Background: Validation of overall survival (OS) extrapolations of immune-checkpoint inhibitors (ICIs) during the National Institute for Health and Care Excellence (NICE) Single Technology Assessment (STA) process is limited due to data still maturing at the time of submission. Inaccurate extrapolation may lead to inappropriate decision-making. The availability of more mature trial data facilitates a retrospective analysis of the plausibility and validity of initial extrapolations. This study compares these extrapolations to subsequently available longer-term data.
Methods: A systematic search of completed NICE appraisals of ICIs from March 2000 to December 2017 was performed. A targeted search was also undertaken to procure published OS data from the pivotal clinical trials for each identified STA made available post-submission to NICE. Initial Kaplan-Meier curves and associated extrapolations from NICE documentation were extracted to compare the accuracy of OS projections versus the most mature data.
Results: The review identified 11 STAs, of which 10 provided OS data upon submission to NICE. The extrapolations undertaken considered parametric or piecewise survival models. Additional data cut-offs provided a mean of 18 months of OS beyond the end of the original data. Initial extrapolations typically under-estimated OS from the most mature data cut-off by 0.4–2.7%, depending on the choice of assessment method and use of the manufacturer- or ERG-preferred extrapolation.
Conclusion: Long-term extrapolation of OS is required for NICE STAs based on initial immature OS data. The results of this study demonstrate that the initial OS extrapolations employed by manufacturers and ERGs generally predicted OS reasonably well when compared to more mature data (when available), although on average they appeared to underestimate OS. This review and validation shows that, while the choice of OS extrapolation is uncertain, the methods adopted are generally aligned with later-published follow-up data and appear appropriate for informing HTA decisions. 相似文献
1. Introduction Development of human industrial civilization and predatory exploitation of natural resources have in- duced tremendous changes of ecological environment. In recent years, territorial and global environmental problems have increased, such as greenhouse effect, depletion of ozone, global warming, water scarcity, water pollution, acid rain, decrease of biodiversity, soil erosion desertification etc. These changes seriously intimidate the health and security of ecosystems, and dist… 相似文献
Recent studies show that corruption is associated with higher military spending [Eur. J. Polit. Econ. 17 (2001) 794] and lower government spending on education and health care [J. Publ. Econ. 69 (1998) 263]. This suggests that policies aimed at reducing corruption may lead to changes in the composition of government outlays toward more productive spending. However, little empirical evidence has been presented to support the claim that public spending improves education and health indicators in developing and transition countries. This paper uses cross-sectional data for 50 such countries to show that increased public expenditure on education and health care is associated with improvements in both access to and attainment in schools, and reduces mortality rates for infants and children. The education regressions are robust to different specifications, but the relationship between health care spending and mortality rates is weaker. 相似文献
By using Data Envelopment Analysis approach, we treat the health production system in a certain province as a Decision Making
Unit (DMU), identify its inputs and outputs, evaluate its technical efficiency in 1982, 1990 and 2000 respectively, and further
analyze the relationship between efficiency scores and social-environmental variables. This paper has found several interesting
findings. Firstly, provinces on frontier in different year are different, but provinces far from the frontier keep unchanged.
The average efficiency of health production has made a significant progress from 1982 to 2000. Secondly, all provinces in
China can be divided into six categories in terms of health production outcome and efficiency, and each category has specific
approach of improving health production efficiency. Thirdly, significant differences in health production efficiencies have
been found among the eastern, middle and western regions in China, and among the eastern and middle regions. At last, there
is significant positive relationship between population density and health production efficiency but negative relationship
(not very significant) between the proportions of public health expenditure in total expense and efficiency. Maybe it is the
result of inappropriate tendency of public expenditure. The relationship between abilities to pay for health care services
and efficiency in urban areas is opposite to that in rural areas. One possible reason is the totally different income and
public services treatments between rural and urban residents. Therefore, it is necessary to adjust health policies and service
provisions which are specifically designed to different population groups.
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Translated from Jingji Yanjiu 经济研究 (Economic Research Journal), 2006, (7): 92–105 相似文献
This paper investigates the causal impact of large unexpected windfalls on individual mental health, physical health, as well as health behaviors. I use a large individual-level panel data set of lottery winners from Germany between the years 2000 and 2011 and observe lottery winners before and after winning a large lottery prize. Mental health declines immediately after winning a large lottery prize for individuals with low education and low levels of financial literacy. While these individuals report being happier after winning the lottery, evidence from commonly used SF-12 measures of mental health indicates that winners with low education experience increased role limitations due to emotional problems, are more anxious, and have less energy after their win. The impact on various measures of mental health is highly robust, statistically significant, economically significant, and persists for up to two years after the win. Unexpected windfalls have no impact on the mental health of individuals with high education or high financial literacy. Winning the lottery has no impact on individuals’ health behaviors such as smoking or alcohol consumption, and it has no impact on doctor visits, hospital stays, or illness-related work absences regardless of education level. 相似文献
To characterize a US population of patients with irritable bowel syndrome with constipation (IBS-C) or chronic idiopathic constipation (CIC) using CONTOR, a real-world longitudinal research platform that deterministically linked administrative claims data with patient-reported outcomes data among patients with these conditions. 相似文献