Demonstrating proof of concept for value-based agreements in Europe: two real-world cases.

OBJECTIVES: Value-based agreements (VBAs) link access, reimbursement, or price to the real-world usage and impact of a medicine, thereby enabling patient access while reducing clinical or financial uncertainty for the payer. VBAs have the potential to support improved patient outcomes, given the value-oriented approach to care, and lead to overall savings, while enabling payers to share risk and reduce uncertainty. METHODS: This commentary outlines the key challenges, enablers, and a framework for successful implementation by comparing the experience of two VBAs for AstraZeneca medicines, aiming to increase confidence in their future use. RESULTS: Engagement by payers, manufacturers, physicians, and provider institutions, and robust data collection systems that are accessible, simple to use, and add little burden to physicians were key to successfully negotiating a VBA that worked for all stakeholders. In both country systems, a legal/policy framework enabled innovative contracting. CONCLUSIONS: These examples demonstrate proof of concept for VBA implementation in different settings, and may inform future VBAs.

Acordo de Compartilhamento de Risco: projeto-piloto no Sistema Único de Saúde / Risk Sharing Agreement: a pilot project in the Brazilian Unified Health System

O Acordo de Compartilhamento de Risco é definido como um acordo no qual o Estado concorda em oferecer acesso temporário a um novo medicamento, enquanto a indústria farmacêutica aceita receber pelo produto conforme o desempenho do medicamento em reais condições de uso. A partilha de risco depende, necessariamente, da coleta de evidências adicionais, que podem se referir aos benefícios terapêuticos ou ao volume de pacientes, conforme avaliação de seu uso na prática. Os autores descreveram a experiência do projeto-piloto de Acordo de Compartilhamento de Risco no Sistema Único de Saúde.

Risk Sharing Agreement is defined as an agreement in which the State agrees to offer temporary access to a new drug, while the pharmaceutical industry accepts to receive the product according to the performance of the drug in real conditions of use. Risk sharing necessarily depends on the collection of additional evidence that may refer to the therapeutic benefits or the volume of patients, according to the assessment of its use in practice. The authors described the experience of the pilot project of a Risk Sharing Agreement in the Unified Health System.

Risk Sharing Agreement: a pilot project in the Brazilian Unified Health System

Risk Sharing Agreement is defined as an agreement in which the State agrees to offer temporary access to a new drug, while the pharmaceutical industry accepts to receive the product according to the performance of the drug in real conditions of use. Risk sharing necessarily depends on the collection of additional evidence that may refer to the therapeutic benefits or the volume of patients, according to the assessment of its use in practice. The authors described the experience of the pilot project of a Risk Sharing Agreement in the Unified Health System.

Measuring Out-of-pocket Payment, Catastrophic Health Expenditure and the Related Socioeconomic Inequality in Peru: A Comparison Between 2008 and 2017.

OBJECTIVES: Describe out-of-pocket payment (OOP) and the proportion of Peruvian households with catastrophic health expenditure (CHE) and evaluate changes in socioeconomic inequalities in CHE between 2008 and 2017. METHODS: We used data from the 2008 and 2017 National Household Surveys on Living and Poverty Conditions (ENAHO in Spanish), which are based on probabilistic stratified, multistage and independent sampling of areas. OOP was converted into constant dollars of 2017. A household with CHE was assumed when the proportion between OOP and payment capacity was ≥0.40. OOP was described by median and interquartile range while CHE was described by weighted proportions and 95% confidence intervals (CIs). To estimate the socioeconomic inequality in CHE we computed the Erreygers concentration index. RESULTS: The median OOP reduced from 205.8 US dollars to 158.7 US dollars between 2008 and 2017. The proportion of CHE decreased from 4.9% (95% CI, 4.5 to 5.2) in 2008 to 3.7% (95% CI, 3.4 to 4.0) in 2017. Comparison of socioeconomic inequality of CHE showed no differences between 2008 and 2017, except for rural households in which CHE was less concentrated in richer households (p<0.05) and in households located on the rest of the coast, showing an increase in the concentration of CHE in richer households (p<0.05). CONCLUSIONS: Although OOP and CHE reduced between 2008 and 2017, there is still socioeconomic inequality in the burden of CHE across different subpopulations. To reverse this situation, access to health resources and health services should be promoted and guaranteed to all populations.

The effect of the health poverty alleviation project on financial risk protection for rural residents: evidence from Chishui City, China.

BACKGROUND: Illness is the leading cause (44%) of poverty in China. Since 2016, The health poverty alleviation project, an integral component of the Targeted Poverty Alleviation in China, was implemented in 2016 to strengthen financial risk protection against illness for financially backward segments of the population. However, the effects of the health poverty alleviation project on financial risk protection have not been explored in the literature, this paper aims to bridge the gap. METHODS: Using panel data on 63,426 rural households in Chishui City, China, from 2014 to 2017, the difference-in-differences with propensity score matching method was employed. RESULTS: The health poverty alleviation project reduces out-of-pocket payments by 15% on average and decreases the probability of catastrophic health expenditure (annual out-of-pocket payments exceeding 10% of annual household income) and impoverishing health spending occurrence (out-of-pocket payments are forcing a household into poverty or into deeper poverty) by 7.7 and 11.7%, respectively. Additionally, the project increases the number of annual hospitalizations per household by 0.035. CONCLUSION: Our study demonstrates that the health poverty alleviation project significantly improves financial risk protection by reducing out-of-pocket payments and decreasing the probability of incurring catastrophic or impoverishing levels of health expenditure. Our study has implications for the poverty reduction policies and reform of the Chinese health financing system.

A incorporação do nusinersena no Sistema Único de Saúde: uma reflexão crítica sobre a institucionalização da avaliação de tecnologias em saúde no Brasil / The incorporation of nusinersen by the Brazilian Unified National Health System: critical thoughts on the institutionalization of health technology assessment in Brazil / La incorporación del nusinersén al Sistema Único de Salud: una reflexión crítica sobre la institucionalización de la evaluación de tecnologías en salud en Brasil

Resumo: Em abril de 2019, foi assinada a portaria de incorporação do medicamento nusinersena no Sistema Único de Saúde (SUS). É o medicamento mais caro já incorporado ao SUS, para uso no tratamento de atrofia muscular espinhal 5q tipo I. A incorporação é referida como um marco na tomada de decisão sobre novas tecnologias no SUS, a ser viabilizada por meio de acordo de partilha de risco. O trabalho discute o processo de incorporação do nusinersena, destacando aspectos contextuais, temporais e técnicos, além de possíveis consequências para a institucionalização da avaliação de tecnologias em saúde (ATS) no SUS. Seguiu método exploratório, com revisão de informações públicas produzidas pela Comissão de Incorporação de Tecnologias no SUS (CONITEC) e busca em bancos de dados governamentais de preços e compras. Foi produzida linha temporal descrevendo os pontos-chave do processo de incorporação. Houve dois pedidos de incorporação do medicamento. O primeiro, submetido pela Secretaria de Ciência, Tecnologia e Insumos Estratégicos (SCTIE) do Ministério da Saúde, negado por unanimidade, em novembro de 2018. Seguiu-se o pedido do Secretário da SCTIE à Advocacia-Geral da União (AGU), para que pudesse decidir de forma contrária à recomendação do plenário da CONITEC. A AGU recomendou uma nova submissão, feita pela empresa produtora e aprovada por unanimidade, em março de 2019. Não houve acréscimo de novas evidências ou redução de preço que justificassem a mudança de decisão. Não foram identificados os elementos constituintes do acordo de partilha de risco. São sinalizados problemas de transparência e accountability, bem como riscos ao processo de institucionalização da ATS que vinha em curso no SUS.

Abstract: In April 2019, a ruling was signed for the incorporation of the drug nusinersen by the Brazilian Unified National Health System (SUS). Nusinersen is the most expensive drug ever incorporated by the SUS and is used to treat type I 5q spinal muscular atrophy. The incorporation has been described as a milestone in decision-making on new technologies in the SUS, enabled through a risk-sharing agreement. The article discusses the process involved in the incorporation of nusinersen, highlighting the context, timing, and technical issues, in addition to possible consequences for the institutionalization of health technology assessment (HTA) in the SUS. The study used an exploratory method, reviewing public information produced by the Commission for Incorporation of Technologies in the SUS (CONITEC) and searches in government databanks on prices and purchases. A timeline was produced, describing the key points in the process of incorporation. There were two formal requests for the drug's incorporation. The first was submitted by the Division of Science, Technology, and Strategic Inputs (SCTIE) of the Brazilian Ministry of Health and was turned down unanimously in November 2018. This was followed by a petition by the head of the SCTIE to the Attorney General's Office (AGU) to overrule the recommendation by the CONITEC plenary. The AGU recommended a new submission, made by the drug's manufacturing company, which was approved unanimously in March 2019. The was no addition of new evidence or a price reduction to justify the change of decision. No elements were identified in the risk-sharing agreement. This suggests problems of transparency and accountability, as well as risks in the process of institutionalization of HTA that had been underway in the SUS.

Resumen: En abril de 2019, se firmó el decreto de incorporación del medicamento nusinersén en el Sistema Único de Salud brasileño (SUS). Es el medicamento más caro que se ha incorporado al SUS para su uso en el tratamiento de la atrofia muscular espinal 5q tipo I. La incorporación del mismo está considerada como un marco de referencia en la toma de decisiones sobre nuevas tecnologías en el SUS, que puede ser viable mediante el acuerdo de distribución de riesgo. El trabajo discute el proceso de incorporación del nusinersén, destacando aspectos contextuales, temporales y técnicos, además de posibles consecuencias para la institucionalización de la evaluación de tecnologías en salud (ETS) en el SUS. El trabajo siguió el método exploratorio, con una revisión de la información pública, generada por la Comisión de Incorporación de Tecnologías en el SUS (CONITEC) y la búsqueda en bancos de datos gubernamentales de precios y compras. Se creó una línea temporal, describiendo los puntos-clave del proceso de incorporación. Hubo dos peticiones de incorporación del medicamento. La primera, sometida a la Secretaría de Ciencia, Tecnología e Insumos Estratégicos (SCTIE) del Ministerio de Salud, rechazada por unanimidad, en noviembre de 2018. A lo que le siguió la petición del Secretario de la SCTIE a la Abogacía-General de la Unión (AGU), para que pudiese decidir en otro sentido respecto a la recomendación del pleno de la CONITEC. La AGU recomendó una nueva remisión, realizada por la empresa productora y aprobada por unanimidad, en marzo de 2019. No se produjo un incremento de nuevas evidencias o una reducción del precio que justificasen el cambio de decisión. No se identificaron los elementos constituyentes del acuerdo de distribución de riesgo. Se señalaron los problemas de transparencia y rendición de cuentas, así como riesgos para el proceso de institucionalización de la ETS que estaba en curso en el SUS.

[Risk sharing contracts in the national health care system: Perceptions of health care professionals]. / Los contratos de riesgo compartido en el sistema nacional de salud: Percepciones de los profesionales sanitarios.

OBJECTIVE: Risk-sharing contracts (RSC) present a novel management tool, which link the payment to the pharmaceutical company to health outcomes. The objective of this work was to know the perception of health professionals about the utility of these agreements in the Spanish National Health System. METHODS: A questionnaire was designed to conduct a series of semi-structured interviews with hospital pharmacy, laboratory and oncology professionals from Spanish hospitals in Madrid, Aragón, Castilla-La Mancha, Castilla y León, Cataluña, La Rioja, País Vasco and Navarra. The selection criteria was for convenience. The interview period was from April to November 2017. A qualitative analysis was performed based on the responses from 14 interviews. RESULTS: All the surveyed affirmed that the CRCs allow to improve the economic and administrative management of the hospital, emphasizing as main advantages the budgetary control, the obtaining of funding and the savings possibilities. CRCs are perceived (13 of 14 respondents) as agreements with positive health implications because they increased the portfolio of treatments and had greater efficacy. The need for CRCs to register patients, involved monitoring and control, also contributed to the improvement of their health. In addition, CRCs were believed (8 out of 14 respondents) to facilitate the introduction of personalized medicine (MP) as both depend on diagnostic tests, one for screening reasons (MP) and the other for obtain clinical evidences that improve economic outcomes (CRC). However, it was considered that signing the CRCs entails the need to modify certain regulations (5 out of 14 respondents) as well as to increase the number of staff to handle bureaucratic tasks and to increase laboratory tests, which can complicate health management. CONCLUSIONS: Qualitative social research techniques have proven to be useful for gathering information on a new topic and understanding the perception of the advantages and disadvantages of CRCs, as well as their association with the MP. In addition, synergies were detected between the CRCs and the MP. Respondents had positive opinions on CRCs about its application, although work must be done in order to improve the normative and organizational context so that the additional complexity that they incorporate does not constitute an obstacle to extend its use.

OBJETIVO: Los contratos de riesgo compartido (CRC) presentan un novedoso instrumento de gestión sanitaria que condiciona el pago a la compañía farmacéutica dependiendi de si el paciente tratado con su fármaco es curado con éxito. El objetivo de este trabajo fue conocer la percepción de los profesionales sanitarios acerca de la utilidad de dichos acuerdos en el Sistema Nacional de Salud español. METODOS: Se diseñó un cuestionario para realizar una serie de entrevistas semiestructuradas con profesionales de farmacia hospitalaria, laboratorio y oncología de hospitales españoles de Madrid, Aragón, Castilla-La Mancha, Castilla y León, Cataluña, La Rioja, País Vasco y Navarra. El criterio de selección fue de conveniencia. El periodo de realización de las entrevistas fue de abril a noviembre de 2017. Se efectuó un análisis cualitativo a partir de las respuestas de 14 entrevistas. RESULTADOS: Todos los encuestados afirmaron que los CRC permiten mejorar la gestión económica y administrativa del hospital, destacando el control presupuestario, la obtención de financiación y la posibilidad de ahorro como ventajas principales. Los CRC son percibidos (13 de 14 entrevistados) como acuerdos con implicaciones positivas para la salud porque aumentaban el portfolio de tratamientos disponibles y porque los tratamientos introducidos contaban con una mayor eficacia. La necesidad de los CRC de registrar a los pacientes implicaba un seguimiento y control que también se entendió contribuía a la mejora de su salud. Además, los CRC se creyó (8 de 14 entrevistados) que favorecían la introducción de la medicina personalizada (MP) ya que tanto la MP como los CRC dependen e impulsan la elaboración de pruebas diagnósticas, ya sea por motivos de cribado (MP) o de aumentar las evidencias clínicas para mejorar los resultados económicos (CRC). No obstante, se consideró que la firma de los CRC conlleva la necesidad de modificar ciertas normativas (5 de 14 entrevistados), de aumentar las plantillas para encargarse de tareas burocráticas, como la elaboración de registros, y también de incrementar las pruebas de laboratorio, lo cual puede complicar la gestión sanitaria. CONCLUSIONES: En general, los CRC contaron con opiniones positivas acerca de su aplicación aunque deba trabajarse para mejorar el contexto normativo y organizativo de modo que la complejidad adicional que incorporan no constituya una traba para extender su uso. Además, se detectaron sinergias entre los CRC y la MP.

Applying Organizational Learning Research to Accountable Care Organizations.

To accomplish the goal of improving quality of care while simultaneously reducing cost, Accountable Care Organizations (ACOs) need to find new and better ways of providing health care to populations of patients. This requires implementing best practices and improving collaboration across the multiple entities involved in care delivery, including patients. In this article, we discuss seven lessons from the organizational learning literature that can help ACOs overcome the inherent challenges of learning how to work together in radically new ways. The lessons involve setting expectations, creating a supportive culture, and structuring the improvement efforts. For example, with regard to setting expectations, framing the changes as learning experiences rather than as implementation projects encourages the teams to utilize helpful activities, such as dry runs and pilot tests. It is also important to create an organizational culture where employees feel safe pointing out improvement opportunities and experimenting with new ways of working. With regard to structure, stable, cross-functional teams provide a powerful building block for effective improvement efforts. The article concludes by outlining opportunities for future research on organizational learning in ACOs.