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.

Linha de cuidados no tratamento da asma: informações úteis para o gestor / Comprehensive care in asthma treatment: useful information for the health-care manager

Realizou-se, no dia 29 de agosto de 2020, um encontro virtual com gestores, representantes de associações de pacientes e médicos prescritores envolvidos no tratamento da asma, com o objetivo de discutir a necessidade e a viabilidade da incorporação de novas tecnologias para o tratamento dessa patologia. A asma é uma enfermidade caracterizada pela inflamação crônica das vias aéreas. É a principal causa de absenteísmo escolar e laboral e estima-se que seja responsável por até cinco mortes diárias em nosso país. Em pesquisa efetuada em operadora de autogestão, com vidas espalhadas por todo o país, observou-se que essa patologia (associada à doença pulmonar obstrutiva crônica ­ DPOC) acomete quase 7% dos seus segurados e que a utilização do plano foi, nesse grupo, 1,9x maior para consultas, 1,6x maior para exames, 2,5x maior para terapias e 2,9x maior para internações, resultando em um aumento de 25,5% nos gastos assistenciais. Observou-se que é de elevada importância que o gestor tenha um profundo conhecimento de sua carteira e que priorize toda a linha de cuidado do paciente. Dessa maneira, atuando diretamente no controle da severidade da patologia, terão os melhores resultados de qualidade de vida e restringirão os doentes que necessitarão de medicações mais modernas e, também, mais caras, com resultado óbvio no controle de custos. A asma ainda não tem, em geral, para os gestores de operadoras de saúde privadas, no Brasil, uma importância tão grande na sinistralidade das suas carteiras. Os novos imunobiológicos são úteis e efetivos e alguma contrapartida por parte do fabricante, como, por exemplo, o compartilhamento de riscos, pode ser necessária para uma incorporação desse arsenal no Rol de produtos que serão disponibilizados para os pacientes que deles necessitarem

On August 29 (2020), a virtual meeting was held with managers, representatives of patient associations and physicians involved in the treatment of asthma, in order to discuss the need and the feasibility of incorporating new technologies for the treatment of this disease. Asthma is a condition characterized by chronic inflammation of the airways. It is the main cause of school and work absenteeism and it is estimated that it is the cause of up to 5 daily deaths in our country, annually. In a survey carried out at a Health Care Plan, with patients spread across the country, it was observed that this pathology (associated with COPD) affects almost 7% of its insured persons and that the use of the plan was, in this group, 1.9x higher for consultations, 1.6x higher for exams, 2.5x higher for therapies and 2.9x higher for hospitalizations, resulting in a 25.5% increase in the global health care expenses. It was observed that it is highly important that managers have a deep knowledge of his portfolio and that they prioritize the entire process of patient care. In this way, acting directly in the control of the severity of the pathology, they will have the best quality of life results and will restrict the number of patients who will need more modern and also more expensive medications, with obvious impact on costs. Asthma still does not have, for the manager of a Private Health Operator, in Brazil, such importance in the expenses of his portfolio. The new immunobiologicals are useful, effective and some counterpart on the part of the manufacturer, such as, for example, the risk-share agreements, may be necessary for the incorporation of this into the arsenal of products that will be made available to patients that need them

Mechanism Study on Risk Sharing and Transfer of Drug Marketing Authorization Holders Based on Drug Defects / 中国药房

OBJECTIVE：To explore the mechanism of risk sharing and transfer of drug marketing authorization holders （MAH）based on drug defects ，and to promote the smooth implementation of MAH system in China. METHODS ：Firstly，the types of drug defects in the whole drug life cycle were analyzed ，and then the sources and “fault party ”of different types of drug defects in the open commissioning environment of the industry chain under the MAH system were also analyzed. Based on it ，the mechanism of risk sharing and transfer of MAH was established combined with foreign experience and China ’s actual situation. RESULTS & CONCLUSIONS ：Drug defects were divided into design defects ，manufacturing & operation & storage & transportation quality defects and use defects ，among which design defects and manufacturing & operation & storage & transportation quality defects were the two most important defects. Design defects mainly included those caused by human factors and those could not be found by current level of science and technology ，the“fault party ”according to different circumstances may be both the research and development institutions and may be the MAH. The “fault party ”of production & operation & storage & transportation quality defects was the drug production & operation & storage & transportation enterprises. For design defects ，it was recommended to establish China ’s adverse drug reaction compensation mechanism through a combination of insurance and relief. For production & operation & storage & transportation quality defects ，it was recommended that the drug quality liability insurance as the main ，contractual agreement as the supplement ，so that the “fault party ”should assume due responsibility.

Inspiration from International Experience on Risk Sharing Agreements of Medical Insurance Access for CAR-T Products / 中国药房

OBJECTIVE：To learn from the experience of foreign listed chimeric antigen receptor T lymphocyte （CAR-T） products in signing risk sharing agreements in medical insurance access ，so as to provide references for relevant decisions of medical insurance departments in China. METHODS ：Taking 9 risk sharing agreements of CAR-T products marketed in the United Kingdom，France，Italy and Germany as samples ，the international experience of medical insurance payment of CAR-T products were analyzed from six dimensions ，such as agreement types ，monitoring indicators ，data collection metho ds，agreement periods ， payment conditions and payment methods. Some suggestions were put forward for the medical insurance access of these products in China. RESULTS & CONCLUSIONS ：Four sample countries generally signed risk sharing agreements of medical insurance access （financial agreement and performance-based agreement ）with pharmaceutical enterprises ；the indicators such as progressive disease and progression-free survival were collected by using data collection system or clinical research data ，so as to monitor the efficacy and safety of CAR-T products. The agreement periods and payment conditions were determined according to different agreement types；“medical insurance advance payment ”or“pharmaceutical enterprise advance payment ”combined with “staged payments ” were adopted for risk control. Solving the risk of medical insurance funds caused by “efficacy uncertainty ”is the core issue of CAR-T product access. The induction of risk sharing agreements may be the way to solve this problem ，and the scientific design of the various elements of risk sharing agreements is a prerequisite to ensure that the agreement is operational.

Compartilhamento de risco na saúde suplementar brasileira / Risk sharing in Brazilian supplementary health

Objetivos: Este trabalho discute os recentes impactos do avanço tecnológico no mercado da saúde e a crescente importância do aumento de custos em toda a sua cadeia e investiga uma alternativa para aumentar o acesso a essas novas tecnologias por um maior número de pessoas na saúde suplementar. Nesse contexto, o objetivo geral proposto para este trabalho foi evidenciar as visões de gestores de saúde com papel de destaque no Brasil a respeito do uso de modelos de compartilhamento de risco (risk sharing) em diferentes geografias e em diferentes financiadores no sistema. Pretendeu-se aprofundar o tema com o cumprimento dos seguintes objetivos específicos, também sob a perspectiva dos participantes da pesquisa: verificar a utilização desses modelos de pagamento e, consequentemente, determinar as características e adaptações necessárias a eles; identificar os desafios para a consolidação do risk sharing na saúde suplementar; verificar o impacto do risk sharing nos custos com saúde das operadoras a partir da percepção dos entrevistados. Métodos: Trata-se de estudo qualitativo, com entrevistas em profundidade com os principais gestores e formadores de opinião em saúde de diferentes instituições no Brasil, totalizando 25 entrevistas. A análise de conteúdo foi escolhida como método de interpretação e construção das categorias para a análise. Resultados: Os resultados foram agrupados nas seguintes categorias: as sugestões e adaptações sugeridas para o melhor funcionamento na realidade brasileira; as críticas e desafios aos modelos propostos. Os resultados sugerem que o compartilhamento de risco pode ser uma alternativa capaz de promover uma nova forma de relacionamento entre os pagadores e a indústria fabricante, substituindo o tradicional modelo de remuneração, que limita a relação linear com todos os setores, buscando sempre o objetivo de atender o paciente com novas alternativas de acesso. Conclusões: Em meio à complexidade do sistema de saúde brasileiro, é importante que os players avaliem novas alternativas de remuneração e incorporação tecnológica. Os desafios são inúmeros, desde a efetivação do perfil dos pacientes elegíveis até a aplicabilidade do risk sharing que ocorre a partir do interesse de ambas as partes em trazer novas tecnologias ao sistema, sem impactos orçamentários significativos, desde que seja viável clínica e economicamente, gerando valor em saúde, na efetividade e nos resultados de desfecho em taxa de sobrevida real

Objectives: This paper discusses the recent impacts of technological advancement on the health market, the growing importance of increasing costs throughout its chain, and investigates an alternative to increasing the access of these new technologies to a greater number of people within supplementary health. In this context, the general objective proposed for this work was to highlight the views of health managers with a prominent role in Brazil regarding the use of risk-sharing models in different regions and in different payers in the system. It was intended to deepen the theme with the fulfillment of the following specific objectives, also from the perspective of the research participants: verify the use of these payment models and consequently determine the characteristics and necessary adaptations for them; identify the challenges for the consolidation of risk-sharing in Supplementary Health; to verify the impact of risk-sharing on health costs of operators based on interviewees' perceptions. Methods: This is a qualitative study with in-depth interviews with main health managers and opinion makers from different institutions in Brazil, totaling 25 interviews. Content analysis was chosen as a method of interpretation and construction of categories for analysis. Results: The results were grouped into the following categories: the suggestions and adaptations suggested for the best operation in the Brazilian reality; criticisms and challenges to the proposed models. The results suggest that risk-sharing may be an alternative capable of promoting a new form of relationship between payers and the manufacturing industry, replacing the traditional remuneration model, which limits the linear relationship with all sectors, always seeking the objective of assisting the patient with new access alternatives. Conclusions: Amid the complexity of the Brazilian health system, it is important for players to evaluate new alternatives for remuneration and technological incorporation. The challenges are innumerable from the realization of the profile of eligible patients and the applicability of risk-sharing that occurs from the interest of both parties in bringing new technologies to the system, without significant budgetary impacts, as long as it is clinically and economically viable, generating health value, effectiveness, and outcomes in survival rates in real world evidences

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.

The Composition of Pharmaceutical Expenditure in National Health Insurance and Implications for Reasonable Spending / 보건행정학회지

BACKGROUND: The proportion of pharmaceutical expenditure out of total health-care expenditure in South Korea is high. In 2016, 25.7% of national health insurance (NHI) spending was for pharmaceuticals. Given the increasing demands for the access to newly introduced medicines and following increase in pharmaceutical spending, the management of NHI pharmaceutical expenditure is becoming more difficult. METHODS: This study analyzed the data claimed to NHI for pharmaceutical reimbursement from 2010 to 2016. RESULTS: The policy implications with respect to the trends and problems in spending by drug groups were elicited. First, the proportion of off-patent drugs spending which were treated to chronic disease was much higher than anti-cancer drug spending. Second, the spending to the newly introduced high-costed medicine increased, however, current price-reduction mechanism was not sufficient to manage their expenditure efficiently. CONCLUSION: Our system seems to need several revisions to improve the efficiency of pharmaceutical expenditure and to cope with high-costed medicines. This study suggested that the prices of off-patent drugs need to be regularly readjusted and the Price-Volume Agreement System should be operated more flexibly as well.

Review of Programs for Improving Patient's Access to Medicines

OBJECTIVE: This study presented the analysis period, the complexity of combined therapy and comparator choice as the key limitations in the economic evaluation of new drugs, and discussed programs for coping with these limitations. METHODS: This study evaluated the post-evaluation, risk-sharing agreement, extra funding program, and flexible incremental cost-effectiveness ratio (ICER) threshold as actions or programs that would increase accessibility to costly new drugs. The study also presented the cases of other countries. The application of the post-evaluation was considered to deal with high uncertainty regarding new drugs. RESULTS: The risk-sharing agreement was introduced in European countries as well as South Korea and has been responsible for the shift from using the financial schemes to outcome-based schemes. The drug funding program has had troubled in securing stable extra funds. The application of higher ICER in the economic evaluation of expensive and innovative oncology drugs was criticized because of the inequity between oncology patients and patients with other diseases. CONCLUSION: Therefore, introducing and applying actions that would increase the accessibility to costly new drugs in South Korea have been deemed necessary after careful reviews and discussions with various stakeholders (insurer, policy makers, pharmaceutical companies and patients).

An Overview of the Risk Sharing Management in Korean National Health Insurance, Focused on the Effect of the Patient Access and Insurance Finance

OBJECTIVE: This study examined the Risk Sharing Agreement (RSA) on pharmaceutical pricing system in Korean national health insurance. Through RSA, the insurer was able to maintain the principles in the price listing process while managing the budget effectively and improving patient access to new drugs. Despite these positive effects, there are still issues raised by some stakeholders, such as lack of transparency in the listing process and doubts about its effectiveness. Therefore, we investigated the impacts of RSA on national health insurance financing and patient access to analyze the effects of RSA. METHODS: The impact of RSA was investigated by analyzing the health insurance claims data for 2014~2016. The degree of improvement in patient access was determined by the decreased amount of patients' payment. RESULTS: Results showed that the financial impact of RSA was not significant and patients' access to the new drug greatly improved. CONCLUSION: These results show that RSA is a good system for improving patient access to new drugs without additional expense on insurance.

The implementing experience of pharmaceutical risk-sharing agreements in Australia, New Zealand, Taiwan area and its implication for mainland China / 中国卫生政策研究

With the rapid development of healthcare technologies, the improvement of patient health expecta-tions, and the increasing of the government or insurer's financial budget pressure, risk-sharing agreements has be-come the focus of the governments or insurer concerned. This article systematically analyzed Australia, New Zealand, Taiwan risk-sharing agreements from five aspects, including the operation main, scope, classification, application processes and the implementation effects. According to the results of the analysis, we suggests that China should im-prove risk-sharing agreements theoretical basis, diversify risk-sharing agreements models, establish risk-sharing a-greements standardization process and so on.

Ethics Research on Physician′s Obligation of Informing-the Reflection on a Case of Medical Dispute / 中国医学伦理学

The realization of patients′right of informed consent depends on the physician′s description of the disease status, medical treatment measures and risks. Performing the obligation of informing according to the laws is conducive to effective communication with patients on the diagnosis and treatment of disease, thereby preventing and reducing medical disputes. Meanwhile, the informing content constitutes its own legal constraint. Based on a case of medical dispute, this paper particularly discussed the performance of obligation of informing, medical risk sharing, as well as the ethical requirements.

The implementing experience of patient access schemes in UK and its implication for China / 中国卫生政策研究

The Risk-sharing agreements have achieved remarkable success in improving patients'access to drugs, lowering the uncertainty of the drugs cost-effectiveness, financial risk control and other aspects of medical in-surance fund , so they have attracted widespread attention by the concerned governments and insurers .This paper sys-tematically reviewed the patient access schemes in UK from several aspects , including the origin of the program , clas-sification , application processes and the implementation effects as well .The results of the research indicated that Chi-na has basically met the conditions for implementation of the risk-sharing agreements .In order to gradually promote the risk-sharing agreements implementation , this paper suggests that China should clarify the main root of risk-sharing agreements implementation , establish risk-sharing agreements standardization process and strengthen the application of health technology assessment in health resources allocation to improve the Chinese medicines bargaining system more scientifically and efficiently .

Thoughts on practice of medical liability insurance at the hospital / 中华医院管理杂志

An analysis of medical liability insurance practice at the hospital in the past 6 years found that medical liability insurance failed to work as expected in medical dispute prevention and risk sharing.The analysis also found that such an insurance will take a greater role in risk sharing,exposure to conflicts,cause discovery,and responsibility,with enhanced government role in guidance and intervention.Thus medical liability insurance is highly advocated by such means as compulsory execution,coordinating mechanism of insurance funding,prevention“medical dispute profiteers”,and adaptation to new approaches in medical dispute settlement.

Medical technology development and globalization: the role of the medical device industry

Modern medical technology aligns with medical device advancement, and new medical devices create the need for new procedure techniques. Unmet needs that physicians experience in clinical practice suggest solutions to problems; to solve these, collaboration between medical device manufacturers and physicians becomes a starting point for new medical device development. Commercialization of medical devices is greatly impacted by the product regulatory approval system, health technology assessment, and reimbursement system. Speed to market plays a far greater role in the medical device market than in the pharmaceutical market. Considering the current trend of evidence-based medicine and value-based pricing, efforts to generate clinical evidence should be strengthened even further while a greater focus should be placed on efforts to introduce global multicenter pre-market clinical trials in the Republic of Korea through strong collaboration with global companies. Since the strength and quality of clinical evidence is comparatively low in medical device studies, this could affect the decision making process and raise the issue of uncertainty. To overcome this issue, a risk-sharing system in the medical device field and 'coverage with evidence development' should be introduced; by doing so, evidence generation opportunities can be created without burying innovative technology and solving the issue of uncertain decisions. In addition, reimbursement coverage to support the costs of clinical studies needs to be established for early evidence generation, as seen in other countries.