A review and validation of overall survival extrapolation in health technology assessments of cancer immunotherapy by the National Institute for Health and Care Excellence: how did the initial best estimate compare to trial data subsequently made available?

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.     

Managing the environmental adaptation process in supplier–customer relationships

This paper details the results and managerial implications from four case studies, which examine how the environmental adaptation process (EAP) is managed within business‐to‐business relationships. The research uses models of supplier–customer interaction and inter‐organization cooperation in order to explore inter‐firm relationships and the process of adaptation. The research findings show that either party might pursue adaptations and also establishes features of the process itself as well as identifying factors that can facilitate or hinder the introduction of environmental changes. Arriving at a satisfactory outcome to the adaptation process can be determined by individual company and relationship characteristics, as well as the behaviour and experience of those managers involved in the process. Guidelines for the management of the process of environmental adaptation are proposed. Copyright © 2001 John Wiley & Sons, Ltd and ERP Environment     

Reviews

Who gets access to training? Machin, S. and Wilkinson, D. 1995: EMPLOYEE TRAINING: UNEQUAL ACCESS AND ECONOMIC PERFORMANCE. London: Institute for Public Policy Research, £3.95 paper.

Spatial planning. Williams, R.H. 1996: EUROPEAN UNION SPATIAL POLICY AND PLANNING. London: Paul Chapman, £16.95 paper.

Small firm growth. Barkham, R., Gudgin, G., Hart, M. and Hanvey, E. 1996:. THE DETERMINANTS OF SMALL FIRM GROWTH: AN INTER-REGIONAL STUDY IN THE UK 1986-90. London: Jessica Kingsley Publishers, £19.95 paper.

Krakow — transition to capitalism. Hardy, J. and Rainnie, A. 1996:. RESTRUCTURING KRAKOW: DESPERATELY SEEKING CAPITALISM. Mansel, £50.00 cased.     

The Performance of State–Owned Enterprises Revisited

Many theoretical and empirical studies look at the ownership–performance relationship. So far, the literature in finance and in accounting mainly refers to the property rights, agency and public choice theories. Despite the fact that the results of these studies are more or less conclusive, it is usually considered that the private enterprise performs better than the state–owned enterprise. In this article, we argue that these studies suffer from one major limitation. They do not recognize that the goals of the state–owned enterprise are different from the ones espoused by the private firm. Using a sample of state–owned entreprises and private firms for the period 1976–1996, we present empirical evidence that the state–owned enterprises, when their main goal is to maximize profit, perform as well as the privately owned enterprises. Therefore, the alleged under–performance of the state–owned enterprises may only be the result of pursuing other goals while the poor quality of public managers may be another urban myth.     

Financial-market effects of Federal Government budget deficits

Conclusion This paper finds that, for the 30-year period 1955–1984, the federal government budget deficit in the United States had a positive and significant effect on the longer-term nominal interest rate. This finding is at odds with most of the existing literature, which finds federal budget deficits to have no measurable impact upon interest rates in the United States [cf. Evans, 1985; 1987; Hoelscher, 1983; Makin, 1983; Motley, 1983; McMillin, 1986; and Mascaro and Meltzer, 1983]. The difference between the findings here and the findings in these other studies can be traced at least in part to the way in which we specify the deficit variable. That is, we distinguish between thestructural deficit, which approximates theexogenous component of the total deficit, and thecyclical deficit, which represents theendogenous component of the total deficit. By contrast, these other related studies measure the deficit in more aggregated ways, which combine the cyclical deficit with the structural deficit into one variable.     

EU enlargement and the evolution of European production networks

We analyse the regionalisation of the European Union's production networks and the impact of enlargement by looking at trends in trade in intermediate and final products in different types of technology between 1995 and 2007. We find that enlargement has coincided with quite major changes in the structure of trade within and beyond the EU. Overall we find that the new member states of the EU12 have become a more important source of goods both for the sub-region itself and for the EU15.We look at trends in different types of products and technologies. We find that there has been greater regional consolidation of production networks in lower tech goods, while in high tech goods global sources of inputs - especially ASEAN + 3 - are becoming more important. Overall the EU12 is becoming a more important source of both intermediate and final products, especially within its own sub-region. This evolution makes the companies in the sub-region very dependent on the EU home market.     

Exploring the effectiveness of cigarette warning labels: findings from the United States and United Kingdom arms of the International Tobacco Control (ITC) Four Country Survey

• This paper explores the effectiveness of cigarette warning labels across two countries, one (the UK) with new and stricter legislation where text based labels have been made more prominent and one (the USA) with less stringent regulation, where labels are less visible. Using longitudinal data from the two countries, the research seeks to investigate the impact of the different types of warning labels on the information processing by consumers. This paper assesses the effectiveness of warning labels in terms of: consumer attention, elaboration, contemplation on quitting and behavioural compliance. This study provides a comprehensive examination of these key factors in a fixed causal sequence. Structural equation modelling was used to test this model based on longitudinal panel survey data from the International Tobacco Control (ITC) Four Country Survey. Analysis of a sample of 901 US smokers and 1459 UK smokers yielded results in full support of all hypothesised relationships in the model proposed for both countries. Findings suggest that the new European Union policy of more prominent warning labels has a direct effect on influencing behavioural compliance by smokers.

Copyright © 2007 John Wiley & Sons, Ltd.