ABSTRACT
@#The umbrella trial has received increasing attention in the design of clinical trials for oncology drugs in recent years. This trial design categorizes a single disease into multiple sub-types based on predictive biomarkers or other predictive factors, and simultaneously evaluates the efficacy of multiple targeted therapies. When compared with the traditional drug development model of phase Ⅰ, phaseⅡ, and phase Ⅲ randomized controlled trials, umbrella trials are a more scientifically rigorous trial design that can speed up drug evaluation to address the conflict between numerous untested drugs and diseases with a lack of effective treatment options. This article will focus on the concept, main characteristics, eligibility criteria, design and statistical considerations, ethical considerations, and future directions of umbrella trials, with the aim of providing methodological guidance for the design of clinical trials for oncology drugs.
ABSTRACT
With the enormous resources having been invested in oncology drugs development in China in recent years, the Center for Drug Evaluation (CDE) of National Medical Products Administration has been issuing a number of technical guidelines to further standardize the requirements on implementation and registration of domestic oncology clinical trials. As data is the cornerstone of clinical trials, data integrity and quality will directly decide the outcome of clinical studies. Given the specific characteristics of oncology therapeutic clinical trials, and combined with the clinical data standards established by the Clinical Data Interchange Standards Consortium (CDISC) and the issued industrial guidelines, this article introduces the general considerations of clinical data management for oncology clinical trials, with the aim of emphasizing normative data collection and timely data monitoring to ensure the data quality and reliability of results of the study. This article discusses the impact of complex study design on CRF, design CRF according to CDASH, develop DVP scientifically, rolling submissions and data cut-off.
ABSTRACT
Dentro de las evaluaciones fármaco-económicas habituales de nuevas drogas, se ha generalizado el uso del análisis de impacto presupuestario, que es un complemento de las evaluaciones más conocidas de costo-efectividad y costo-utilidad y tiene una importancia fundamental al momento de decidir la incorporación de una nueva molécula (o intervención terapéutica) al formulario terapéutico de una organización o subsistema de salud. Varios factores, no utilizados habitualmente en evaluaciones de costo-efectividad, son necesarios para los análisis de impacto presupuestario, incluyendo el tamaño de la población pasible de recibir tratamiento y las tasas de difusión en el mercado de la nueva intervención, entre otros. Se presenta en este artículo un prototipo básico de modelo de impacto presupuestario (MIP) y se discute la relevancia de los datos que se obtienen de ellos y su utilidad para quien toma las decisiones dentro de las organizaciones de salud.
In recent years, budget impact analysis have become morecommon among pharmaco-economic evaluations, this typeof analysis is complementary to the more traditional healthtechnology assessments like cost effectiveness analysisand cost utility analysis, and have an important role toplay when making decisions regarding the incorporationof a new drug (or technology) to the therapeutic formularyof a health organization. Several factors not usedin standard cost effectiveness analysis are needed whenperforming a budget impact analysis, including the sizeof the whole population able to be treated with the newdrug and the penetration rates of the new technology inthe target population. In this paper, a basic prototype of abudget impact model is presented, and the relevance forthe decision makers of the data obtained by the budgetimpact models is discussed.