[Judicialization of high priced medications in Argentina: quali-quantitative study]. / Judicialización de medicamentos de alto precio en Argentina: estudio cuali-cuantitativo.

INTRODUCTION: The economic consequences of mandatory coverage, through judicial means, of high-priced medications constitutes a growing problem, which merits knowing its local characteristics to provide possible solutions. OBJECTIVE: To identify medications, diseases involved, economic impact and contextual factors of the judicialization of high-priced medications in the Argentine Health System(MEP). METHODS: Quali-quantitative descriptive study that retrospectively analyzed legal protection resources by MEP from three national and provincial databases from January 2017 to December 2020, evaluating the existing relationship between lawsuits with regulatory approval, inclusion in benefit packages and relationship with journalistic articles for the three most frequently prosecuted drugs. RESULTS: 405 lawsuits were included, mainly from the Ministry of National Health. The three most prosecuted medications were nusinersen (21.7%), palbociclib (5.9%) and agalsidase-alfa (4.7%). Only 69.4% of medications were approved for marketing in Argentina at the time of the protection; 45.7% were incorporated into the Single Reimbursement System, and 16.8% had a report from the National Commission for the Evaluation of Health Technologies and Clinical Excellence (CONETEC), which was negative in 87.1% of cases. The average time from request to provision of the medication was 150 days. A temporal correlation was observed between the appearance of the MEP in the national graphic press and the appeals occurrence. CONCLUSIONS: Judicialization focused on very highpriced medications for rare or oncological diseases. The rulings were mostly in favor of the plaintiff, and access times to the medication took a long time. The mass media anticipated the judicial processes.

Introducción: Las consecuencias económicas de la cobertura obligatoria, vía judicial, de medicamentos de alto precio constituye un problema creciente, que amerita conocer sus características locales para aportar posibles soluciones. OBJETIVO: Identificar medicamentos, enfermedades, impacto económico y factores contextuales de la judicialización de medicamentos de alto precio (MEP) Argentina. Métodos: Estudio descriptivo cuali-cuantitativo que analizó retrospectivamente recursos de amparos legales por MEP de tres bases de datos nacionales y provinciales durante 4 años, evaluando relación existente entre amparos con aprobación regulatoria, inclusión de los MEP al paquete de beneficios y relación con notas periodísticas. RESULTADOS: Se incluyeron 405 amparos provenientes principalmente del Ministerio de Salud Nacional. Los tres medicamentos más judicializados fueron nusinersen (21.7%), palbociclib (5.9%) y agalsidasa-alfa (4.7%). Solo el 69.4% de los medicamentos se encontraban aprobados para la comercialización en Argentina al momento del amparo; el 45.7% se encontraban incorporados al Sistema Único de Reintegros y el 16.8% contaban con informe de la Comisión Nacional de Evaluación de Tecnologías Sanitarias y Excelencia Clínica (CONETEC), negativa en el 87.1% de casos. El tiempo promedio desde la solicitud hasta la provisión del medicamento fue de 150 días. Se observó una correlación temporal entre la aparición del MEP en la prensa nacional gráfica y la presentación de amparos de dicho MEP. CONCLUSIONES: La judicialización se concentró en medicamentos de altísimo precio para enfermedades poco frecuentes u oncológicas. Los fallos fueron mayoritariamente a favor del demandante, siendo los tiempos de acceso al medicamento prolongados. Los medios de comunicación anticiparon los procesos judiciales.

A Systematic Review of Value Criteria for Next-Generation Sequencing/Comprehensive Genomic Profiling to Inform Value Framework Development.

OBJECTIVES: To comprehensively identify and map an exhaustive list of value criteria for the assessment of next-generation sequencing/comprehensive genomic profiling (NGS/CGP), to be used as an aid in decision making. METHODS: We conducted a systematic review to identify existing value frameworks (VFs) applicable to any type of healthcare technology. VFs and criteria were mapped to a previously published Latin American (LA) VF to harmonize definitions and identify additional criteria and or subcriteria. Based on this analysis, we extracted a comprehensive, evidence-based list of criteria and subcriteria to be considered in the design of a NGS/CGP VF. RESULTS: A total of 42 additional VFs were compared with the LA VF, 88% were developed in high-income countries, 30% targeted genomic testing, and 16% specifically targeted oncology. A total of 242 criteria and subcriteria were extracted; 227 (94%) were fully/partially included in the LA VF; and 15 (6%) were new. Clinical benefit and economic aspects were the most common criteria. VFs oriented to genomic testing showed significant overlap with other VFs. Considering all criteria and subcriteria, a total of 18 criteria and 36 individual subcriteria were identified. CONCLUSIONS: Our study provides an evidence-based set of criteria and subcriteria for healthcare decision making useful for NGS/CGP as well as other health technologies. The resulting list can be beneficial to inform decision making and will serve as a foundation to co-create a multistakeholder NGS/CGP VF that is aligned with the needs and values of health systems and could help to improve patient access to high-value technologies.

Toward the Implementation of a Value Framework for Diagnostic Technologies: Operationalization, Piloting, and Validation in Latin America.

OBJECTIVES: In 2020, a group of 30 stakeholders from Latin America established 15 criteria for a diagnostic technologies value framework (D-VF) to help assess and inform decisions on diagnostic technologies. This article aims to present the operationalization, piloting, and initial validation of the framework for its implementation. METHODS: This work was carried out collaboratively with a variety of stakeholders. Three sequential phases were undertaken: (1) operationalization of the D-VF through a literature search for conceptual definitions and assessment tools, (2) piloting of the D-VF through a rapid health technology assessment document applying the methodology of the framework, and (3) a face validation process conducted through a virtual workshop, where usefulness and implementation aspects of the framework were assessed. RESULTS: The operationalization of the framework was conducted, and a methodological user guide was published. The D-VF criteria were applied in a health technology assessment document on human papilloma virus testing in cervical cancer screening. Also, an open-access training program was developed. Stakeholders agreed on the usefulness of the D-VF for assessment and decision-making stages of diagnostic technologies. However, they highlighted the need to improve technical capacities and the potential for added complexity when applying a D-VF with many criteria. The absence of an established value framework for diagnostic technologies in Latin America and the potential for strengthening technical capacities made the project valuable to those involved. CONCLUSIONS: The diagnostic technologies value framework was shown to be fit for implementation in real-life decision-making settings after the operationalization, piloting, and initial validation phases. Further experiences are important to support its implementation.

Deliberative processes in decision making informed by health technology assessment in Latin America.

OBJECTIVE: The objective of Health Technology Assessment International's 6th Latin America Policy Form, held in 2021, was to explore the implementation of deliberative processes in the framework of health technology assessment (HTA) and how agencies in the region could involve stakeholders in this process. METHODS: This paper is based on a preparatory survey, a background document, and the deliberative work of participants at the virtual Forum conducted in 2021. There were ninety-one participants in the open session and fifty-two in the closed sessions, representing twelve countries and diverse areas of the health sector. RESULTS: While there are mechanisms in most countries in Latin America to consider stakeholder involvement to some degree, it remains reduced or limited to a consultative role, making true participative involvement rare. There are significant barriers and structural and contextual limitations that have impeded or slowed progress toward deliberative processes. Relatively low levels of institutionalization and knowledge about HTA, as well as the lack of trust among stakeholders are important challenges. This situation has impacted health systems by diminishing the legitimacy of decisions and the very structures and processes of HTA. CONCLUSION: The Forum's broad group of participants identified barriers, facilitators, and recommendations to improve the use of deliberative processes in Latin America to foster improved fairness and reasonableness in HTA and decision making.

Evidence-Informed Update of Argentina's Health Benefit Package: Application of a Rapid Review Methodology.

OBJECTIVES: Argentina has a fragmented healthcare system with social security covering almost two thirds of the population. Its benefit package-called compulsory medical program (PMO; by its Spanish acronym Programa Médico Obligatorio)-has not been formally and widely updated since 2005. However, laws, clinical practice guidelines (CPGs), and a high-cost technology reimbursement fund complement it. Our objective was to comprehensively review such a PMO and propose an update considering the corresponding complementary sources. METHODS: We followed four steps: (i) identification of health technologies from the current PMO and complementary sources, (ii) prioritization, (iii) assessment through rapid health technology assessment (HTA), and (iv) appraisal and recommendations. We evaluated three value domains: quality of evidence, net benefit, and economics, which were summarized in a five-category recommendation traffic-light scale ranging from a strong recommendation in favor of inclusion to a strong recommendation for exclusion. RESULTS: Eight hundred fifty technologies were identified; 164 of those, considered as high priority, were assessed through rapid HTAs. Those technologies mentioned in laws and CPGs were mostly outpatient essential medicines, whereas those from the reimbursement system were mostly high-cost drugs; of these 101 technologies, 50 percent were recommended to be kept in the PMO. The other 63 (identified by the Superintendence of Health Services, technology producers, and patients) were mostly medical procedures and high-cost drugs; only 25 percent of those resulted in a favorable recommendation. CONCLUSIONS: A methodology based on four clearly identified steps was used to carry out a comprehensive review of an outdated and fragmented benefit package. The use of rapid HTAs and a traffic-light recommendation framework facilitated the deliberative evidence-based update.

Identification and selection of health technologies for assessment by agencies in support of reimbursement decisions in Latin America.

OBJECTIVE: There is no health system that has the resources to evaluate all technologies. The presence of a clear process to prioritize health technologies for assessment by health technology assessment (HTA) agencies is a good practice principle recognized at the international level. The objective of Health Technology Assessment International's 2020 Latin American Policy Forum (LatamPF) was to explore how to improve the way HTA agencies in Latin America identify and prioritize technologies for assessment. METHODS: This paper is based on a background document, a survey, and the deliberations of the members of the LatamPF (forty-six participants from eleven countries) using a design thinking methodology. RESULTS: Participants agreed that a lack of clear prioritization mechanisms results in HTA processes and decisions that are perceived to be of low transparency and overly exposed to political or interest group pressures. The LatamPF identified barriers and recommended actions to improve HTA prioritization mechanisms in Latin America. The criteria identified as the most important to be taken into consideration by HTA agencies in the region when prioritizing a technology for assessment were: the burden of illness, the potential clinical benefit, the alignment with national health priorities, the potential impact on equity, a lack of treatment alternatives for patients, and the potential economic impact. CONCLUSIONS: Forum participants agreed that the establishment of transparent prioritization processes is a key element for all health systems. Improvements in these processes will strengthen HTA and provide greater legitimacy to decision making.

[Mecanismos de identificación y priorización de las tecnologías sanitarias a ser evaluadas por las Agencias de ETESA para decisiones de cobertura en Latino America].

OBJETIVO: Ningún sistema de salud cuenta con los recursos necesarios para evaluar todas las tecnologías. Contar con un proceso claro para priorizar qué tecnologías serán evaluadas por las agencias de evaluación de tecnologías sanitarias (ETESA) constituye un principio de buena práctica reconocido a nivel internacional. El objetivo del Foro de Políticas en Latino América (LatamPF) 2020 de Health Technology Assessment International fue explorar cómo puede mejorarse la forma en que las agencias de ETESA de Latino América identifican y priorizan las tecnologías a ser evaluadas. MÉTODOS: Este manuscrito está basado en un documento base, una encuesta, y en el trabajo deliberativo realizado por los miembros (cuarenta y seis participantes, once países) que participaron del LatamPF, a través de la metodología design-thinking. RESULTADOS: Los participantes coincidieron en que la falta de mecanismos claros de priorización trae como consecuencia una falta de legitimidad de las decisiones y procesos de ETESA, que son percibidos como poco transparentes y demasiado expuestos a presiones políticas o de grupos de interés. También se identificaron barreras y acciones para mejorar los mecanismos de priorización de ETESA en América Latina. Los criterios identificados como más importantes para ser tenidos en cuenta por las agencias de ETESA de la región al momento de priorizar una tecnología para ser evaluada fueron la carga de enfermedad, el potencial beneficio clínico, la alineación con prioridades de salud nacionales, el potencial impacto en la equidad, ausencia de otras alternativas para los pacientes, y el potencial impacto económico. CONCLUSIONES: Los participantes del Foro coincidieron en que el establecimiento de procesos transparentes de priorización es un elemento clave para todos los sistemas de salud. Las mejoras en este proceso fortalecerán la ETESA en Latino América y darán mayor legitimidad a sus decisiones.

A Value Framework for the Assessment of Diagnostic Technologies: A Proposal Based on a Targeted Systematic Review and a Multistakeholder Deliberative Process in Latin America.

OBJECTIVES: there are very few value frameworks (VFs) to assess health technologies that are focused on diagnostic tests; they usually do not reflect a multistakeholder process; and they are all developed in high-income countries. Our project performed a targeted systematic review, with the objective of proposing an evidence-based, up-to-date VF informed by a multinational multistakeholder group working in the health technology assessment (HTA) space. METHODS: (1) A targeted systematic review, with the aim to identify existing VFs and their dimensions; and (2) generation a VF proposal through a mixed-methods, qualitative-quantitative approach. RESULTS: From 73 citations identified, 20 met our inclusion criteria and served to provide the initial list of dimensions for our VF. An initial list of criteria and subcriteria for a preliminary VF was proposed. After a full-day deliberative face-to-face meeting with 30 relevant stakeholders from seven Latin American countries and the United Kingdom, the final VF was defined, consisting of 15 criteria: five "essential or core," six highly relevant, three moderately relevant, and one of low relevance. Barriers and facilitators of value assessment of diagnostic technologies were also discussed. CONCLUSIONS: We propose a VF oriented to diagnostic technologies based on a targeted systematic review and a participatory process with key HTA stakeholders. It is the first to be produced in a lower and middle income setting but can also be potentially useful in other contexts aimed to assist decision-making processes with these particularly complex health technologies.

[Epidemiology, resource use and costs of rheumatoid arthritis in Argentina]. / Epidemiología, uso de recursos y costos de la artritis reumatoidea en Argentina.

This review aims to identify information on epidemiological parameters and estimate the cost of moderate to severe rheumatoid arthritis (RA). A search for related literature was carried out in major databases. A consensus of local rheumatology experts was used to find the most realistic parameters, using a modified Delphi method. Direct medical costs were estimated, using information collected from the cost per unit database of the Instituto de Efectividad Clínica y Sanitaria de Argentina. Indirect costs were estimated using the human resources approach. Costs were expressed in US dollars (USD) as of November 2017. The reported prevalence of RA in Argentina was 0.94% (95%CI: 0.86 to 1.02), with an annual incidence rate of 19 per 100,000 people (95%CI: 17 to 20). The annual cost of disease-modifying drugs was 33,936.10 USD per patient. The cost attributed to serious infections was 2,474.6 USD. The cost of bilateral knee replacement per patient was $5,276.8 USD; and the cost for total hip replacement was $9,196.4. Both, the cost of hospitalization days per patient per year, and the indirect costs of RA increased as the disability score increased. This review reports useful information on epidemiological and cost parameters of moderate to severe RA, in the era of biological agents, in order to be useful for conducting economic evaluations regarding health in Argentina.

La presente revisión tiene como objetivo identificar información sobre parámetros epidemiológicos y estimar el costo de la artritis reumatoidea (AR) moderada a severa. Se llevó a cabo una búsqueda de la literatura en las principales bases de datos. Se recurrió a un consenso de expertos locales en reumatología para encontrar los parámetros más realistas, utilizando un método Delphi modificado. Se estimaron los costos médicos directos, utilizando información recopilada en la base de datos de costos unitarios del Instituto de Efectividad Clínica y Sanitaria de Argentina. Los costos indirectos se estimaron a través del enfoque del capital humano. Los costos se expresaron en dólares estadounidenses (USD) a noviembre de 2017. La prevalencia reportada de AR en Argentina fue 0,94% (IC95%: 0,86 a 1,02), con una tasa de incidencia anual de 19 cada 100 000 personas (IC95%: 17 a 20). El costo anual de las drogas modificadoras de la enfermedad fue de USD 33 936,10 por paciente. El costo atribuido a las infecciones serias fue de USD 2474,6. El costo del reemplazo bilateral de rodillas por paciente fue de USD 5276,8, y el del reemplazo total de cadera, de USD 9196,4. El costo por paciente por año de días de hospitalización y los costos indirectos de la AR se acrecentaron al aumentar el puntaje de discapacidad. La revisión reporta información útil acerca de parámetros epidemiológicos y de costos de la AR moderada a severa en la era de los agentes biológicos, con el fin de resultar de utilidad para la conducción de evaluaciones económicas de salud en Argentina.

Epidemiología, uso de recursos y costos de la artritis reumatoidea en Argentina / Epidemiology, resource use and costs of rheumatoid arthritis in Argentina

RESUMEN La presente revisión tiene como objetivo identificar información sobre parámetros epidemiológicos y estimar el costo de la artritis reumatoidea (AR) moderada a severa. Se llevó a cabo una búsqueda de la literatura en las principales bases de datos. Se recurrió a un consenso de expertos locales en reumatología para encontrar los parámetros más realistas, utilizando un método Delphi modificado. Se estimaron los costos médicos directos, utilizando información recopilada en la base de datos de costos unitarios del Instituto de Efectividad Clínica y Sanitaria de Argentina. Los costos indirectos se estimaron a través del enfoque del capital humano. Los costos se expresaron en dólares estadounidenses (USD) a noviembre de 2017. La prevalencia reportada de AR en Argentina fue 0,94% (IC95%: 0,86 a 1,02), con una tasa de incidencia anual de 19 cada 100 000 personas (IC95%: 17 a 20). El costo anual de las drogas modificadoras de la enfermedad fue de USD 33 936,10 por paciente. El costo atribuido a las infecciones serias fue de USD 2474,6. El costo del reemplazo bilateral de rodillas por paciente fue de USD 5276,8, y el del reemplazo total de cadera, de USD 9196,4. El costo por paciente por año de días de hospitalización y los costos indirectos de la AR se acrecentaron al aumentar el puntaje de discapacidad. La revisión reporta información útil acerca de parámetros epidemiológicos y de costos de la AR moderada a severa en la era de los agentes biológicos, con el fin de resultar de utilidad para la conducción de evaluaciones económicas de salud en Argentina.

ABSTRACT This review aims to identify information on epidemiological parameters and estimate the cost of moderate to severe rheumatoid arthritis (RA). A search for related literature was carried out in major databases. A consensus of local rheumatology experts was used to find the most realistic parameters, using a modified Delphi method. Direct medical costs were estimated, using information collected from the cost per unit database of the Instituto de Efectividad Clínica y Sanitaria de Argentina. Indirect costs were estimated using the human resources approach. Costs were expressed in US dollars (USD) as of November 2017. The reported prevalence of RA in Argentina was 0.94% (95%CI: 0.86 to 1.02), with an annual incidence rate of 19 per 100,000 people (95%CI: 17 to 20). The annual cost of disease-modifying drugs was 33,936.10 USD per patient. The cost attributed to serious infections was 2,474.6 USD. The cost of bilateral knee replacement per patient was $5,276.8 USD; and the cost for total hip replacement was $9,196.4. Both, the cost of hospitalization days per patient per year, and the indirect costs of RA increased as the disability score increased. This review reports useful information on epidemiological and cost parameters of moderate to severe RA, in the era of biological agents, in order to be useful for conducting economic evaluations regarding health in Argentina.

The link between health technology assessment and decision making for the allocation of health resources in Latin America.

OBJECTIVE: One of the good practice principles for health technology assessment (HTA) is having a clear link between the assessment and decision making. The objective of the 2019 Latin American Policy Forum (LatamPF) of Health Technology Assessment International was to explore different models of connection between HTA and decision making and to discuss the potential applicability of such models in Latin America. METHODS: This paper is based on a background document and the deliberations of the members of the LatamPF (fifty-four participants from twelve countries) where a design-thinking methodology was used. RESULTS: The participants agreed that insufficient links between HTA and decision making undermine the legitimacy of decisions, expose the HTA process to excessive political and judicial influence, and promote the exclusion of some stakeholders from participating in the assessment process and decision making. High priority aspects of the HTA process that could feasibly be improved and which hold the greatest potential to generate positive changes in the health systems in the region were identified. The majority of these aspects were associated with the appropriate institutionalization of HTA, a greater degree of participation by different stakeholders, and improved transparency in the HTA process. CONCLUSIONS: The LatamPF identified barriers and recommended actions to strengthen the link between HTA and decision making. Participants emphasized that there is now a window of opportunity in the region as many societal actors see this as a priority. For this reason, health system stakeholders must take this opportunity to increase efforts toward strengthening the link between HTA and decision making.

[Vínculo Entre La Evaluación De Tecnologías Sanitarias y La Toma De Decisión Para La Asignación De Recursos Sanitarios en Latino-América].

OBJETIVOS: Un vínculo claro entre la evaluación y la toma de decisión constituye un principio de buena práctica en evaluación de tecnologías sanitarias (ETESA) reconocido a nivel internacional. El objetivo del Foro de Políticas en Latino-América (LatamPF) 2019 de Health Technology Assessment International fue explorar los diferentes modelos que vinculan la ETESA y la toma de decisión y discutir su potencial aplicabilidad en Latino-América. MÉTODOS: Este manuscrito está basado en un documento base y en el trabajo deliberativo realizado por los miembros (54 participantes, 12 países) que asistieron al LatamPF, a través de la metodología design thinking. RESULTADOS: Los participantes coincidieron en que la relación inapropiada entre la ETESA y la toma de decisión atenta hoy contra la legitimidad de las decisiones, expone al proceso de ETESA a una excesiva influencia política y judicial, y condiciona que algunos actores se sientan relegados del proceso de evaluación y toma de decisión. Se identificaron los atributos del proceso de ETESA más prioritarios y factibles de ser mejorados en la región, y con el mayor potencial para generar un cambio positivo en los sistemas de salud. La mayor parte de estos están vinculados con la apropiada institucionalización de la ETESA, ampliar la participación de los diferentes actores y mejorar la transparencia de los procesos de ETESA. CONCLUSIONES: El LatamPF ha identificado barreras y recomendado acciones para reforzar el vínculo entre ETESA y la decisión. A su vez, existe en estos momentos una ventana de oportunidad en la región, ya que el tema es visualizado como una prioridad por gran parte de los actores de la sociedad. Por ello, los diferentes actores de los sistemas sanitarios deberían ahora tomar esta oportunidad para avanzar en el fortalecimiento del vínculo entre ETESA y toma de decisión.