RESUMO
BACKGROUND: Economic evaluations have been widely used to inform and guide policy-making process in healthcare resources allocation as a part of an evidence package. An intervention is considered cost-effective if an ICER is less than a cost-effectiveness threshold (CET), where a CET represents the acceptable price for a unit of additional health gain which a decision-maker is willing to pay. There has been discussion to increase a CET in many settings such as the United Kingdom and Thailand. To the best of our knowledge, Thailand is the only country that has an explicit CET and has revised their CET, not once but twice. Hence, the situation in Thailand provides a unique opportunity for evaluating the impact of changing CET on healthcare expenditure and manufacturers' behaviours in the real-world setting. Before we decide whether a CET should be increased, information on what happened after the CET was increased in the past could be informative and helpful. OBJECTIVES: This study protocol describes a proposed plan to investigate the impact of increased cost-effectiveness threshold using Thailand as a case study. Specifically, we will examine the impact of increasing CET on the drug prices submitted by pharmaceutical companies to the National List of Essential Medicine (NLEM), the decision to include or exclude medications in the NLEM, and the overall budget impact. MATERIALS AND DESIGNS: Retrospective data analysis of the impact of increased CET on national drug committee decisions in Thailand (an upper middle-income country) will be conducted and included data from various sources such as literature, local organizations (e.g. Thai Food and Drug Administration), and inputs from stakeholder consultation meetings. The outcomes include: (1) drug price submitted by the manufacturers and final drug price included in the NLEM if available; (2) decisions about whether the drug was included in the NLEM for reimbursement; and (3) budget impact. The independent variables include a CET, the variable of interest, which can take values of THB100,000, THB120,000, or THB160,000, and potential confounders such as whether this drug was for a chronic disease, market size, and primary endpoint. We will conduct separate multivariable regression analysis for each outcome specified above. DISCUSSION: Understanding the impact of increasing the CET would be helpful in assisting the decision to use and develop an appropriate threshold for one's own setting. Due to the nature of the study design, the findings will be prone to confounding effect and biases; therefore, the analyses will be adjusted for potential confounders and statistical methods will be explored to minimize biases. Knowledge gained from the study will be conveyed to the public through various disseminations such as reports, policy briefs, academic journals, and presentations.
Assuntos
Formulação de Políticas , Análise Custo-Benefício , Preparações Farmacêuticas , Estudos Retrospectivos , TailândiaRESUMO
El cáncer de mama es la causa más común de muerte por cáncer en el mundo, y la resistencia a los medicamentos es una de las barreras más importantes para el éxito de la terapia de la enfermedad. Es fundamental tener una comprensión sólida de los procesos moleculares que impulsan la resistencia al tratamiento en el cáncer de mama para diseñar terapias dirigidas con el potencial de superar esta resistencia. Estos mecanismos son complejos y multifacéticos e incluyen la activación de vías de señalización que promueven la supervivencia y proliferación celular, la regulación positiva de las bombas de salida de fármacos, la aparición de células madre cancerosas y cambios genéticos y epigenéticos. Esta revisión de la literatura brinda una descripción general de estos mecanismos y analiza las posibles estrategias para superar la resistencia a los medicamentos en el cáncer de mama, incluido el uso de terapias dirigidas que se dirigen específicamente a las vías y los mecanismos involucrados en la resistencia a los medicamentos. La revisión también destaca la necesidad de más investigación para identificar estrategias efectivas para superar la resistencia a los medicamentos y mejorar los resultados del tratamiento en pacientes con cáncer de mama.
Breast cancer is the most common cause of death from cancer in the world, and drug resistance is one of the most significant barriers to successful therapy for the disease. It is critical to have a solid understanding of the molecular processes driving treatment resistance in breast cancer to design targeted therapies with the potential to overcome this resistance. These complex and multifaceted mechanisms include the activation of signaling pathways that promote cell survival and proliferation, the upregulation of drug efflux pumps, the emergence of cancer stem cells, and genetic and epigenetic changes. This literature review provides an overview of these mechanisms. It discusses potential strategies for overcoming drug resistance in breast cancer, including targeted therapies that specifically target the pathways and mechanisms involved in drug resistance. The review also highlights the need for further research to identify effective strategies for overcoming drug resistance and improving treatment outcomes in breast cancer patients.