Cost-Effectiveness Analysis of Vaccines for COVID-19 According to Sex, Comorbidity and Socioeconomic Status: A Population Study.

BACKGROUND AND OBJECTIVE: Coronavirus disease 2019 (COVID-19) vaccines are extremely effective in preventing severe disease, but their real-world cost effectiveness is still an open question. We present an analysis of the cost-effectiveness and economic impact of the initial phase of the COVID-19 vaccination rollout in the Basque Country, Spain. METHODS: To calculate costs and quality-adjusted life years for the entire population of the Basque Country, dynamic modelling and a real-world data analysis were combined. Data on COVID-19 infection outcomes (cases, hospitalisations, intensive care unit admissions and deaths) and population characteristics (age, sex, socioeconomic status and comorbidity) during the initial phase of the vaccination rollout, from January to June of 2021, were retrieved from the Basque Health Service database. The outcomes in the alternative scenario (without vaccination) were estimated with the dynamic model used to guide public health authority policies, from February to December 2020. Individual comorbidity-adjusted life expectancy and costs were estimated. RESULTS: By averting severe disease-related outcomes, COVID-19 vaccination resulted in monetary savings of €26.44 million for the first semester of 2021. The incremental cost-effectiveness ratio was €707/quality-adjusted life year considering official vaccine prices and dominant real prices. While the analysis by comorbidity showed that vaccines were considerably more cost effective in individuals with pre-existing health conditions, this benefit was lower in the low socioeconomic status group. CONCLUSIONS: The incremental cost-effectiveness ratio of the vaccination programme justified the policy of prioritising high-comorbidity patients. The initial phase of COVID-19 vaccination was dominant from the perspective of the healthcare payer.

Cost of lost productivity from acute respiratory infections in South America.

Objectives: To estimate the burden of permanent productivity losses caused by acute respiratory infections in South American countries in 2019. Methods: Mortality data from the Global Burden of Disease Study 2019 were analyzed to estimate the burden of disease attributable to acute respiratory infections. An approach based on the human capital method was used to estimate the cost of permanent productivity losses associated with respiratory diseases. To calculate this cost, the sum of the years of productive life lost for each death was multiplied by the proportion in the workforce and the employment rate, and then by the annual minimum wage or purchasing power parity in United States dollars (US$) for each country in the economically active age groups. Separate calculations were done for men and women. Results: The total number of deaths from acute respiratory infections in 2019 was 30 684 and the years of productive life lost were 465 211 years. The total cost of permanent productivity loss was about US$ 835 million based on annual minimum wage and US$ 2 billion in purchasing power parity, representing 0.024% of the region's gross domestic product. The cost per death was US$ 33 226. The cost of productivity losses differed substantially between countries and by sex. Conclusion: Acute respiratory infections impose a significant economic burden on South America in terms of health and productivity. Characterization of the economic costs of these infections can support governments in the allocation of resources to develop policies and interventions to reduce the burden of acute respiratory infections.

Cost of lost productivity from acute respiratory infections in South America

[ABSTRACT]. Objectives. To estimate the burden of permanent productivity losses caused by acute respiratory infections in South American countries in 2019. Methods. Mortality data from the Global Burden of Disease Study 2019 were analyzed to estimate the burden of disease attributable to acute respiratory infections. An approach based on the human capital method was used to estimate the cost of permanent productivity losses associated with respiratory diseases. To calculate this cost, the sum of the years of productive life lost for each death was multiplied by the proportion in the work- force and the employment rate, and then by the annual minimum wage or purchasing power parity in United States dollars (US$) for each country in the economically active age groups. Separate calculations were done for men and women. Results. The total number of deaths from acute respiratory infections in 2019 was 30 684 and the years of productive life lost were 465 211 years. The total cost of permanent productivity loss was about US$ 835 mil- lion based on annual minimum wage and US$ 2 billion in purchasing power parity, representing 0.024% of the region’s gross domestic product. The cost per death was US$ 33 226. The cost of productivity losses differed substantially between countries and by sex. Conclusion. Acute respiratory infections impose a significant economic burden on South America in terms of health and productivity. Characterization of the economic costs of these infections can support governments in the allocation of resources to develop policies and interventions to reduce the burden of acute respiratory infections.

[RESUMEN]. Objetivos. Estimar la carga de la pérdida permanente de productividad causada por infecciones respiratorias agudas en países sudamericanos en el 2019. Métodos. Se analizaron los datos de mortalidad del estudio sobre carga mundial de enfermedad del 2019 para estimar la carga de enfermedad atribuible a las infecciones respiratorias agudas. Se empleó un enfoque basado en el método del capital humano para estimar el costo de las pérdidas permanentes de productividad relacionadas con las enfermedades respiratorias. Para ello, la suma de los años perdidos de vida productiva por cada muerte se multiplicó por la proporción de la fuerza de trabajo y la tasa de empleo y, a continuación, por el salario mínimo anual o la paridad del poder adquisitivo en dólares estadounidenses en los grupos etarios económicamente activos de cada país. Se realizaron cálculos separados para hombres y mujeres. Resultados. El número total de muertes por infecciones respiratorias agudas en el 2019 fue de 30 684 y se perdieron 465 211 años de vida productiva. El costo total de la pérdida permanente de productividad fue de aproximadamente US$ 835 millones según el salario mínimo anual y de US$ 2000 millones en cuanto a la paridad de poder adquisitivo, lo que representa el 0,024% del producto interno bruto de la región. El costo por muerte fue de US$ 33 226. El costo de la pérdida de productividad difirió sustancialmente entre los países y según el sexo. Conclusión. Las infecciones respiratorias agudas suponen una carga económica significativa para América del Sur en términos de salud y productividad. La caracterización de los costos económicos de estas infecciones puede ayudar a los gobiernos en la asignación de recursos para elaborar políticas e intervenciones que permitan reducir la carga de las infecciones respiratorias agudas.

[RESUMO]. Objetivos. Estimar a carga de perdas permanentes de produtividade causadas por infecções respiratórias agudas em países da América do Sul em 2019. Métodos. Dados de mortalidade do estudo Carga Global de Doença 2019 foram analisados para estimar a carga de doença atribuível a infecções respiratórias agudas. Utilizou-se uma abordagem baseada no método do capital humano para estimar o custo das perdas permanentes de produtividade associadas às doenças respiratórias. Para calcular esse custo, a soma dos anos de vida produtiva perdidos devido a cada morte foi multiplicada pela proporção da força de trabalho e da taxa de emprego. Em seguida, esse valor foi multiplicado pelo salário mínimo anual ou pela paridade do poder de compra, em dólares dos Estados Unidos (US$), de cada país nas faixas etárias economicamente ativas. Foram feitos cálculos separados para homens e mulheres. Resultados. O número total de mortes por infecções respiratórias agudas em 2019 foi de 30 684, com 465 211 anos de vida produtiva perdidos. O custo total da perda permanente de produtividade foi de cerca de US$ 835 milhões com base no salário mínimo anual e US$ 2 bilhões em paridade de poder de compra, o que representa 0,024% do produto interno bruto da região. O custo por morte foi US$ 33 226. O custo da perda de produtividade diferiu substancialmente entre os países e por sexo. Conclusão. As infecções respiratórias agudas impõem uma carga econômica significativa à América do Sul em termos de saúde e produtividade. A caracterização dos custos econômicos dessas infecções pode fundamentar as decisões de alocação de recursos tomadas pelos governos para desenvolver políticas e intervenções com o intuito de reduzir a carga das infecções respiratórias agudas.

The impact of conventional cost-effectiveness analysis on pricing dynamics in the market of new medicines: a proposed countervailing approach.

BACKGROUND: Countries using cost effectiveness ratio as a decision tool for price and reimbursement decisions still witness accelerating price increases. The objective of this paper is to propose a change in the application of the incremental cost effectiveness ratio as a criterion for price policy. RESEARCH DESIGN: We develop a model that sets a price for marginal effectiveness equal to the marginal willingness to pay, but it reimburses average effectiveness according to the size of increased QALY gain. RESULTS: This new formula also allows to split the economic value of drug between patients and the industry and creates a reward to invest into QALY gains. We show some empirical data of the new prices derived from the application of the new formula, as well as the implications in terms of the consumer and manufacturer´s surplus based on two potential scenarios of the incentives generated by this new formulation. DISCUSSION: We propose that small increases in life expectancy be priced differently from substantial as a way of containing the price dynamics. CONCLUSIONS: A change in the application of the ICER threshold will help to reduce the price pressure on public budgets.

Cost of lost productivity from acute respiratory infections in South America / Costo de la pérdida de productividad por infecciones respiratorias agudas en América del Sur / Custo da perda de produtividade devido a infecções respiratórias agudas na América do Sul

ABSTRACT Objectives. To estimate the burden of permanent productivity losses caused by acute respiratory infections in South American countries in 2019. Methods. Mortality data from the Global Burden of Disease Study 2019 were analyzed to estimate the burden of disease attributable to acute respiratory infections. An approach based on the human capital method was used to estimate the cost of permanent productivity losses associated with respiratory diseases. To calculate this cost, the sum of the years of productive life lost for each death was multiplied by the proportion in the workforce and the employment rate, and then by the annual minimum wage or purchasing power parity in United States dollars (US$) for each country in the economically active age groups. Separate calculations were done for men and women. Results. The total number of deaths from acute respiratory infections in 2019 was 30 684 and the years of productive life lost were 465 211 years. The total cost of permanent productivity loss was about US$ 835 million based on annual minimum wage and US$ 2 billion in purchasing power parity, representing 0.024% of the region's gross domestic product. The cost per death was US$ 33 226. The cost of productivity losses differed substantially between countries and by sex. Conclusion. Acute respiratory infections impose a significant economic burden on South America in terms of health and productivity. Characterization of the economic costs of these infections can support governments in the allocation of resources to develop policies and interventions to reduce the burden of acute respiratory infections.

RESUMEN Objetivos. Estimar la carga de la pérdida permanente de productividad causada por infecciones respiratorias agudas en países sudamericanos en el 2019. Métodos. Se analizaron los datos de mortalidad del estudio sobre carga mundial de enfermedad del 2019 para estimar la carga de enfermedad atribuible a las infecciones respiratorias agudas. Se empleó un enfoque basado en el método del capital humano para estimar el costo de las pérdidas permanentes de productividad relacionadas con las enfermedades respiratorias. Para ello, la suma de los años perdidos de vida productiva por cada muerte se multiplicó por la proporción de la fuerza de trabajo y la tasa de empleo y, a continuación, por el salario mínimo anual o la paridad del poder adquisitivo en dólares estadounidenses en los grupos etarios económicamente activos de cada país. Se realizaron cálculos separados para hombres y mujeres. Resultados. El número total de muertes por infecciones respiratorias agudas en el 2019 fue de 30 684 y se perdieron 465 211 años de vida productiva. El costo total de la pérdida permanente de productividad fue de aproximadamente US$ 835 millones según el salario mínimo anual y de US$ 2000 millones en cuanto a la paridad de poder adquisitivo, lo que representa el 0,024% del producto interno bruto de la región. El costo por muerte fue de US$ 33 226. El costo de la pérdida de productividad difirió sustancialmente entre los países y según el sexo. Conclusión. Las infecciones respiratorias agudas suponen una carga económica significativa para América del Sur en términos de salud y productividad. La caracterización de los costos económicos de estas infecciones puede ayudar a los gobiernos en la asignación de recursos para elaborar políticas e intervenciones que permitan reducir la carga de las infecciones respiratorias agudas.

RESUMO Objetivos. Estimar a carga de perdas permanentes de produtividade causadas por infecções respiratórias agudas em países da América do Sul em 2019. Métodos. Dados de mortalidade do estudo Carga Global de Doença 2019 foram analisados para estimar a carga de doença atribuível a infecções respiratórias agudas. Utilizou-se uma abordagem baseada no método do capital humano para estimar o custo das perdas permanentes de produtividade associadas às doenças respiratórias. Para calcular esse custo, a soma dos anos de vida produtiva perdidos devido a cada morte foi multiplicada pela proporção da força de trabalho e da taxa de emprego. Em seguida, esse valor foi multiplicado pelo salário mínimo anual ou pela paridade do poder de compra, em dólares dos Estados Unidos (US$), de cada país nas faixas etárias economicamente ativas. Foram feitos cálculos separados para homens e mulheres. Resultados. O número total de mortes por infecções respiratórias agudas em 2019 foi de 30 684, com 465 211 anos de vida produtiva perdidos. O custo total da perda permanente de produtividade foi de cerca de US$ 835 milhões com base no salário mínimo anual e US$ 2 bilhões em paridade de poder de compra, o que representa 0,024% do produto interno bruto da região. O custo por morte foi US$ 33 226. O custo da perda de produtividade diferiu substancialmente entre os países e por sexo. Conclusão. As infecções respiratórias agudas impõem uma carga econômica significativa à América do Sul em termos de saúde e produtividade. A caracterização dos custos econômicos dessas infecções pode fundamentar as decisões de alocação de recursos tomadas pelos governos para desenvolver políticas e intervenções com o intuito de reduzir a carga das infecções respiratórias agudas.

Analysis of the Prescription of Antibiotics During the Implementation of COVID-19 Personal Protection Measures in a Regional Health System.

INTRODUCTION: The rational consumption of antibiotics is a valuable goal for developed countries. Antibiotic resistance (AMR) was the main health threat before the coronavirus-19 (COVID-19) pandemic. To restrict COVID-19 transmission, the Spanish government implemented personal protection measures, including the decree of a lockdown. The objective of this study is to analyze the prescription of antibiotics during the implementation of COVID-19 personal protection measures in primary care in a Spanish region. MATERIALS AND METHODS: Primary monthly official dispensations of antibacterials for systemic use (J01) were analyzed, disaggregating by clinical (active principle, age and sex of the patient) and socio-economic variables (basic health zone and level of income). Defined daily doses per thousand inhabitants (DID) prescribed in 2020 were compared with the prescription in 2019 using Chi-square test. RESULTS: In 2020, the prescription of antibiotics (11.37 DID) was significantly lower (-23.73%) than in 2019 (14.91 DID). The main reduction in the amount prescribed was found in May (-42.64%). However, the prescription was significantly higher (71.34%) in February 2020 than in the same month in 2019 in nursing homes. In March 2020, prescriptions were lower (-13.71%) than in the same month in 2019, except for the middle-income group, in which prescriptions were higher (9.67%), as well as azithromycin (10.11%). In April and May 2020, prescriptions were significantly lower than in 2019 in all age groups and both in women and men. CONCLUSION: Personal protection measures against COVID-19 transmission (lockdown, mask, social distance and increased hygiene) coincide in time with the greatest reduction in antibiotic prescription.

Drug prices in Latin American countries: the case of rheumatoid arthritis Biosimilars.

BACKGROUND: The objective of this paper is to analyze the prices of biological drugs in the treatment of Rheumatoid Arthritis (RA) in three Latin American countries (Brazil, Colombia and Mexico), as well as in Spain and the United States of America (US), from the point of market entry of biosimilars. METHODS: We analyzed products authorized for commercialization in the last 20 years, in Brazil, Colombia, and Mexico, comparing them to the United States of America (USA) and Spain. For this analysis, we sought the prices and registries of drugs marketed between 1999 and October 1, 2019, in the regulatory agencies' databases. The pricing between countries was based on purchasing power parity (PPP). RESULTS: The US authorized the commercialization of 13 distinct biologicals and four biosimilars in the period. Spain and Brazil marketed 14 biopharmaceuticals for RA, ten original, four biosimilars. Colombia and Mexico have authorized three biosimilars in addition to the ten biological ones. For biological drug prices, the US is the most expensive country. Spain's price behavior seems intermediate when compared to the three LA countries. Brazil has the highest LA prices, followed by Mexico and Colombia, which has the lowest prices. Spain has the lowest values in PPP, compared to LA countries, while the US has the highest prices. CONCLUSION: The economic effort that LA countries make to access these medicines is much higher than the US and Spain. The use of the PPP ensured a better understanding of the actual access to these inputs in the countries analyzed.

Efficiency of Diagnostic Testing for Helicobacter pylori Infections-A Systematic Review.

BACKGROUND: The most recommended treatment for a Helicobacter pylori infection is high doses of combined antibiotics. The objective of this article is to perform a systematic review of the economic evaluation studies applied to assess the efficiency of diagnostic testing for H. pylori infections, so that their main characteristics can be identified and to learn from the literature how the antimicrobial resistance (AMR) issue is incorporated into these economic evaluations. METHODS: We conducted a systematic review to compare the costs and clinical effectiveness of diagnostic strategies for H. pylori infections. We followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines and extracted the items from the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. RESULTS: We found thirteen articles that were of good quality according to CHEERS: six studies focused on diagnostics of Helicobacter pylori infections associated with dyspepsia and four on duodenal ulcers. Testing was found to be the most cost-effective strategy in eight articles. Four studies considered AMR. CONCLUSIONS: Testing was more cost-effective than empirical treatment, except in cases of high prevalence (as with developing countries) or when patients could be stratified according to their comorbidities. The introduction of AMR into the model may change the efficiency of the testing strategy.

Diagnostic Testing for Sepsis: A Systematic Review of Economic Evaluations.

INTRODUCTION: Sepsis is a serious and expensive healthcare problem, when caused by a multidrug-resistant (MDR) bacteria mortality and costs increase. A reduction in the time until the start of treatment improves clinical results. The objective is to perform a systematic review of economic evaluations to analyze the cost-effectiveness of diagnostic methods in sepsis and to draw lessons on the methods used to incorporate antimicrobial resistance (AMR) in these studies. MATERIAL AND METHODS: the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines were followed, and the Consolidated Health Economic Evaluation Reporting standards (CHEERS) checklist was used to extract the information from the texts. RESULTS: A total of 16 articles were found. A decision model was performed in 14. We found two ways to handle resistance while modelling: the test could identify infections caused by a resistant pathogen or resistance-related inputs, or outcomes were included (the incidence of AMR in sepsis patients, antibiotic use, and infection caused by resistant bacterial pathogens). CONCLUSION: Using a diagnostic technique to detect sepsis early on is more cost-effective than standard care. Setting a direct relationship between the implementation of a testing strategy and the reduction of AMR cases, we made several assumptions about the efficacy of antibiotics and the length-of-stay of patients.

Drug prices in Latin American countries: the case of rheumatoid arthritis Biosimilars

Abstract Background: The objective of this paper is to analyze the prices of biological drugs in the treatment of Rheumatoid Arthritis (RA) in three Latin American countries (Brazil, Colombia and Mexico), as well as in Spain and the United States of America (US), from the point of market entry of biosimilars. Methods: We analyzed products authorized for commercialization in the last 20 years, in Brazil, Colombia, and Mexico, comparing them to the United States of America (USA) and Spain. For this analysis, we sought the prices and registries of drugs marketed between 1999 and October 1, 2019, in the regulatory agencies' databases. The pricing between countries was based on purchasing power parity (PPP). Results: The US authorized the commercialization of 13 distinct biologicals and four biosimilars in the period. Spain and Brazil marketed 14 biopharmaceuticals for RA, ten original, four biosimilars. Colombia and Mexico have authorized three biosimilars in addition to the ten biological ones. For biological drug prices, the US is the most expensive country. Spain's price behavior seems intermediate when compared to the three LA countries. Brazil has the highest LA prices, followed by Mexico and Colombia, which has the lowest prices. Spain has the lowest values in PPP, compared to LA countries, while the US has the highest prices. Conclusions: The economic effort that LA countries make to access these medicines is much higher than the US and Spain. The use of the PPP ensured a better understanding of the actual access to these inputs in the countries analyzed.(AU)

Health economic evaluation of gene replacement therapies: methodological issues and recommendations.

Objective: To provide recommendations for addressing previously identified key challenges in health economic evaluations of Gene Replacement Therapies (GRTs), including: 1) the assessment of clinical effectiveness; 2) the valuation of health outcomes; 3) the time horizon and extrapolation of effects beyond trial duration; 4) the estimation of costs; 5) the selection of appropriate discount rates; 6) the incorporation of broader elements of value; and 7) affordability. Methods: A literature review on economic evaluations of GRT was performed. Interviews were conducted with 8 European and US health economic experts with experience in evaluations of GRT. Targeted literature reviews were conducted to investigate further potential solutions to specific challenges. Recommendations: Experts agreed on factors to be considered to ensure the acceptability of historical cohorts by HTA bodies. Existing prospective registries or, if not available, retrospective registries, may be used to analyse different disease trajectories and inform extrapolations. The importance of expert opinion due to limited data was acknowledged. Expert opinion should be obtained using structured elicitation techniques. Broader elements of value, beyond health gains directly related to treatment, can be considered through the application of a factor to inflate the quality-adjusted life years (QALYs) or a higher cost-effectiveness threshold. Additionally, the use of cost-benefit analysis and saved young life equivalents (SAVE) were proposed as alternatives to QALYs for the valuations of outcomes of GRT as they can incorporate broader elements of value and avoid problems of eliciting utilities for paediatric diseases. Conclusions: While some of the limitations of economic evaluations of GRT are inherent to limited clinical data and lack of experience with these treatments, others may be addressed by methodological research to be conducted by health economists.

Policies to Reduce Antibiotic Consumption: The Impact in the Basque Country.

In 2013, a change in copayment rate was introduced in the Basque Country (one year later than in the other regions in Spain), and improvements were made to drug packaging. In 2014, a National Program Against Bacterial Resistance (Spanish abbreviation: PRAN) was approved. The aim of this study is to analyze the impact of change to the copayment rate, the adjustment of drug packaging, and the approval of PRAN on the consumption of antibiotics. Raw monthly data on the consumption of antibiotics (costs, packages, and daily defined doses per thousand people (DID)) were collected from January 2009 to December 2018 in the Basque Country. Counterfactual and intervention analysis (Autoregressive integrated moving average (ARIMA) model) was performed for the total series, disaggregated by group of antibiotics (2019 WHO Access, Watch, and Reserve (AWaRe) Classification) and active substances with the highest cost per prescription (cefditoren and moxifloxacin), the lowest cost per prescription (doxycycline and cloxacillin), and the most prescribed active ingredients (amoxicillin, azithromycin, and levofloxacin). Introduction of copayment led to a 'stockpiling effect' one month before its implementation, equal to 8% in the three consumption series analyzed. Only the adjustment of drug packaging significantly reduced the number of packages dispensed (-12.19%). PRAN approval reduced consumption by 0.779 DID (-4.51%), representing a significant decrease for both 'access' and 'watch' group antibiotics. Despite the delay in implementing changes to copayment, there was a 'stockpiling effect'. With the adjustment of packaging, fewer packs were prescribed but with a higher drug load and price. PRAN approval reduced both the consumption of 'access group antibiotics' (first-line treatment) and 'watch group antibiotics' (second-line treatment).

The Use of Risk-Sharing Contracts in Healthcare: Theoretical and Empirical Assessments.

OBJECTIVE: The aim of this review is to provide a summary of the literature on risk-sharing agreements, including conceptual, theoretical and empirical (number of agreements and their achievements) perspectives, and stakeholders'  perceptions. METHODS: We conducted a systematic literature search in MEDLINE from 2000 to April 2019, following PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) methodology, and completed it with a manual search of other publications (mainly grey literature). The search was restricted to publications with English abstracts; the initial identification of articles was restricted to the title, abstract and key words fields. The geographical scope was not restricted. RESULTS: Over 20 studies proposed different taxonomies of risk-sharing contracts, which can be summarised as financial and paying-for-performance agreements. Theoretical studies modelling the incentives to implement risk-sharing agreements are scarce; they addressed different types of contracts and regulatory contexts, characterizing the drug prices and the optimal strategies of the involved agents. Empirical studies describing specific agreements are abundant and referred to different geographical contexts; however, few articles showed the economic results and assessed the value of such contracts. Stakeholders' perceptions of risk-sharing contracting were favourable, but little is known about the economic and clinical advantages of specific agreements. Whether risk-sharing contracts have yielded the desired results for healthcare systems remains uncertain. CONCLUSION: Risk-sharing contracts are increasingly used, although the lack of transparency and aggregated registries makes it difficult to learn from these experiences and assess their impact on healthcare systems.

[Regulations on expanded access and compassionate use of medicines in South AmericaRegulación de los programas de acceso ampliado y uso compasivo de medicamentos en América del Sur]. / Normatização de programas de acesso expandido e uso compassivo de medicamentos na América do Sul.

OBJECTIVE: To describe and compare the regulatory framework governing policies on rapid/alternative access to medicines (expanded access and compassionate use) in South American countries. METHOD: An exploratory descriptive study with analysis of documents was performed. In addition to a literature review, official rules issued by regulatory agencies were reviewed if available. Information was collected on how countries define the concepts of expanded access and compassionate use, clinical phase in which the medicine becomes available for these alternative uses, and role of physicians and sponsors. RESULTS: Argentina, Brazil, Chile, Peru, and Uruguay were included in the study. The information obtained revealed that the regulatory scenario is more structured in Argentina and Brazil than in the other countries. In Chile, rules on expanded access and compassionate use are available, however without an explicit definition of these concepts. In Peru and Uruguay, important definitions are missing regarding expanded access. The search did not reveal any databases with information on expanded access and compassionate use programs, supporting the notion that empirical data to evaluate the results of these policies are lacking. CONCLUSIONS: All the countries analyzed have a regulatory framework that contemplates rapid/alternative access to medicines by patients at risk. However, databases and transparent information are lacking, preventing a snapshot of the medicines covered and patients benefiting from alternative access programs and assessments of these policies in South America.

OBJETIVO: Describir y comparar los marcos regulatorios de las políticas de acceso rápido y alternativo a medicamentos (acceso ampliado y uso compasivo) en países de América del Sur. MÉTODOS: Se realizó un estudio exploratorio y descriptivo, con análisis documental. Además de revisión de la bibliografía científica sobre el tema, se obtuvo información sobre las normas oficiales, si estaba disponible en las páginas electrónicas de las autoridades regulatorias de medicamentos. Se recolectó información sobre la forma en que cada país define los conceptos de acceso ampliado y uso compasivo, la fase clínica en que se ofrece el medicamento para uso alternativo y las obligaciones de los médicos y patrocinadores. RESULTADOS: A partir de los criterios de inclusión, se seleccionaron para el estudio Argentina, Brasil, Chile, Perú y Uruguay. La información obtenida mostró que Argentina y Brasil presentan un escenario regulatorio más estructurado en comparación con los demás países del grupo citado. Chile presenta una norma sobre acceso ampliado y uso compasivo, pero sin definir explícitamente esos conceptos. En Perú y Uruguay, se constató la falta de definiciones importantes en cuanto al acceso ampliado. No se encontraron bases de datos con información sobre acceso ampliado y uso compasivo, lo cual corroboró la percepción sobre la escasez de datos empíricos para evaluar los resultados de esas políticas. CONCLUSIONES: Todos los países analizados cuentan con un marco regulatorio que permite el acceso rápido y alternativo a medicamentos de los pacientes en situación de riesgo. Sin embargo, no existen bancos de datos ni información transparente que permitan determinar qué medicamentos y pacientes se benefician de ese acceso alternativo y evaluar los resultados de esas políticas en América del Sur.

Implementation of risk-sharing contracts as perceived by Spanish hospital pharmacists.

BACKGROUND: Concerns about financial sustainability of health systems have promoted the adoption of risk-sharing agreements. Nevertheless, few insights have been derived, due to their confidentiality. The purpose of this study is to analyze to what extent these agreements have been implemented in Spain and the importance of several clinical and management variables concerning their use. We also explore whether risk-sharing agreements promote the adoption of personalized medicine. We give a descriptive analysis based on a questionnaire sent to members of the Spanish Society of Hospital Pharmacy, asking about the implementation of risk-sharing contracts in their hospitals. RESULTS: There were 80 replies. Implementation of risk-sharing agreements was high (90%), being oncology, neurology, dermatology and infectious diseases the main specialties. The most relevant variables were the number of units of medication per year (89%) in price-volume agreements, and the efficacy and uncertainty of treatments (over 75%) in pay-for-performance agreements. Price-volume agreements were suitable for both conventional and personalized medicine and pay-for-performance more specific for personalized medicine. Paying for performance promotes genetic testing (85%). CONCLUSIONS: The results suggest health authorities should encourage the assessment of financial and health outcomes of real-world contracts of conventional and personalized medicine to better know the variables influencing their use.