Emicizumab prophylaxis for people with hemophilia A: Waste estimation and the Brazilian perspective.

Costs of hemophilia A treatment are increasing. Waste of clotting products should be avoided. To estimate the first-year waste of emicizumab prophylaxis for people with hemophilia A and inhibitors (PwHAi) who failed immune tolerance induction (ITI), in Brazil. We evaluated the manufacturer and the Brazilian Ministry of Health (MoH) protocol-recommended regimens in a budget impact model. The loading dose consisted of 3.0 mg/kg/Q1W for 4 weeks, for both recommendations. The manufacturer maintenance regimens comprised 1.5 mg/kg/Q1W, 3.0 mg/kg/Q2W, and 6.0 mg/kg/Q4W. The MoH protocol maintenance regimen encompassed a hybrid Q1W/Q2W administration, depending on the body weight. The Q4W regimen was not recommended by the MoH protocol. Analyses were performed to estimate waste given its expense based on the World Health Organization body weight range (percentiles [P] 15, 50, and 85). The first-year emicizumab waste was estimated individually and for the disclosed PwHAi who failed ITI (n = 114). The highest emicizumab waste was estimated for the lowest body weights and the Q1W regimen. The Q4W regimen resulted in the lowest emicizumab waste, followed by the MoH protocol regimen. The total reconstituted costs estimated for the PwHAi who failed ITI according to the hybrid MoH protocol ranged from US$32,858,777 (P15) to US$47,186,858 (P85), with emicizumab waste ranging from 7.9 % (US$2,594,515) to 3.7 % (US$1,738,750), respectively. Lost resources due to current protocols for emicizumab prophylaxis for PwHAi who failed ITI in Brazil are considerable. Waste was more pronounced due to lower body weight and shorter administration intervals.

A Cross-Sectional Study of Quality of Life Among Brazilian Adults With Type 1 Diabetes Treated With Insulin Glargine: Findings and Implications.

This article describes a cross-sectional study involving 401 adults with type 1 diabetes treated with insulin glargine in Minas Gerais, Brazil. Health-related quality of life was assessed, and worse scores were found to be associated with a low level of education, self-perceived health reported as poor/very poor, being bedridden and not physically exercised, having seen a doctor more than four times in the past year, and having reported comorbidities and episodes of hypoglycemia.

Validity of the Frequency of Suicidal Ideation Inventory in Brazilian adults.

We examined the validity of the Brazilian Frequency of Suicidal Ideation Inventory (FSII-Br), an adaptation of the Frequency of Suicidal Ideation Inventory (FSII), in 946 Brazilian undergraduates. Confirmatory factor analysis of the FSII-Br demonstrated a one-factor model accounting for 62.39% of the variance in FSII-Br scores with a good FIT index. Correlation analyses demonstrated that the FSII-Br was positively correlated with both measures of suicide ideation and suicide risk (viz., depressive symptoms, anxious symptoms, and hopelessness), providing construct validity. Thus, the FSII-Br was found to be a promising tool in assessing suicide risk among Brazilian adults.

Real-world evaluation of the impact of statin intensity on adherence and persistence to therapy: A Scottish population-based study.

AIM: To assess associations between statin intensity and adherence, persistence and discontinuation of statin therapy in Scotland. METHOD: Retrospective cohort study, using linked electronic health records covering a period from January 2009 to December 2016. The study cohort included adult patients (≥18 years) newly initiating statins within Greater Glasgow and Clyde, Scotland. Study outcomes comprised adherence, discontinuation and persistence to treatment, stratified by three exposure groups (high, moderate and low intensity). Discontinuation and persistence were calculated using the refill-gap and anniversary methods, respectively. Proportion of days covered (PDC) was used as a proxy for adherence. Kaplan-Meier survival curves and Cox proportional hazard models were used to evaluate discontinuation, and associations between adherence/persistence and statin intensity were assessed using logistic regression. RESULTS: A total of 73 716 patients with a mean age of 61.4 ± 12.6 years were included; the majority (88.3%) received moderate intensity statins. Discontinuation rates differed between intensity levels, with high-intensity patients less likely to discontinue treatment compared to those on moderate intensity (prior cardiovascular disease [CVD]: HR 0.43 [95% CI 0.34-0.55]; no prior CVD: 0.80 [0.74-0.86]). Persistence declined over time, and high-intensity patients had the highest persistence rates. Overall, 52.6% of patients were adherent to treatment (PDC ≥ 80%), but adherence was considerably higher among high-intensity patients (63.7%). CONCLUSION: High-intensity statins were associated with better persistence and adherence to treatment, but overall long-term persistence and adherence remain a challenge, particularly among patients without prior CVD. This needs addressing.

[Suicide and indigenous populations in Brazil: systematic reviewEl suicidio y los pueblos indígenas brasileños: revisión sistemática]. / Suicídio e povos indígenas brasileiros: revisão sistemática.

OBJECTIVE: To describe the frequency, characteristics, and factors contributing to suicide in indigenous populations in Brazil. METHOD: A systematic review of the literature was performed in PubMed, SciELO, PsycINFO, and LILACS. All population-based studies focusing on suicide among indigenous populations in Brazil were included. RESULTS: The search identified 111 articles, of which nine met the inclusion criteria. Three of these studies were performed in the Midwest and four in the North of Brazil, while two covered all Brazilian regions. The ethnic groups investigated were specified in three studies (Terena, Kadiweu, Guato, Ofaie-Xavante, Guarani, Guarani-Kaiowá, and Guarani-Nandeva). Suicide rates were highest among males, single individuals, those with 4 to 11 years of schooling, and those aged 15 to 24 years. Suicides occurred most often in the home and on weekends, mostly by hanging. The main risk factors for suicide identified in the articles were poverty, historical and cultural factors, poor wellbeing indicators, family disintegration, social vulnerability, and lack of life or future perspective. CONCLUSIONS: All the studies indicated the need to engage communities in developing strategies, considering their cosmovision and the social, historic, and cultural view of each ethnic group to minimize risk factors and reduce suicide rates.

OBJETIVO: Describir la frecuencia y las características del suicidio y sus factores contribuyentes en los pueblos indígenas brasileños. MÉTODO: Se realizó una revisión sistemática de la bibliografía a partir de las bases de datos PubMed, SciELO, PsycINFO y LILACS. Se incluyeron estudios de base poblacional enfocados en el suicidio en las poblaciones indígenas en el territorio brasileño. RESULTADOS: En la búsqueda se encontraron 111 artículos, de los cuales nueve se ciñeron a los criterios de inclusión. Se realizaron tres estudios en la región centro oeste y cuatro en la región norte, además de otros dos que abarcaron todas las regiones de Brasil. En tres estudios se citaron todas las siete etnias estudiadas (Terena, Kadiweu, Guato, Ofaie-Xavante, Guaraní, Guaraní-Kaiowá y E-Nandeva). En los estudios se demostró una mayor tasa de mortalidad por suicidio en personas solteras del sexo masculino, con 4 a 11 años de escolaridad, en el grupo de 15 a 24 años de edad, en el domicilio y en los fines de semana, principalmente por el método de ahorcamiento. Los principales factores de riesgo de suicidio fueron la pobreza, factores históricos y culturales, el índice de bienestar bajo, la desintegración de las familias, la vulnerabilidad social, y la falta de sentido de la vida y del futuro. CONCLUSIONES: Todos los estudios indicaron la necesidad de formular estrategias junto con las comunidades, dentro del marco de su cosmovisión y de los aspectos sociales, históricos y culturales de cada etnia, con el fin de reducir al mínimo los factores de riesgo y la tasa de mortalidad por suicidio.

Confidentiality agreements: a challenge in market regulation.

BACKGROUND: Sustainability and the ability to maintain the right to health, with the guarantee of access to quality medicines and health services, have been a great challenge for countries with universal health systems. The great technological advances bring with it an expressive increase in the expenditures of the health systems, especially those directed towards the acquisition of high-cost drugs, which are still under patent protection, have a high cost and, in some cases, present uncertainties about their effectiveness and safety. As a way of maintaining the proper functioning of the systems and guaranteeing access to these medicines, some countries started to negotiate discounts with manufacturing companies. Pricing agreements have been adopted by developed countries with the objective of reducing their spending on high-cost medicines and, although they represent an opportunity for better negotiation with the industries, they violate the principle of transparency that regulates the world market. However, the existence of confidentiality agreements has meant that the declared prices are not the actual prices, unfairly harming the countries that use these price lists as beacons in their systems. METHODS: Representatives of health, judicial, legislative, patient organizations and academics from eight countries in Latin America and South Korea participated in a meeting in September 2017 in Chile to discuss price confidentiality agreements and the impact on public health policies. During the meeting, participants were presented with a hypothetical case to subsidize the discussion on the topic. Divided into groups, participants should propose recommendations for the problem by pointing out the pros and cons if each proposed recommendation was adopted. The groups were then confronted by a simulated jury and finally issued a single and final recommendation for the problem. RESULTS: The topic was widely discussed and recommendations were raised by the participants. Among them, it is worth noting the elaboration of norms that regulate the negotiations of prices between the countries bringing transparency and harmony in the adopted conducts. In addition, the possible consequences and potential impacts of confidentiality on drug prices and inputs, such as information asymmetry and inequity of access between countries, were pointed out. CONCLUSION: Despite there are efforts to make price negotiations more transparent, there is still no well-established standardization that promotes a well-functioning market. Confidentiality agreements hamper the fairness of access to essential health products.

Who should pay for the continuity of post-trial health care treatments?

BACKGROUND: The bioethical debate in the world on who should pay for the continuity of post-trials treatment of patients that have medical indication remains obscure and introduces uncertainties to the patients involved in the trials. The continuity of post-trial treatment was only incorporated in the 2000s by the Helsinki Declaration. The Universal Declaration on Bioethics and Human Rights, published in 2006, points out that post-trial continuity may present a broader scope than just the availability of the investigated medicine. In the latest version of this Declaration, in 2013, it was stated that "prior to the start of the clinical trial, funders, researchers and governments of the countries participating in the research should provide post-trial access for all participants who still require an intervention that was identified as beneficial. This information should also be disclosed to participants during the informed consent process". However, a systematic review on the registration of phase III and IV clinical trials, from the clinical trials website, demonstrated that the understanding of the various guidelines and resolutions is conflicting, generating edges in the post-trial setting. For the health authorities of countries where clinical trials take place, the uncertainties about the continuity of the treatments generate gaps in care and legal proceedings against health systems, which are forced to pay for the treatments, even if they are not included in the list of medicines available to the population. METHODS: Fifty-one representatives from the health, judicial, legislative, patient and academic organizations of eight countries of Latin American and South Korea took part in a meeting in Chile, in 2017, to discuss the responsibility of the treatment continuation after clinical trials. From a hypothetical case of development of a new drug and its studies of efficacy and safety, the participants, divided in groups, proposed recommendations for the problem and pointed out the pros and cons of adopting each recommendation. The groups were, afterwards, confronted by a simulated jury and, finally, issued a final recommendation for the problem. Then, an analysis was made on the content of the recommendations and the pros and cons in adopting conservative or liberal positions, besides the possible impacts of a restrictive regulation regarding the conduction of clinical trials, pointed out by the groups, before and after the simulated jury. RESULTS: The theme was widely discussed and about 12 recommendations were proposed by the participants. The main ones took into account aspects related to patients' rights, economic factors and the development of new technologies, above the position of industry and research institutes, as well as the legislation in force in each country. CONCLUSION: The countries of Latin America and South Korea, currently, do not have laws that address patients' rights, moreover, there is no definition on who should be responsible for post-trial treatments. It is suggested that the World Health Organization issue a resolution recommending that all associated countries determine that the pharmaceutical and medical device industries, or those that sponsored it, should continue to provide treatment to all patients who participated in clinical trials and have medical indication to the continuity.

Pharmaceutical innovativeness index: methodological approach for assessing the value of medicines - a case study of oncology drugs.

BACKGROUND: We propose a framework to assess the value of pharmaceutical innovations, with explicit clinical and methodological parameters, based on the therapeutic value and health needs. RESEARCH DESIGN AND METHODS: The study was based on the adaptation of health technology assessment methods documented in the literature, which was applied to a sample of oncological drugs. Difficulties and issues during the application of those tools were identified and addressed to develop a new framework with new and revised domains and clear classification criterion for each domain. Scores were assigned to each level and domain according to their relevance to generate the final score of innovativeness. RESULTS: The Pharmaceutical Innovation Index (PII) includes four domains, two related to clinical and social dimensions - Therapeutic Need and Added Therapeutic Value - and other two about methodological features - Study Design and Quality (risk of bias). The scores combined after assigned to each domain results Index of the Innovativeness of the medicines represents the degree of pharmaceutical innovation. CONCLUSION: This work proposes a transparent methodology with well-defined criteria and script; the algorithm developed with authors' weightings and criteria may be switched to best adjust to other applications, perspective or clinical indications, while keeping the transparency and objectiveness.

Effectiveness of nirmatrelvir-ritonavir for the treatment of patients with mild to moderate COVID-19 and at high risk of hospitalization: Systematic review and meta-analyses of observational studies.

OBJECTIVE: To assess the effectiveness of nirmatrelvir-ritonavir in the treatment of outpatients with mild to moderate COVID-19 who are at higher risk of developing severe illness, through a systematic review with meta-analyses of observational studies. METHODS: A systematic search was performed, in accordance with the Cochrane search methods, to identify observational studies that met the inclusion criteria. The outcomes of mortality and hospitalization were analyzed. Search was conducted on PubMed, EMBASE, and The Cochrane Library. Two reviewers independently screened references, selected the studies, extracted the data, assessed the risk of bias using ROBINS-I tool and evaluated the quality of evidence using the GRADE tool. This study followed the PRISMA reporting guideline. RESULTS: A total of 16 observational studies were finally included. The results of the meta-analysis showed that in comparison to standard treatment without antivirals, nirmatrelvir-ritonavir reduced the risk of death by 59% (OR = 0.41; 95% CI: 0.35-0.52; moderate certainty of evidence). In addition, a 53% reduction in the risk of hospital admission was observed (OR = 0.47; 95% CI: 0.36-0.60, with very low certainty of evidence). For the composite outcome of hospitalization and/or mortality, there was a 56% risk reduction (OR = 0.44; 95% CI: 0.31-0.64, moderate certainty of evidence). CONCLUSION: The results suggest that nirmatrelvir-ritonavir could be effective in reducing mortality and hospitalization. The results were valid in vaccinated or unvaccinated high-risk individuals with COVID-19. Data from ongoing and future trials may further advance our understanding of the effectiveness and safety of nirmatrelvir-ritonavir and help improve treatment guidelines for COVID-19.

Efficacy/effectiveness and safety of emicizumab prophylaxis of people with hemophilia A: a systematic review and meta-analysis.

BACKGROUND: Emicizumab is a monoclonal antibody approved for prophylaxis against bleeds for people with hemophilia A (PwHA). A systematic review was conducted evaluating the efficacy/effectiveness and the safety of emicizumab as prophylaxis for PwHA compared to prophylaxis with factor VIII (FVIII) or bypassing agents (BPA), respectively in patients without and with inhibitors. RESEARCH DESIGN AND METHODS: Database-directed search strategies were performed in Aug/26/2022 and updated in Mar/16/2023. Studies evaluating the prophylaxis with emicizumab versus prophylaxis with FVIII or BPA in PwHA without or with inhibitors, respectively, were selected by two independent reviewers. Data were extracted by two independent reviewers. Annualized bleeding rates for total treated bleeding events (ABR-all) were evaluated by meta-analysis. The quality of studies and certainty of evidence were assessed. RESULTS: A total of 11 studies were included. The standard mean differences for ABR-all were -0.6 (95%CI -1.0 to -0.2, p-value = 0.0002), among PwHA without inhibitors, and -1.7 (95%CI -2.4 to -0.9, p-value <0.00001), among PwHA with inhibitors. However, there was moderate heterogeneity in both meta-analyses. The most frequent adverse event was injection site reaction. CONCLUSIONS: Emicizumab prophylaxis was superior in reducing the ABR-all when compared with prophylaxis with FVIII or BPA.

Economic Evaluation of Immune Tolerance Induction in Children With Severe Hemophilia A and High-Responding Inhibitors: A Cost-Effectiveness Analysis of Prophylaxis With Emicizumab.

OBJECTIVE: This study aimed to measure the cost-effectiveness of prophylaxis with emicizumab in PsHAhri on ITI in Brazil. METHODS: A cost-effectiveness modeling analysis was used to estimate the costs per PsHAhri on ITI and the number of prevented bleedings from undertaking one intervention (prophylaxis with BpA) over another (prophylaxis with emicizumab), based on the Brazilian Ministry of Health perspective. Costs of ITI with recombinant FVIII, prophylaxis with BpA or emicizumab, and treated bleeding episodes with BpA costs were evaluated for PsHAhri who had ITI success or failure. This study was conducted with the perspective of the Brazilian Ministry of Health (payer). RESULTS: During ITI, prophylaxis with BpA cost US $924 666/PsHAhri/ITI, whereas prophylaxis with emicizumab cost US $488 785/PsHAhri/ITI. During ITI, there was an average of 9.32 bleeding episodes/PsHAhri/ITI when BpA were used as prophylaxis and 0.67 bleeding/PsHAhri/ITI when emicizumab was used. By univariate deterministic sensitivity analysis, emicizumab remained dominant whichever variable was modified. CONCLUSION: In this study, prophylaxis with emicizumab during ITI is a dominant option compared with prophylaxis with BpA during ITI.

Economic evaluation of adalimumab versus etanercept for psoriatic arthritis in a Brazilian real-world model.

BACKGROUND: TNF inhibitors are costly drugs supplied generally on health systems or private insurances. Performance analysis is essential to verify the results achieved by health technologies in these systems. The objective of the study was to compare the two most used biological drugs for the treatment of psoriatic arthritis (PsA) in Brazil. METHODS: A cost-utility analysis was built using a Markov model, with a five-year time horizon, a discount rate of 5%, and from the perspective of the Unified Health System. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: Etanercept was the most cost-effective drug. Adalimumab became the most cost-effective drug in one of the four analysis scenarios with a willingness to pay from one gross domestic product per capita. The deterministic sensitivity analysis identified that the cost parameters had the greatest impact on the most effective drug. The probabilistic sensitivity analysis indicated that etanercept is the drug most likely to be cost-effective. CONCLUSION: The difference between the drugs in terms of utility was minimal and the costs were the main factor that impacted the cost-utility ratio, which points to the benefits of price renegotiation for the efficient allocation of resources in the health system.

Conventional Synthetic Disease-Modifying Anti-rheumatic Drugs for Psoriatic Arthritis: Findings and Implications From a Patient Centered Longitudinal Study in Brazil.

Background: Conventional synthetic disease-modifying antirheumatic drugs are the first-line treatment to inhibit the progression of psoriatic arthritis. Despite their widespread clinical use, few studies have been conducted to compare these drugs for psoriatic arthritis. Methods: a longitudinal study was carried out based on a centered patient national database in Brazil. Market share of drugs, medication persistence, drug costs, and cost per response were evaluated. Results: a total of 1,999 individuals with psoriatic arthritis were included. Methotrexate was the most used drug (44.4%), followed by leflunomide (40.6%), ciclosporin (8.2%), and sulfasalazine (6.8%). Methotrexate and leflunomide had a greater market share than ciclosporin and sulfasalazine over years. Medication persistence was higher for leflunomide (58.9 and 28.2%), followed by methotrexate (51.6 and 25.4%) at six and 12 months, respectively. Leflunomide was deemed the most expensive drug, with an average annual cost of $317.25, followed by sulfasalazine ($106.47), ciclosporin ($97.64), and methotrexate ($40.23). Methotrexate was the drug being the lowest cost per response. Conclusion: Methotrexate had the best cost per response ratio, owing to its lower cost and a slightly lower proportion of persistent patients when compared to leflunomide. Leflunomide had a slightly higher medication persistence than methotrexate, but it was the most expensive drug.

Functionality assessment in patients with rheumatic diseases undergoing treatment in the Public Health System.

OBJECTIVE: To evaluate the therapeutic response (functionality) and its associated factors in patients on biological drugs on the Public Health System for treatment of rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis. METHODS: An open prospective cohort was carried out from 2011 to 2019, in Belo Horizonte (MG). Functionality was assessed using the Health Assessment Questionnaire Disability-Index at baseline, and after 6 and 12 months of follow-up. Factors associated with poor functionality were identified through logistic regression. RESULTS: The median Health Assessment Questionnaire Disability-Index at baseline was 1.5 (interquartile range of 0.8-1.9), with poor functionality observed in patients with rheumatoid arthritis. Improved functionality was seen at 6 months of treatment for the three diseases. The predictors of poor functionality at 6 months for psoriatic arthritis and ankylosing spondylitis were female sex, low education levels, and high disease activity; and for rheumatoid arthritis and psoriatic arthritis were female sex, advanced age, and high disease activity. In 12 months, the three diseases had predictors of worse functionality: female sex, low education, and high disease activity. CONCLUSION: There was a significant improvement in functionality during the follow-up, with better response at 6 months of treatment. Poor functionality was observed in older, female patients, with low education and high disease activity.

First-line biologic therapy with tumor necrosis factor inhibitors for psoriatic arthritis: a prospective observational study.

BACKGROUND: Psoriatic arthritis (PsA) is a chronic inflammatory disease that affects multiple joints. It is associated with psoriasis and treated with synthetic and biologic drugs. OBJECTIVE: The objective of this study was to assess the outcomes of patients who received biologic therapy with tumor necrosis factor (TNF) inhibitors in terms of effectiveness, safety, functionality, and quality of life. DESIGN AND SETTING: A prospective observational study was performed at a single center in Belo Horizonte, Brazil. METHODS: Patients with PsA who received their first TNF inhibitor treatment were followed up for 12 months. Disease activity was measured using the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) and Clinical Disease Activity Index (CDAI). Functionality was measured using the Health Questionnaire Assessment (HAQ), and quality of life was evaluated using the European Quality of Life Five Dimensions (EQ-5D). Multiple linear regression was used to identify predictors of the clinical response at 12 months. RESULTS: A total of 143 patients treated with adalimumab or etanercept were evaluated. Most of the clinical measures were significantly improved at 12 months. However, 31%-51% of the patients did not achieve good clinical control. No differences were observed between adalimumab and etanercept, except for poor functionality at 12 months among patients treated with etanercept. The main predictors of a worse clinical response were female sex, etanercept use, poor functionality, or lower quality of life at baseline. The main adverse reactions were alopecia, headache, injection site reaction, sinusitis, flu, dyslipidemia, and infections. CONCLUSION: TNF inhibitor therapy was effective and safe. However, despite improvements in clinical measures, most patients did not achieve satisfactory control of the disease.

Acceptability and willingness to pay for a hypothetical vaccine against SARS CoV-2 by the Brazilian consumer: a cross-sectional study and the implications.

INTRODUCTION: The new coronavirus pandemic has appreciably impacted morbidity and mortality, as well as having an economic impact worldwide. New vaccines are a potential way forward to reduce transmission rates and subsequent infection. In Brazil, vaccines are being distributed via the public sector; however, in the future, they will be available in the private market. Information about consumers' willingness to pay (WTP) for a hypothetical vaccine against SARS CoV-2 can help future price setting discussions. METHODS: A cross-sectional study was conducted with consumers in the five regions of Brazil regarding the WTP for a hypothetical vaccine against SARS CoV-2 with a 50% efficacy. RESULTS: A total of 1402 individuals over 18 years of age who declared not having COVID-19 at the time of the survey were interviewed. The acceptability for this hypothetical vaccine was 80.7%. In addition, the amount of WTP by Brazilian consumers for a hypothetical SARS CoV-2 vaccine was estimated at US$ 22.18(120.00 BRL). CONCLUSION: This study can contribute to decision-making to inform potential pricing for a hypothetical SARS CoV-2 vaccine.

Budget impact analysis of medicines: estimated values versus real-world evidence and the implications.

OBJECTIVES: Budget Impact Analyses (BIA) of medicines helps managers in promoting health systems' sustainability when assessing the role and value of new medicines. However, it is not clear whether BIAs typically underestimate or overestimate the impact on real-world budgets. This retroactive analysis seeks to compare estimated values obtained by a BIA and Real-World Evidence (RWE) to guide discussions. METHODS: The estimated values were obtained through a BIA concerning the incorporation of adalimumab for the treatment of Rheumatoid Arthritis into the Brazilian Unified Health System (SUS) carried out retroactively and per international guidelines. RWE data was extracted from SUS computerized systems. We subsequently compared the number of treatments, costs, and Incremental Budget Impact (IBI). RESULTS: - The total number of treatments was underestimated by 10% (6,243) and the total expenditure was overestimated by 463% (US$ 4.7 billion). In five years, the total difference between the estimated values and real IBI reached US$ 1.1 billion. A current expenditure of US$ 1.0 was observed for every US$ 5.60 of estimated expenditure. CONCLUSION: - The higher estimates from the BIA might cause decision makers to be more cautious with the introduction of a new medicine to reduce the opportunity costs for other interventions.

A Strategy to Improve Knowledge about Health Policies and Evidence Based Medicine for Federal Magistrates in Health Litigation.

Several countries maintain universal health coverage, which implies responsibility to organize delivery formats of healthcare services and products for citizens. In Brazil, the health system has a principle of universal access for more than 30 years, but many deficiencies remain and the country observes a day practice for those seeking judicial decisions to determine provision of healthcare.

Cost-utility analysis of the anti-TNF therapy for rheumatoid arthritis in a real-world based model.

BACKGROUND: Spending on drugs provided by the Brazilian Public Health System (BPHS) for the treatment of rheumatoid arthritis (RA) increased substantially with the beginning of the supply of biological disease-modifying anti-rheumatic drugs (bDMARD). This study aims to perform a cost-utility analysis of the most used biological drugs for the treatment of RA in Brazil. METHODS: a Markov model was used to carry out the cost-utility analysis. The data were obtained from a prospective cohort of RA patients using adalimumab, etanercept, and golimumab in Brazil. The BPHS perspective was adopted and the time horizon was five years. Deterministic and probabilistic sensitivity analyses were performed to evaluate the uncertainty. RESULTS: golimumab was the most cost-effective drug. Etanercept was dominated by golimumab. Adalimumab presented an incremental cost-utility ratio (ICUR) of $95,095.37 compared to golimumab in five years of follow-up. These results were confirmed by sensitivity analyses. CONCLUSION: the utility among adalimumab, etanercept, and golimumab was similar and the cost was the component that most impacted the economic model. Therefore, depending on the agreed price with the drug manufacturers, the incremental cost-utility ratio may vary among them.

Medication adherence and persistence of psoriatic arthritis patients treated with biological therapy in a specialty pharmacy in Brazil: a prospective observational study.

BACKGROUND: Pharmaceutical services in Brazil provide access, supply, and rational use of drugs for all population and an effort has been made to improve the quality of these services. Biological drugs are high-cost drugs supplied in Brazil that can inhibit disease progression and improve the quality of life of psoriatic arthritis (PsA) patients. However, some patients did not achieve therapeutic goals. OBJECTIVE: To evaluate the medication adherence and persistence of PsA patients treated with tumor necrosis factor inhibitors (anti-TNF) drugs and their associated factors. METHODS: A prospective observational study was performed at a single-specialty pharmacy in Belo Horizonte, Brazil. Medication adherence, persistence, and clinical outcomes were evaluated at 12 months of follow-up. Medication persistence was historically compared to overall PsA patients treated in Brazil. Associated factors were identified through log-binomial regression. RESULTS: One hundred ninety-seven PsA patients were included in the study, of whom 147 (74.6%) and 142 (72.1%) had medication adherence and persistence, respectively. Patients treated with infliximab presented the highest adherence (90.5%) and persistence rate (95.2%) in comparison to patients treated with other drugs, except for adalimumab versus infliximab for adherence outcome. All clinical measures significantly improved in patients with medication adherence and persistence. Medication persistence was higher for patients attended by specialty pharmacy than other PsA patients in Brazil. The associated factors to higher medication adherence were lower disease activity by BASDAI, being non-white race, and intravenous drug use. The associated factors to higher medication persistence were lower disease activity by Bath Ankylosing Spondylitis Activity Index (BASDAI), intravenous drug use, non-use of corticoids and non-steroidal anti-inflammatory drugs, and comorbidity. CONCLUSIONS: Patients with medication adherence and persistence had significant improvements in clinical measures, functionality, and quality of life. High medication adherence and persistence to biological therapy were observed and associated with lesser disease activity at baseline. Also, medication persistence to PsA patients attended in specialty pharmacy was higher than the overall PsA population in Brazil, which indicates the importance of pharmaceutical services to provide health care and promote the effectiveness and safety of biological therapies.