intervention at health care centres) did not find differences between groups. No study found cost differences between alternatives. CONCLUSIONS: Interventions to improve medication adherence in adults with mental health problems could be cost-effective, especially those based on financial incentives, although more research is needed. KEYPOINTS There are several types of interventions designed to enhance medication adherence in patients with mental health problems. Few of them have demonstrated cost-effectiveness. Two studies found that a financial incentive per depot injection in patients with psychotic disorders improved the medication adherence. Two other studies found improvement in adherence due to two specific interventions: a value-based benefit design policy in a company setting and a pharmacogenetic test supporting the medication treatment decision. No study found differences in costs between the intervention and the comparator. More research is needed to implement cost-effective interventions.
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Análise Custo-Benefício , Adesão à Medicação , Transtornos Mentais/tratamento farmacológico , Motivação , Avaliação de Resultados em Cuidados de Saúde , Psicotrópicos/administração & dosagem , HumanosRESUMO
BACKGROUND: Knee osteoarthritis (OA) represents a heavy burden for patients and the society as a whole. The Oxford Knee Score (OKS) is a well known tool to assess the quality of life in patients with Knee OA. The purpose of this study was to analyze the psychometric properties of the Spanish version of the OKS, including its reliability, validity, and responsiveness. METHODS: Prospective observational study that included 397 patients diagnosed with knee OA according to the criterion of the American Rheumatism Association, which were recruited in 3 different Spanish regions. Their self-perceived health-related quality of life (HRQL) was assessed through 3 questionnaires: a generic one (the EQ-5D-5 L) and two specific ones adapted to Spanish (the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) and the Oxford Knee Score (OKS). The follow-up period was 6 months, and the acceptability of the OKS was evaluated, together with its psychometric properties, presence of ceiling and floor effects, validity, reliability, and sensitivity to change. RESULTS: The OKS was fully answered in 99.5% of cases, with no evidence of ceiling or floor effects. Its factor structure can be explained in a single dimension. Its discriminating capacity was very good compared to the groups generated by the WOMAC and the EQ-5D-5 L. The correlation of the OKS with the dimensions of the latter questionnaires was around 0.7. The test-retest reliability was excellent (ICC 0.993; CI 95%: 0.990-0.995) and so was its internal consistency (Cronbach's α = 0.920). The effect size was 0.7 for moderate improvements in the HQRL, which is similar to that of the dimensions of the WOMAC and greater than for the EQ-5D-5 L. The minimum clinically significant difference that was detected by the questionnaire was 6.1 points, and the minimum detectable change was 4.4 points. CONCLUSIONS: The Spanish-adapted version of the OKS is a useful, valid tool for assessing the perceived HRQL in patients suffering from knee OA, with psychometric properties similar to the WOMAC, and that allows for discriminating the patient's condition at a particular moment as well as for appraising changes over time.
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Osteoartrite do Joelho/psicologia , Qualidade de Vida , Inquéritos e Questionários/normas , Idoso , Feminino , Humanos , Masculino , Medição da Dor/psicologia , Estudos Prospectivos , Reprodutibilidade dos Testes , TraduçõesRESUMO
Cost-of-illness (COI) studies quantify the economic burden of a disease, including direct healthcare and non-healthcare costs and productivity losses. Different approaches can be adopted to evaluate the resources associated to a disease and to calculate the total costs. Prevalence-based studies estimate the total costs of a disease during a given period, while incidence-based studies measure lifetime costs from onset until death. Data can be collected from individuals, using a bottom-up approach, or from population statistics, using a top-down approach. Different perspectives are possible, but the broadest and also mostly used is the societal one. Appropriate discounting should be applied for future costs and a sensitivity analyses of main parameters should be performed. The main limitation of COI studies is that they don't account the outcomes or benefits of possible treatments.There is a lack of COI studies in the field of rare diseases. A multinational COI study (BURQOL-RD) evaluated recently the burden of 10 rare diseases in Europe, using a prevalence-based method with a bottom-up approach to quantify resources from a societal perspective, which is the mostly used methodology for COI studies in rare diseases; however, several other studies illustrate different approaches to conduct COI analysis in this field, such as incidence-based methods or narrower perspectives.COI studies are useful to inform policy-makers about the magnitude of a disease. To support correctly the decision-making process, it is necessary to identify the cost-drivers through COI studies with robust design and standardized methodology.
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Efeitos Psicossociais da Doença , Custos de Cuidados de Saúde , Gastos em Saúde , Renda , Doenças Raras/economia , Absenteísmo , Eficiência , Recursos em Saúde/economia , Humanos , Incidência , Modelos Econômicos , Prevalência , Qualidade de Vida , Anos de Vida Ajustados por Qualidade de Vida , Doenças Raras/diagnóstico , Doenças Raras/epidemiologia , Doenças Raras/terapia , Licença Médica/economia , Resultado do TratamentoRESUMO
OBJECTIVES: The methodological quality of an economic evaluation performed alongside a clinical trial can be underestimated if the paper does not report key methodological features. This study discusses methodological assessment issues on the example of a systematic review on cost-effectiveness of physiotherapy for knee osteoarthritis. METHODS: Six economic evaluation studies included in the systematic review and related clinical trials were assessed using the 10-question check-list by Drummond and the Physiotherapy Evidence Database (PEDro) scale. RESULTS: All economic evaluations were performed alongside a clinical trial but the studied interventions were too heterogeneous to be synthesized. Methodological quality of the economic evaluations reported in the papers was not free of drawbacks, and in some cases, it improved when information from the related clinical trial was taken into account. CONCLUSIONS: Economic evaluation papers dedicate little space to methodological features of related clinical trials; therefore, the methodological quality can be underestimated if evaluated separately from the trials. Future economic evaluations should follow more strictly the recommendations about methodology and the authors should pay special attention to the quality of reporting.
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Ensaios Clínicos como Assunto/normas , Osteoartrite do Joelho/reabilitação , Modalidades de Fisioterapia/economia , Literatura de Revisão como Assunto , Análise Custo-Benefício , Humanos , Anos de Vida Ajustados por Qualidade de Vida , Avaliação da Tecnologia Biomédica/normasRESUMO
BACKGROUND: Many health care systems claim to incorporate the cost-effectiveness criterion in their investment decisions. Information on the system's willingness to pay per effectiveness unit, normally measured as quality-adjusted life-years (QALYs), however, is not available in most countries. This is partly because of the controversy that remains around the use of a cost-effectiveness threshold, about what the threshold ought to represent, and about the appropriate methodology to arrive at a threshold value. OBJECTIVES: The aim of this article was to identify and critically appraise the conceptual perspectives and methodologies used to date to estimate the cost-effectiveness threshold. METHODS: We provided an in-depth discussion of different conceptual views and undertook a systematic review of empirical analyses. Identified studies were categorized into the two main conceptual perspectives that argue that the threshold should reflect 1) the value that society places on a QALY and 2) the opportunity cost of investment to the system given budget constraints. RESULTS: These studies showed different underpinning assumptions, strengths, and limitations, which are highlighted and discussed. Furthermore, this review allowed us to compare the cost-effectiveness threshold estimates derived from different types of studies. We found that thresholds based on society's valuation of a QALY are generally larger than thresholds resulting from estimating the opportunity cost to the health care system. CONCLUSIONS: This implies that some interventions with positive social net benefits, as informed by individuals' preferences, might not be an appropriate use of resources under fixed budget constraints.
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Análise Custo-Benefício/métodos , Avaliação da Tecnologia Biomédica/economia , Pesquisa Empírica , Humanos , Modelos Econômicos , Anos de Vida Ajustados por Qualidade de VidaRESUMO
BACKGROUND: Juvenile idiopathic arthritis (JIA) refers to a number of rare chronic inflammatory diseases. Although JIA imposes a significant societal burden, limited data are available on the cost of JIA. The study's objective is to quantify the socioeconomic burden of JIA patients in the United Kingdom (UK), along with their health-related quality of life (HRQoL). METHODS: A bottom-up, cross-sectional, cost-of-illness analysis of 23 patients was carried out. To collect data on demographic characteristics, health resource utilization, informal care, productivity losses and HRQoL, questionnaires were administered to and completed by patients or their caregivers. The EuroQol five dimensions (EQ-5D) instrument was used to measure HRQoL. RESULTS: This study found that the average annual cost for a JIA patient was 31,546, with direct health care costs equalling 14,509 (46.0 % of total costs), direct non-health care costs amounting to 8,323 (26.4 %) and productivity losses being 8,715 (27.6 %). This was calculated using unit costs for 2012. The largest expenditures on average were accounted for by early retirement (27.0 %), followed by informal care (24.1 %), medications (21.1 %), outpatient and primary health care visits (13.2 %) and diagnostic tests (7.9 %). Important differences existed between JIA patients in need of caregiver assistance and those with no need (39,469 vs. 25,452 respectively). Among adult JIA patients, mean EQ-5D index scores and visual analogue scale (VAS) scores were found to be 0.26 and 49.00 respectively; the same scores among caregivers were 0.66 and 67.14 respectively. CONCLUSION: JIA poses a significant cost burden on the UK society. Over half of the total average costs (54 %) are related to non-health care and productivity losses. HRQoL of JIA patients is considerably worse than the UK general population.
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Artrite Juvenil/economia , Efeitos Psicossociais da Doença , Custos de Cuidados de Saúde/estatística & dados numéricos , Qualidade de Vida , Adulto , Cuidadores/economia , Criança , Pré-Escolar , Estudos Transversais , Feminino , Gastos em Saúde/estatística & dados numéricos , Recursos em Saúde , Humanos , Masculino , Pessoa de Meia-Idade , Fatores Socioeconômicos , Inquéritos e Questionários , Reino UnidoRESUMO
BACKGROUND: This study aimed to determine the societal economic burden and health-related quality of life (HRQOL) of cystic fibrosis (CF) patients in the UK. METHODS: A bottom-up cost-of-illness, cross-sectional, retrospective analysis of 74 patients was conducted aiming to estimate the economic impact of CF. Data on demographic characteristics, health resource utilisation, informal care, productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) instrument. RESULTS: Using unit costs for 2012 we found that the average annual cost for a CF patient was 48,603, with direct health care costs amounting to 20,854 (42.9 % of total costs), direct non-health care costs being 21,528 (44.3 %) and indirect costs attributable to productivity losses being 6,222 (12.8 %). On average, the largest expenditures by far were accounted for by informal care (44.1 %), followed by medications (14.5 %), acute hospitalisations (13.9 %), early retirement (9.1 %) and outpatient and primary health care visits (7.9 %). Sharp differences existed depending on whether CF patients were in need of caregiver help (76,271 versus 26,335). In adult CF patients, mean EQ-5D index scores were 0.64 (0.93 in the general population) and mean EQ-5D visual analogue scale scores were 62.23 (86.84 in the general population); among caregivers, these scores were 0.836 and 80.85, respectively. DISCUSSION: Our analysis highlights the importance of the economic and quality of life consequences of CF from a societal perspective. The results highlight that beyond conventional costs such as acute hospitalisations, medication and outpatient and primary care visits, indirect costs related to informal care and early retirement, have significant societal implications. Similarly, our analysis showed that the average EQ-5D index score of adult CF patients was significantly lower than in the general population, an indication that a methodological bias may exist in using the latter in economic analyses. CONCLUSION: CF poses a significant cost burden on UK society, with non-health care and indirect costs representing 57 % of total average costs, and HRQOL being considerably lower than in the general population.
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Efeitos Psicossociais da Doença , Fibrose Cística/economia , Fibrose Cística/psicologia , Nível de Saúde , Pacientes Ambulatoriais , Qualidade de Vida , Meio Social , Adolescente , Adulto , Cuidadores/estatística & dados numéricos , Criança , Pré-Escolar , Estudos Transversais , Fibrose Cística/fisiopatologia , Feminino , Custos de Cuidados de Saúde/estatística & dados numéricos , Recursos em Saúde/economia , Humanos , Masculino , Assistência ao Paciente/economia , Atenção Primária à Saúde/economia , Aposentadoria/economia , Estudos Retrospectivos , Inquéritos e Questionários , Reino Unido , Adulto JovemRESUMO
OBJECTIVE: The aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with cystic fibrosis (CF) in Bulgaria. MATERIALS AND METHODS: We conducted a cross-sectional study of 33 patients with CF and 17 caregivers from Bulgaria. Data on socio-demographic characteristics, health resource utilisation, informal care, labor productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was evaluated with the EuroQol 5-domain (EQ-5D-3L) questionnaire. RESULTS: Median annual costs of CF in Bulgaria were 24 152 per patient in 2012 as a reference year. Median annual costs for children were found to be significantly higher than those for adults - 31 945 vs. 15 714 (p = 0.012). This outcome came from statistically significant differences in costs for main informal carer (p < 0.001) and costs for other informal carers (p = 0.022). As a single cost item, drugs had the biggest monetary impact. Median annual drug costs were 13 059. Bulgarian CF patients showed low HRQOL results - 50 median VAS score and 0.592 median health utilities. A quarter of patients even rated their health state as worse than death. CONCLUSION: CF patients from Eastern Europe remain a vulnerable population with risk factors for worse health outcomes. Our study provided a state-of-the art analysis that facilitates the elaboration, adoption and application of targeted public health policies to tackle CF-related problems at national and European level.
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Fibrose Cística/psicologia , Qualidade de Vida , Adolescente , Adulto , Bulgária , Criança , Efeitos Psicossociais da Doença , Estudos Transversais , Fibrose Cística/economia , Custos de Medicamentos , Feminino , Humanos , MasculinoRESUMO
BACKGROUND: Inherited epidermolysis bullosa (EB) is a clinically and genetically heterogeneous group of skin fragility disorders characterized by blister formation following minor trauma. Four major types are distinguished based on the level of cleavage within the skin. Most EB forms present severely disabling cutaneous and systemic signs and symptoms. Management relies on daily time-consuming and distressing topical medications, and symptomatic treatment of systemic findings. Disease manifestations, symptoms, and daily care strongly affect patient and caregiver quality of life (QoL). To date, there are two validated EB-specific questionnaires, the "Quality of Life in Epidermolysis Bullosa" (QOLEB) and the "Epidermolysis Bullosa Burden of Disease" (EB-BoD) for the evaluation of patient and family disease burden, respectively. The aim of our study was to develop an Italian translation of the two questionnaires and to pilot-test them. METHODS: The guidelines for translation and cross-cultural adaptation of health-related QoL measures were followed. Initially, two separate translations were generated for each questionnaire, and subsequently reconciled by an expert committee. This was followed by a back-translation process. The original texts and all translations underwent revision by the expert committee, resulting in definitive versions. The final versions were then tested in a pilot study involving cognitive debriefing in a group of 17 families, representative of all EB major types. RESULTS: The translation and reconciliation process led to minor changes to obtain semantic/idiomatic/cultural equivalence of the Italian versions with the original ones and to reconcile the questions with the answer options. The cognitive debriefing process showed a good understanding and did not require text modifications. CONCLUSIONS: The Italian versions of the QOLEB and EB-BoD provide valuable tools in everyday clinical practice of reference centers, and they allow the participation in multicenter international real-life observational studies as well as in controlled clinical trials. They enable the identification of disease-specific psychological and socioeconomic challenges for EB patients and their families, guiding targeted interventions to ensure appropriate and timely care.
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Epidermólise Bolhosa , Qualidade de Vida , Humanos , Efeitos Psicossociais da Doença , Epidermólise Bolhosa/diagnóstico , Epidermólise Bolhosa/terapia , Epidermólise Bolhosa/psicologia , Itália , Projetos Piloto , Qualidade de Vida/psicologia , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Percutaneous ethanol injection (PEI) has been shown to be a valuable treatment for thyroid nodular pathology and metastatic cervical adenopathies. OBJECTIVE: To evaluate the effectiveness, safety, and cost-effectiveness of PEI in thyroid nodular pathology and metastatic cervical adenopathies. METHODS: A systematic review (SR) using meta-analysis was conducted on the effectiveness and safety of PEI. A SR on cost-effectiveness was also performed. The SRs were conducted according to the methodology developed by the Cochrane Collaboration with reporting in accordance with the PRISMA statement. A cost-minimization analysis was carried out using a decision tree model. Assuming equal effectiveness between two minimally invasive techniques (PEI and radiofrequency ablation (RFA)), the model compared the costs of the alternatives with a horizon of six months and from the perspective of the Spanish National Health System. RESULTS: The search identified three RCTs (n=157) that evaluated PEI versus RFA in patients diagnosed with benign thyroid nodules: ninety-six patients with predominantly cystic nodules and sixty-one patients with solid nodules. No evidence was found on other techniques or thyroid nodular pathology. No statistically significant differences were observed between PEI and RFA in volume reduction (%), symptom score, cosmetic score, therapeutic success and major complications. No economic evaluations were identified. The cost-minimization analysis estimated the cost per patient of the PEI procedure at 326 compared to 4781 for RFA, which means an incremental difference of -4455. CONCLUSIONS: There are no differences between PEI and RFA regarding their safety and effectiveness, but the economic evaluation determined that the former option is cheaper.
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Ablação por Cateter , Linfadenopatia , Nódulo da Glândula Tireoide , Humanos , Nódulo da Glândula Tireoide/patologia , Análise Custo-Benefício , Ablação por Cateter/métodos , Etanol/uso terapêutico , Linfadenopatia/tratamento farmacológico , Linfadenopatia/cirurgiaRESUMO
All EU countries have introduced Human papilloma virus (HPV) vaccination for adolescent girls and many countries are expanding the strategy to include adolescent boys. There is uncertainty about the cost-effectiveness and epidemiological impact of a gender-neutral HPV vaccination strategy. Here we present the results of an economic model adapted for Spain. Five vaccination strategies were compared from the Spanish healthcare system perspective, combining two vaccines (4-valent and 9-valent) in a gender-neutral or girls-only programme in a dynamic population-based model with a discrete-time Markov approach. Costs and benefits were discounted at 3%. The benefits of immunization were measured with quality-adjusted life years (QALYs), which are achieved by reducing the incidence of diseases attributable to HPV. Incremental cost-effectiveness ratio (ICER) was compared with the willingness-to-pay threshold in Spain. The two most effective strategies were compared: gender-neutral 9-valent vaccination vs. girls-only 9-valent vaccination, resulting in an ICER of 34,040/QALY, and an important number of prevented cases of invasive cancers and anogenital warts. The sensitivity analysis revealed that gender-neutral 9-valent vaccination would become cost-effective if protection against oropharyngeal and penile cancers was included or if the price per dose decreased from 45 to 28. The gender-neutral 9-valent HPV vaccination in Spain offers more benefits than any other modeled strategy, although in the conservative base case it is not cost-effective. However, certain plausible assumptions would turn it into an efficient strategy, which should be borne in mind by the decision makers together with equity and justice arguments.
What is the context? Human papillomavirus (HPV) is a group of viruses that causes sexually transmitted diseases, including certain cancers. European countries offer HPV vaccination to adolescent girls.Many countries have also introduced the vaccination in adolescent boys.There are doubts about whether it is worth vaccinating adolescents of both genders.What this study adds? We estimated costs and benefits of the vaccination in Spain, comparing two types of vaccine, only in girls and in both genders. This analysis considered indirect protection of vaccinated people to unvaccinated ones.It seems that the benefits of HPV vaccination in all adolescents do not compensate the costs.However, when we included likely protection against additional cancers or we reduced the price per vaccine dose, it would be worth vaccinating all adolescents in Spain.We also stated ethical arguments in favor of HPV vaccination for both genders.What is the impact? HPV vaccination in adolescent boys and girls in Spain will prevent more HPV-related diseases.For certain scenarios, HPV vaccination in both genders would be worth in Spain.Ethically, vaccinating also boys would be fair, equitable and would not discriminate a part of the population.
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Infecções por Papillomavirus , Vacinas contra Papillomavirus , Neoplasias do Colo do Útero , Adolescente , Masculino , Feminino , Humanos , Infecções por Papillomavirus/epidemiologia , Infecções por Papillomavirus/prevenção & controle , Análise Custo-Benefício , Neoplasias do Colo do Útero/epidemiologia , Neoplasias do Colo do Útero/prevenção & controle , Vacinação , Anos de Vida Ajustados por Qualidade de VidaRESUMO
Most of rare disease (RD) patients are assisted in their homes by their family as informal caregivers, causing a substantial burden among family members devoted to care. The role of informal caregivers has been associated with increased levels of stress, poor physical/mental health and impaired HRQOL. The present study assessed the impact on HRQOL and perceived burden of long-term informal caregiving, as well as the inter-relationships of individuals affected by different RD in six European countries, taking advantage of the data provided by the BURQOL-RD project (France, Germany, Italy, Spain, Sweden and UK). Correlation analysis was used to explore the relation between caregiver HRQOL and caregiver burden (Zarit Burden Interview). Multinomial logistic regression models were used to explore the role of explanatory variables on each domain of caregivers HRQOL measured by EQ-5D. Caregivers' HRQOL is inversely correlated with burden of caring. Mobility dimension of EQ-5D was significantly associated with patients age, time devoted to care by secondary caregivers, patient gender and patient utility index. Patients' age, burden scores and patient utility index significantly predict the capacity of caregivers to perform activities of daily living. Employed caregivers are less likely of reporting 'slight problems' in pain/discomfort dimensions than unemployed caregivers. The EQ-5D instrument is sensitive to measure differences in HRQOL between caregivers with different levels of burden of care.
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Cuidadores , Qualidade de Vida , Atividades Cotidianas , Cuidadores/psicologia , Efeitos Psicossociais da Doença , Estudos Transversais , Humanos , Qualidade de Vida/psicologia , Doenças Raras , Inquéritos e QuestionáriosRESUMO
BACKGROUND: When healthcare budgets are exogenous, cost-effectiveness thresholds (CETs) used to inform funding decisions should represent the health opportunity cost (HOC) of such funding decisions, but HOC-based CET estimates have not been available until recently. In recent years, empirical HOC-based CETs for multiple countries have been published, but the use of these CETs in the cost-effectiveness analysis (CEA) literature has not been investigated. Analysis of the use of HOC-based CETs by researchers undertaking CEAs in countries with different decision-making contexts will provide valuable insights to further understand barriers and facilitators to the acceptance and use of HOC-based CETs. OBJECTIVES: We aimed to identify the CET values used to interpret the results of CEAs published in the scientific literature before and after the publication of jurisdiction-specific empirical HOC-based CETs in four countries. METHODS: We undertook a scoping review of CEAs published in Spain, Australia, the Netherlands and South Africa between 2016 (2014 in Spain) and 2020. CETs used before and after publication of HOC estimates were recorded. We conducted logit regressions exploring factors explaining the use of HOC values in identified studies and linear models exploring the association of the reported CET value with study characteristics and results. RESULTS: 1171 studies were included in this review (870 CEAs and 301 study protocols). HOC values were cited in 28% of CEAs in Spain and in 11% of studies conducted in Australia, but they were not referred to in CEAs undertaken in the Netherlands and South Africa. Regression analyses on Spanish and Australian studies indicate that more recent studies, studies without a conflict of interest and studies estimating an incremental cost-effectiveness ratio (ICER) below the HOC value were more likely to use the HOC as a threshold reference. In addition, we found a small but significant impact indicating that for every dollar increase in the estimated ICER, the reported CET increased by US$0.015. Based on the findings of our review, we discuss the potential factors that might explain the lack of adoption of HOC-based CETs in the empirical CEA literature. CONCLUSIONS: The adoption of HOC-based CETs by identified published CEAs has been uneven across the four analysed countries, most likely due to underlying differences in their decision-making processes. Our results also reinforce a previous finding indicating that CETs might be endogenously selected to fit authors' conclusions.
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Orçamentos , Custos de Cuidados de Saúde , Austrália , Análise Custo-Benefício , Humanos , Países Baixos , Anos de Vida Ajustados por Qualidade de VidaRESUMO
LAY ABSTRACT: Professional guidance and support in response to first concerns appears to be an important predictor of the level of satisfaction with the detection process of autism in young children. In this study, we analyzed the views of 1342 family members, including 1278 parents, who completed an online survey form collecting information about their experience and satisfaction with the early detection of autism in their child. Specifically, we were interested in how specific experiences with the detection process relate to the satisfaction with it and whether we could identify important predictors of satisfaction. The detection process is an emotionally charged period for parents, often described as painful, chaotic, and lengthy. A better understanding of their experiences is important to take appropriate action to improve the detection process. In our sample, the level of satisfaction with the detection process varied greatly from one respondent to another. Among the different experiences we considered, whether or not respondents received professional guidance and support in response to first concerns explained most of this variation. We also found that difficulty finding information about detection services, lack of professional guidance and support in response to first concerns, having to find a diagnostic service on one's own, and longer delays between confirmation of concerns and first appointment with a specialist were experiences associated with a greater likelihood of being unsatisfied. The findings of this study highlight the importance of the parent-professional relationship in the detection process and have important practical implications for health administrations to improve the detection process.
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Transtorno do Espectro Autista , Transtorno Autístico , Criança , Humanos , Pré-Escolar , Transtorno Autístico/diagnóstico , Transtorno Autístico/psicologia , Satisfação Pessoal , Transtorno do Espectro Autista/diagnóstico , Transtorno do Espectro Autista/psicologia , Pais/psicologia , FamíliaRESUMO
OBJECTIVE: The aim of this scoping review was to overview the cost-of-illness studies conducted in rare diseases. METHODS: We searched papers published in English in PubMed from January 2007 to December 2018. We selected cost-of-illness studies on rare diseases defined as those with prevalence lower than 5 per 10,000 cases. Studies were selected by one researcher and verified by a second researcher. Methodological characteristics were extracted to develop a narrative synthesis. RESULTS: We included 63 cost-of-illness studies on 42 rare diseases conducted in 25 countries, and 9 systematic reviews. Most studies (94%) adopted a prevalence-based estimation, where the predominant design was cross-sectional with a bottom-up approach. Only four studies adopted an incidence-based estimation. Most studies used questionnaires to patients or caregivers to collect resource utilisation data (67%) although an important number of studies used databases or registries as a source of data (48%). Costs of lost productivity, non-medical costs and informal care costs were included in 68%, 60% and 43% of studies, respectively. CONCLUSION: This review found a paucity of cost-of-illness studies in rare diseases. However, the analysis shows that the cost-of-illness studies of rare diseases are feasible, although the main issue is the lack of primary and/or aggregated data that often prevents a reliable estimation of the economic burden.
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Efeitos Psicossociais da Doença , Doenças Raras , Cuidadores , Estudos Transversais , Custos de Cuidados de Saúde , HumanosRESUMO
OBJECTIVES: Surgery is the treatment of choice for symptomatic disc herniation after conservative management. Several studies have suggested the potential utility of intradiscal ozone infiltration in this pathology. The aim of this trial was to compare intradiscal ozone infiltration vs. oxygen infiltration vs. surgery. DESIGN AND INTERVENTIONS: This was a randomized, double-blinded, and controlled trial in patients on a waiting list for herniated disc surgery. There were three treatment groups: surgery; intradiscal ozone infiltration (plus foraminal infiltration of ozone, steroids, and anesthetic); intradiscal oxygen infiltration (plus foraminal infiltration of oxygen, steroids, and anesthetic). MAIN OUTCOME MEASURES: The requirements for surgery. RESULTS: Five years after the treatment of the last recruited patient (median follow-up: 78 months), the requirement for further surgery was 20 % for patients in the ozone group and 60 % for patients in the oxygen group. 11 % of patients initially treated with surgery also required a second surgery. Compared to the surgery group, the ozone group showed: 1) significantly lower number of inpatient days: median 3 days (interquartile range: 3-3.5 days) vs. 0 days (interquartile range: 0-1.5 days), p = 0.012; 2) significantly lower costs: median EUR 3702 (interquartile range: EUR 3283-7630) vs. EUR 364 (interquartile range: EUR 364-2536), p = 0.029. CONCLUSIONS: Our truncated trial showed that intradiscal ozone infiltrations decreased the requirements for conventional surgery, resulting in decreased hospitalization durations and associated costs. These findings and their magnitude are of interest to patients and health services providers. Further validation is ongoing.
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Degeneração do Disco Intervertebral , Deslocamento do Disco Intervertebral , Dor Lombar , Ozônio , Humanos , Degeneração do Disco Intervertebral/cirurgia , Deslocamento do Disco Intervertebral/cirurgia , Vértebras Lombares/cirurgia , Ozônio/uso terapêutico , Resultado do TratamentoRESUMO
BACKGROUND: Human papillomavirus (HPV) infection can have severe consequences both in women and men. Preadolescent girls are vaccinated against HPV worldwide but a gender-neutral vaccination is being adopted only little by little. This systematic review offers an overview of cost-effectiveness of the gender-neutral HPV vaccination. METHODS: Economic evaluations of gender-neutral HPV vaccination with a two-dose schedule compared with girls-only strategy were systematically searched in Medline, Embase and WOS up to June 2020. Incremental cost-effectiveness ratios and key parameters were analysed. RESULTS: Nine studies met the inclusion criteria. Four studies concluded in favour of the gender-neutral programme, another four found it cost-effective only in alternative scenarios. The most influential parameters are the discount rate of benefits (same as for costs vs reduced), vaccine price (listed vs publicly negotiated) and included health problems (inclusion of oropharyngeal and penile cancers). Sponsorship was not decisive for the final result, but there were differences between industry-funded and independent studies in some cost categories. CONCLUSIONS: The evidence of the cost-effectiveness of extending HPV vaccination to boys is scarce and ambiguous. Before the adoption of such a strategy, countries should carry out context-specific cost-effectiveness analyses, but the decision should also take into account other criteria, such as gender-related equality.
Assuntos
Infecções por Papillomavirus , Vacinas contra Papillomavirus , Neoplasias do Colo do Útero , Análise Custo-Benefício , Feminino , Humanos , Masculino , Infecções por Papillomavirus/prevenção & controle , Anos de Vida Ajustados por Qualidade de Vida , VacinaçãoRESUMO
BACKGROUND: The Stepped Care Model (SCM) proposes a sequential approach in the treatment of depression, applying interventions of increasing intensity according to the level of severity of the individual. METHODS: A systematic review was carried out until January 2020 in eight electronic databases. We included randomized (RCT) and non-randomized controlled trials comparing the SCM to usual non-sequential care. RESULTS: Eighteen RCT with patients with confirmed or probable depression diagnosis were included. Meta-analyses yielded high heterogeneity, and subgroup analyses showed significant effects of the SCM only in studies with baseline moderately severe symptoms on average, compared to samples with mild/moderate depression. In the former subgroup, effects at 3-6 and 9-12 months were small for symptoms' reduction (g = -0.33, 95%CI: -0.55, -0.17 and -0.34, 95%CI: -0.53, -0.16) and moderate-to-strong in response and remission (Risk Ratios between 1.70-1.90). Overall, a significant benefit on quality of life was also observed (6 months: g = 0.31, 95%CI: 0.12, 0.49; 12 months: g = 0.18, 95%CI: 0.06, 0.31). More patients in the SCM groups were prescribed antidepressants at 6 months (RR = 1.31, 95%CI: 1.09, 1.57; I² = 87%). LIMITATIONS: The search does not guarantee the identification of all the relevant literature. Most included studies show uncertain or high risk of bias. CONCLUSIONS: The SCM seems to outperform usual care in populations with at least moderately severe symptoms on average. Results show high heterogeneity and future research should explore its sources.
Assuntos
Depressão , Qualidade de Vida , Antidepressivos/uso terapêutico , Depressão/terapia , HumanosRESUMO
This study evaluated the cost-effectiveness of platelet-rich plasma (PRP) added to usual care versus usual care alone in elderly patients with chronic diabetic foot ulcer (DFU) from the Spanish health care system perpective. A 6-state Markov model with 3-month cycles was used to estimate costs and outcomes of wound healing and risk of recurrences, infections, and amputations over 5 years. Three treatment strategies were compared: (a) usual care plus PRP obtained with a commercial kit, (b) usual care plus PRP obtained manually, and (c) usual care. Data on effectiveness were taken from a recent meta-analysis. Outcomes and costs were discounted at 3% and resources were valued in 2018 euro. Compared with usual care, the PRP treatment with the manual method was more effective and less costly (dominant option), whereas the PRP treatment with the commercial kit was more effective but also more costly, with the incremental ratio being above the cost-effectiveness threshold (57 916 per quality-adjusted life year). These results are sensitive to the price of PRP kits (a 20% discount would make the PRP treatment a cost-effective option) and effectiveness data, due to the heterogeneity of primary studies. In conclusion, PRP treatment for DFUs could be considered a cost-effective or even cost-saving alternative in Spain, depending on the method of obtaining the PRP. Despite the dominance of the manual method, its general use is limited to hospitals and specialized centers, whereas PRP kits could be used in primary care settings, but their prices should be negotiated by health authorities.
Assuntos
Diabetes Mellitus , Pé Diabético , Plasma Rico em Plaquetas , Idoso , Análise Custo-Benefício , Pé Diabético/epidemiologia , Pé Diabético/terapia , Humanos , Espanha/epidemiologia , CicatrizaçãoRESUMO
Newborn screening programs are a fundamental tool for secondary prevention or pre-symptomatic detection of certain conditions. The implementation of a newborn screening program requires an evaluation of effectiveness, safety, cost-effectiveness, feasibility and budget impact. Economic evaluation aims to contribute to the sustainability and solvency of health systems, especially when it comes to informing about financing health interventions with public funds. This funding must be justified on the basis of robust evidence of effectiveness, safety, cost-effectiveness, and acceptability. One of the most important limitations when evaluating the cost-effectiveness of a newborn screening program for hereditary disorders or congenital errors of metabolism is the scarcity of scientific evidence that limits the robustness of the economic analysis. Given the low availability of data, the use of expert opinion as a data source is unavoidable to complete the information. However, two main problems make it difficult to synthesize data obtained from various sources: biases and heterogeneity. Moreover, the measurement of quality-adjusted life years (QALYs) in pediatric populations poses serious methodological challenges. In Spain, although there is some heterogeneity in the supply of newborn screening programs between regions, guidelines are being established based on the best available scientific evidence to achieve the homogenization of newborn screening policies and programs at national level.
Los programas de cribado neonatal son una herramienta fundamental para la prevención secundaria o detección presintomática de determinadas afecciones. La implantación de un programa de cribado neonatal requiere necesariamente de una evaluación de su efectividad, seguridad, coste-efectividad, factibilidad e impacto presupuestario. La evaluación económica pretende contribuir a la sostenibilidad y solvencia de los sistemas sanitarios, especialmente a la hora de informar sobre la posible financiación, con fondos públicos, de intervenciones sanitarias como el cribado poblacional. Esta financiación debe justificarse en base a pruebas robustas de efectividad, seguridad, coste-efectividad y aceptabilidad. Una de las limitaciones más importantes a la hora de evaluar el coste-efectividad de un programa de cribado neonatal de trastornos hereditarios o de errores congénitos del metabolismo es la escasez de evidencia científica que limita la solidez y robustez del análisis de evaluación económica. Dada la baja disponibilidad de datos, el uso de la opinión de expertos como fuente de datos es inevitable para completar la información necesaria. Sin embargo, dos problemas principales dificultan la síntesis de datos obtenidos de varias fuentes: sesgos y heterogeneidad. Por otro lado, la medición de los años de vida ajustados por calidad (AVAC) en poblaciones pediátricas plantea serios desafíos metodológicos en un análisis de evaluación económica. En España, aunque existe cierta heterogeneidad en la oferta de programas de cribado neonatal entre CC.AA., se están estableciendo directrices basadas en la mejor evidencia científica disponible para conseguir la homogeneización de políticas y programas de cribado neonatal a nivel nacional.