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
Summary. Starr (1973) showed that, if people have different subjective probabilities, ex ante and ex post efficiency conflict. Conversely, under the simple preferences that he considered, the discrepancy between ex ante and ex post efficiency disappears when subjective probabilities are identical. Here I consider identical subjective probabilities, but
more general preferences. First, risk attraction is admitted. Second, I dispense with the double requirement (dubbed IZU)
of additive separability and state-independence of the utility of zero-date consumption, an unrealistic requirement when modeling
the investment in durable goods. I find that, under IZU, and as long as ex post preferences satisfy the natural assumption of quasiconcavity (and satisfy some technical qualifications), an ex ante efficient allocation is indeed ex post efficient, but the converse is not necessarily true under risk attraction. If, on the other hand, IZU is violated, then one
can have ex ante efficient allocations that are not ex post efficient, and vice-versa, even under risk aversion.
Received: June 25, 1999; revised version: March 2001 相似文献
Consumption cues (e.g., brands, money, and advertisements) can have powerful effects on cognition, perception, and behavior, yet how people regulate responses to such cues is not well understood. This is surprising given that consumption cues are increasingly present in nontraditional consumer contexts, such as healthcare, education, and politics. This research develops a measure of two types of consumer regulation strategies, cue‐based and budget‐based (studies 1–4), and demonstrates that these strategies influence how people respond to consumption cues in a political context (study 5). Specifically, in a study involving the 2012 American Presidential Election, priming survey participants as consumers (versus citizens) influenced both voting intentions and self‐reported voting behavior, and the newly developed consumer regulation scale was instrumental in detecting this effect. These findings suggest there may be merit in the escalating debate and concern over referring to voters as consumers. 相似文献
Logistic and power law methodologies for both retrospective and prospective analyses of extended time series describing evolutionary growth processes, in environments with finite resources, are confronted. While power laws may eventually apply only to the early stages of said growth process, the Allee logistic model seems applicable over the entire span of a long range process. On applying the Allee logistic model to both the world population and the world gross domestic product time series, from 1 to 2008 AD, a projection was obtained that along the next few decades the world should experience a new economic boom phase with the world GDP peaking around the year 2020 and proceeding from then on towards a saturation value of about 142 trillion international dollars, while the world population should reach 8.9 billion people by 2050. These results were then used to forecast the behavior of the supply and consumption of energy and food, two of the main commodities that drive the world system. Our findings suggest that unless the currently prevailing focus on economic growth is changed into that of sustainable prosperity, human society may run into a period of serious economical and social struggles with unpredictable political consequences. 相似文献
This paper examines control measures for improving food safetyin the dairy chain, using an integer linear programming model.The chain includes feed (compound feed production and delivery),farm (dairy farm) and dairy processing (transport and processingof raw milk, delivery of pasteurised milk) blocks. Results showthat 65 per cent of the maximum possible food safety improvementcan be achieved at relatively low extra cost per ton of milk(4.27), fairly evenly distributed across the blocks. Highersafety levels can be attained by increasing the farm block'scontribution, but at much higher extra cost (44.37 for the maximumattainable). 相似文献
The interactions between customers and organizations have been regarded as a core aspect in cocreating and developing new products and practices. The current study explores these interactions in the context of citizens and nonprofit organizations. The study comprises two parts: (a) a qualitative approach using interviews and the analysis of social networks to better understand the organization's social media marketing practices and corresponding outcomes and (b) a quantitative approach to analyze antecedents and outcomes of trust and being committed to participate in a nonprofit organization such as Amnesty International. From the qualitative approach, the findings highlight that Amnesty International uses social media (Facebook) to share information, and citizens tend to consider sharing posts, but they do not express new ideas or interact with the organization. These findings contribute to structure the model analyzed in the second stage of the research. Regarding the qualitative approach, the results show that organizational credibility is the most significant driver of trust followed by communicative ability. Affectively commitment to participation and relationship perception act as outcomes of overall commitment. 相似文献
We investigate the relationship between the transmission of price volatility and market power in the German fresh pork supply chain. We use a theoretical model underpinning this relationship followed by an empirical application that uses monthly farm, slaughterhouse and retail pork price data for the period 2000–2011. We examine both the relationships of market power with price level transmission and price volatility transmission in the chain. We use a vector error correction model and least squares regressions to analyse price transmission and price volatility transmissions, respectively. Results show that retail market power limited both types of transmissions. Competition inducing policy measures coupled with measures that support price risk management initiatives of chain actors are suggested. 相似文献
A new multi-logistic methodology to analyze long range time series of evolutionary S-shaped processes is presented. It conceptually innovates over the traditional logistic approach. The ansatz includes computing the residuals to an optimized multi-logistic trend curve least squares fitted to the time-series data. The elements of the residuals series are checked for autocorrelations and once detected the residuals series is further analyzed to search for eventual presence of underlying periodic structures using a truncated Fourier sine series. The method foundations ensures both a universal applicability and a capacity to disclose the existence of active clocks that can be possibly traced to the driving motors of the evolutionary character of the time series, due to the responsiveness of corresponding process to the development of economic cycles. On associating these two views, it is found that the methodology has a strong potential to improve the quality of short-term forecasts. These findings have been put to test through applications of the methodology to studying the time evolution of two commodities of strong economic and social importance (corn and steel) and good results were consistently obtained for both the analytical and forecasting aspects. 相似文献