A functional approach for studying technological progress: Extension to energy technology

This paper extends a broad functional category approach for the study of technological capability progress recently developed and applied to information technology to a second key case—that of energy based technologies. The approach is applied to the same three functional operations—storage, transportation and transformation—that were used for information technology by first building a 100 plus year database for each of the three energy-based functional categories. In agreement with the results for information technology in the first paper, the energy technology results indicate that the functional approach offers a stable methodology for assessing longer time technological progress trends. Moreover, similar to what was found with information technology in the first study, the functional capability for energy technology shows continual—if not continuous—improvement that is best quantitatively described as exponential with respect to time. The absence of capability discontinuities—even with large technology displacement—and the lack of clear saturation effects are found with energy as it was with information. However, some key differences between energy and information technology are seen and these include:

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Lower rates of progress for energy technology over the entire period: 19-37% annually for Information Technology and 3-13% for Energy Technology.

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Substantial variability of progress rates is found within given functional categories for energy compared to relatively small variation within any one category for information technology. The strongest variation is found among capability progress among different energy types.

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More challenging data recovery and metric definition for energy as compared to information technology.

These findings are interpreted in terms of fundamental differences between energy and information including the losses and efficiency constraints on energy. We apply Whitney's insight that these fundamental differences lead to naturally modular information technology artifacts. The higher progress rates of information-based as opposed to energy-based technologies follows since decomposable systems can progress more rapidly due to the greater ease of independent as opposed to simultaneous development. In addition, the broad implications of our findings to studies of the relationships between technical and social change are briefly discussed.     

How many singularities are near and how will they disrupt human history?

This paper reviews a large number of approaches that have been used for considering technologically driven profound societal change. We agree with Vinge's suggestion for naming events that are “capable of rupturing the fabric of human history” (or leading to profound societal changes) as a “singularity”. This is a useful terminology especially since a mathematically rigorous singularity seems impossible for technological and related societal change. The overview of previous work is done within the context of a broader look at the role of technological change within human history. The review shows that a wide variety of methods have been used and almost all point to singularities in the present century particularly in the middle of the century. The diversity of the methods is reassuring about the potential robustness of these predictions. However, the subjectivity of labeling events as singularities (even well studied past events) is a concern about all of the methods and thus one must carefully pause when relying in any way on these predictions. The general lack of empirical research in this area is also a concern.Quantitative considerations (by proponents and opponents) about past singularities or future singularities often confound two types of metrics. The first type is essentially related to diffusion of technologies (or bundles of technologies) where the logistic curve is empirically well established as the proper time dependence. The second type of metric is for technological capability where hyper-exponentials are empirically well established for their time dependence. In this paper, we consider two past singularities (arguably with important enough social change to qualify) in which the basic metric is alternatively of one type or another. The globalization occurring under Portuguese leadership of maritime empire building and naval technological progress is characterized by a metric describing diffusion. The revolution in time keeping, on the other hand, is characterized by a technological capability metric. For these two cases (and thus robust to the choice of metric type), we find that:

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  People undergoing profound technologically-driven societal change do not sense a singularity.

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  The societal impacts depend in complicated ways on human needs, institutional variables and other more uncertain factors and thus are particularly hard to project;

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  The societal impact is apparently not determined by the rate of progress on either type of metric or by projections to mathematical points with either kind of metric. This finding supports the existing concept that social change due to technology is a more holistic phenomenon than can be characterized by any technical metric.

In the final section, we use these empirical findings as the basis for exploring the possibilities for and nature of future singularities. In this we speculate that the potential for a future strong singularity based upon computational capability does not appear particularly probable but that one may already be occurring and is not fully noticed by those (us) going through it. Other possible 21st century singularities (life extension and fossil fuel elimination are two examples considered) may also be already underway rather than waiting for the predicted mid-century changes.     

‘Chariots of fire’: the evolution of tank technology, 1915–1945

We reconsider the notion of technological trajectories by means of a detailed case-study of the evolution of tank technology between 1915 and 1945. We use principal component analysis to analyze the distribution of technical characteristics and how they map into specific service characteristics. We find that, despite the existence of differences in technical leadership, tank designs of different countries show a high degree of overlap and closeness along a common technological trajectory. In the conclusions, we speculate on whether this pattern can be explained by common heuristics that influenced the rate and direction of design activities or by doctrinal viewpoints influencing the development and use of tanks in the battlefield.

Localized innovation, localized diffusion and the environment: an analysis of reductions of CO2 emissions by passenger cars

We investigate technological change with regard to CO₂ emissions by passenger cars, using a Free Disposal Hull methodology to estimate technological frontiers. We have a sample of cars available in the UK market in the period 2000–2007. Our results show that the rates of technological change (frontier movement) and diffusion (distance to frontier at the car brand level) differ substantially between segments of the car market. We conclude that successful policies should be aimed at the diffusion of best-practice technology, and take account of the different potential for further progress between different segments of the market (e.g. diesel vs. gasoline engines and small vs. large engines).

Fully endogenous mechanism selection on finite outcome sets

Summary This paper defines a choice process over social outcomes in which agents choose the institutional rules ormechanisms themselves without outside interference. Truly endogenizing the mechanism selection process in this way, however, involves facing an infinite regress problem in which outcomes are chosen by games which are themselves chosen by games, ad infinitum. This paper allows the possibility of such an infinite regress which we callfully endogenous mechanism selection.We introduce the notion ofFree Choice which restricts the class of mechanisms in the regress to those which prevent agents from being locked in to an equilibrium outcome by the actions of others. Under this condition, the infinite regress is shown to get truncated with the number of selection iterations endogenously determined. It turns out that the outcomes resulting from a Free Choice-constrained regress are (Weakly) Pareto optimal; in particular, these outcomes solve a weighted Rawlsian Maxmin criterion. We also show that these outcomes are invariant to the equilibrium concept used to evaluate games in the regress.This paper is based on the author's dissertation from the University of Minnesota (November, 1989).I am very grateful for the guidance, advice, and encouragement from my advisor, Marcel K. Richter, and for the many helpful suggestions from David Levine. I have also benefited from conversations with Nabil Al-Najjar, Gerhard Glomm, Leonid Hurwicz, James Jordan, Ramon Marimon, Andrew McClennan, Ariel Rubinstein, and William Thomson.     

Constructing Clinical Use: An Activity-Theoretical Perspective on Implementing New Technology

Abstract

The paper presents recent insights from the ongoing FORLEARN project, which aims to develop Foresight theory and practise by supporting the sharing of experience (‘mutual learning’) in Europe. Six functions of Foresight for policy-making are elaborated on:

• (1)?Informing policy: generating insights regarding the dynamics of change, future challenges and options, along with new ideas, and transmitting them to policymakers as an input to policy conceptualisation and design.

• (2)?Facilitating policy implementation: enhancing the capacity for change within a given policy field by building a common awareness of the current situation and future challenges, as well as new networks and visions among stakeholders.

• (3)?Embedding participation in policy-making: facilitating the participation of civil society in the policy-making process, thereby improving its transparency and legitimacy.

• (4)?Supporting policy definition: jointly translating outcomes from the collective process into specific options for policy definition and implementation.

• (5)?Reconfiguring the policy system: in a way that makes it more apt to address long-term challenges.

• (6)?Symbolic function: indicating to the public that policy is based on rational information.

The relationship between these functions and the tensions that can arise when a Foresight exercise attempts to address more than one function are discussed. Possible approaches for Foresight practice to better achieve the targeted impact on policy-making are outlined and emerging guidelines for improving Foresight practice are presented.     

Evolving provider payment models and patient access to innovative medical technology

Objective:

To investigate the evolving use and expected impact of pay-for-performance (P4P) and risk-based provider reimbursement on patient access to innovative medical technology.

Methods:

Structured interviews with leading private payers representing over 110 million commercially-insured lives exploring current and planned use of P4P provider payment models, evidence requirements for technology assessment and new technology coverage, and the evolving relationship between the two topics.

Results:

Respondents reported rapid increases in the use of P4P and risk-sharing programs, with roughly half of commercial lives affected 3 years ago, just under two-thirds today, and an expected three-quarters in 3 years. All reported well-established systems for evaluating new technology coverage. Five of nine reported becoming more selective in the past 3 years in approving new technologies; four anticipated that in the next 3 years there will be a higher evidence requirement for new technology access. Similarly, four expected it will become more difficult for clinically appropriate but costly technologies to gain coverage. All reported planning to rely more on these types of provider payment incentives to control costs, but didn’t see them as a substitute for payer technology reviews and coverage limitations; they each have a role to play.

Limitations:

Interviews limited to nine leading payers with models in place; self-reported data.

Conclusion:

Likely implications include a more uncertain payment environment for providers, and indirectly for innovative medical technology and future investment, greater reliance on quality and financial metrics, and increased evidence requirements for favorable coverage and utilization decisions. Increasing provider financial risk may challenge the traditional technology adoption paradigm, where payers assumed a ‘gatekeeping’ role and providers a countervailing patient advocacy role with regard to access to new technology. Increased provider financial risk may result in an additional hurdle to the adoption of new technology, rather than substitution of provider- for payer-based gatekeeping.     

Budget impact analysis of a novel gene expression assay for the diagnosis of malignant melanoma

Background:

Traditional pathology techniques alone can be insufficient to reliably distinguish between malignant melanoma, dysplastic nevi, and benign nevi in biopsies of suspicious pigmented lesions. Numerous studies have shown high rates of ambiguity when assessing such samples. A novel gene expression assay has been developed to objectively differentiate malignant melanoma from benign nevi.

Objective:

The purpose of this study was to quantify the economic impact of the gene expression assay on a US commercial health plan.

Methods:

The clinical paradigm of care was modeled for a hypothetical cohort of patients with suspicious pigmented lesions that are difficult-to-diagnose. Costs were assigned to each unit of care provided based on 2013 Medicare fee-for-service rates. Patients were followed for 10 years and were modeled to progress according to the natural history of their disease. The total cost of care was calculated for two scenarios: a Reference Scenario, representing current clinical practice, and a Test Scenario, in which each lesion was tested with the gene expression assay and diagnosed. Total cost of care was compared between the two scenarios to determine overall budget impact. Sensitivity analyses were performed to test the robustness of the model.

Results:

The gene expression assay reduces costs by $1268 per patient tested over 10 years, a decrease of 8.3%, after accounting for the cost of the assay. For a health plan with 10 million members, this would translate to over $8 million in savings. The largest portion of this saving comes from reducing the number of missed melanomas, which would otherwise progress to advanced disease. In sensitivity analyses, no single model input changed within a reasonable range of values caused the model to show that the assay was not cost-saving.

Conclusion:

In addition to improving the diagnosis of melanoma, this gene expression assay would likely reduce costs for health plans that choose to cover it.     

Dynamic interdependence of sovereign credit default swaps in BRICS and MIST countries

This article examines the interactions of emerging markets sovereign credit default swaps (CDS). Using a generalized vector autoregressive framework and principal component analysis, we find significant spillover effects within the two groups of emerging markets under study. Using the principal component analysis, we show that global financial market factors are important drivers of BRICS and MIST sovereign CDS spreads variability. Focusing on the forecast error variance decomposition, most of the spillover effects are documented among the emerging markets CDS. Brazil and Mexico contribute the largest net directional spillovers to the other emerging markets studied.

Highlights:

• There exist significant CDS spillover effects for MIST and BRICS countries.

• Mexico dominates the spillover effects within the MIST group while Brazil dominates the spillover effects within the BRICS group.

• As determined by principal component analysis, global financial market factors are important drivers of BRICS and MIST sovereign CDS spreads variability.

• There exists a relatively small net directional spillover from global financial market factors to the countries under study; however, the total spillover is time-varying.

• A large proportion of the forecast error variance in the markets studied comes from spillovers.

     

Technological and geographical proximity effects on knowledge spillovers: evidence from the US patent citations

The purpose of this paper is to investigate the pattern of knowledge flows as indicated by the patent citations in three areas: USA, Japan and Europe. In each economic area, we use information from United States Patent and Trademarks Office data to assess empirically the impact of the technological and geographical proximities for 530 international firms. In particular, the contribution to the existing literature is twofold: first, we use an international sample in such a way that we may compare the empirical results among different economic markets; second, we extend the analysis of the determinants of knowledge spillovers, taking into account the time dimension of the effects of the proximities. In order to compute the technological proximity, we follow the methodology developed by A.B. Jaffe (1986 Jaffe, A. B. 1986. Technological opportunity and spillovers of R&D: Evidence from firms’ patents, profits and market value. American Economic Review, 76(5): 984–1001. [Web of Science ®] , [Google Scholar]. Technological opportunity and spillovers of R&D: Evidence from firms’ patents, profits and market value. American Economic Review 76, no. 5: 984–1001), where a technological vector is based on the distribution of patents of each firm across technology classes. As far as the geographic proximity is concerned, we use the latitude and the longitude coordinates of the city in which each firm is situated to obtain the distance, in miles, between the firms. The empirical results, in line with results from previous studies, indicate that there is a statistically significant relationship between the knowledge flows, proxied by the patent citations, and the proximities, but the effects are rather differentiated according to the proximity type.     

技术进步的“体现观”与技术收敛测度指标构建

技术进步在经济增长与区域经济协调发展中的动力作用一直是人们关注的话题。区域间技术收敛是实现区域间经济收敛的必要条件。文章从经济增长理论中技术进步的分类出发,剖析了外生与内生、体现式与非体现式、资本偏向和劳动偏向等概念之间的关系,进一步给出了技术收敛的测度指标体系。文章认为,当前关于技术内生和外生的研究并没有与技术进步的"体现观"结合起来。新古典增长理论中所指的技术进步主要是非体现式的技术进步。内生增长理论中的体现式技术进步主要是体现在资本或劳动力中的技术进步。所以,技术收敛的分析应该在经济收敛分析方法的基础上,充分考虑体现在资本和劳动力中的技术和没有体现在要素中的技术,建立相应的测度指标体系。这种测度技术收敛的方法与一般性的技术收敛测度相比具有明显的优点,对相关的产业技术政策、人力资本流动政策以及产业转移等政策的制定具有重要的指导意义。     

Empirical Regularities of Financial Market Volatility and Good Modelling Process: Developing Countries’ Exchange Rate Markets Perspective

A survey of contemporary literature suggests that empirical studies on developing economies are few or almost non-existent. Engle and Patton (2001, What good is a volatility model. Quantitative Finance, 1, 237–245) as well as Poon (2005, A Practical Guide to Forecasting Financial Market Volatility. New Jersey: Wiley.) suggest that a good volatility model is one that utilizes the empirical regularities of financial market volatility (of which most were observed on industrialized economies markets). This paper uses exchange rate series from Ghana, Mozambique and Tanzania to show that;

1. they are not different from other financial markets as they exhibit most of the empirical regularities including volatility sign asymmetry, non-normal distribution and volatility clustering. It is however observed that the three exchange rate series are very volatile, with induced volatile shocks highly persistent and asymmetric, and extreme prices commonplace;

2. the ARCH technique (which has been well documented to capture these empirical regularities and produce good forecasts) generally produced a good fit to the three exchange rate series when compared with volatility forecasts generated using the EWMA technique. In the simple analysis of a day-ahead volatility forecast abilities of estimated models, it was observed that best fit does not necessarily ensure best forecast.

     

Knowledge growth, complexity and the returns to R&D

The introduction of a production function of technology embodying laws of returns to research and development (R&D) is now standard practice in growth theory. This paper offers a critical evaluation, in the light of a generalized N–K model, of some recent contributions suggesting foundations for the existence of laws of returns to R&D. It is argued that such contributions fail to analyze the way in which research and development activity in the technological and scientific domains affect the dimension, the hierarchic structure and the complexity of knowledge search spaces. In the attempt at moving some analytical steps in this direction, this paper considers the possibility that modularity effectively counters the rise in complexity which would follow from idea growth and the increasing number of potential interactions between component ideas. It is argued that the force of the modularity argument finds its limits in the face of radical innovations that are general purpose, but entail a deconstruction and reconstruction of the hierarchy of technological interactions. It is also suggested that niche creation and knowledge spillovers elicit the early development and subsequent diffusion of such radical innovations.

Bendamustine-rituximab: a cost-utility analysis in first-line treatment of indolent non-Hodgkin’s lymphoma in England and Wales

Objective:

To evaluate the cost-effectiveness of bendamustine-rituximab (B-R) compared with CHOP-R (cyclophosphamide, doxorubicin, vincristine, prednisone, rituximab) and CVP-R (cyclophosphamide, vincristine, prednisone, rituximab) as first-line treatment for patients with advanced indolent non-Hodgkin’s lymphoma (NHL).

Methods:

A patient-level simulation was adapted from the model used by the University of Sheffield School of Health and Related Research (ScHARR) in a health technology appraisal of rituximab for first-line treatment of follicular lymphoma. This approach allowed modelling of the complex treatment pathways in indolent NHL. Data from a Phase 3 randomized, open-label trial were used to compare B-R with CHOP-R. The relative efficacy of CHOP-R and CVP-R was estimated using an indirect treatment comparison similar to the original ScHARR approach. The analysis was conducted from the perspective of the National Health Service in England and Wales, using a lifetime time horizon. A number of one-way sensitivity and scenario analyses were conducted, including one using recently published data comparing CVP-R with CHOP-R.

Results:

The deterministic incremental cost-effectiveness ratio (ICER) was £5249 per quality adjusted life year (QALY) for B-R vs CHOP-R, and £8092 per QALY for B-R vs CVP-R. The alternative scenario using direct data comparing CVP-R with CHOP-R approximately halved the ICER for B-R vs CVP-R to £4733. Owing to its better toxicity profile, B-R reduced the cost of treating adverse events by over £1000 per patient vs CHOP-R.

Limitations:

The main limitations were: immaturity of overall survival data from the Phase 3 trial; reliance on quality-of-life data from previous health technology appraisals (as this was not collected in the trial); and a lack of direct evidence or a network of connected evidence comparing B-R with CVP-R.

Conclusions:

The ICERs for B-R vs CHOP-R and CVP-R were considerably below the thresholds normally regarded as cost-effective in England and Wales (£20,000–30,000 per QALY).     

Choosing propensity score matching over regression adjustment for causal inference: when,why and how it makes sense

Summary

This study identified when regression adjustment fails to adjust adequately for differences in observed covariates and where propensity score matching is the only alternative.

Multivariate analysis might fail to adjust for observed confounders if:

• 1. The means of the propensity scores in the two groups are more than one-half a standard deviation apart unless distributions of the covariates in both groups are nearly symmetric, sample sizes of the two groups are approximately the same and distributions of the covariates in the two groups have similar variances;

• 2. The ratio of the propensity score variances in the two groups is significantly different from one;

• 3. The ratio of residual variances in the two groups after adjusting for the propensity score is significantly different from one.

Conducted retrospective analysis showed that the treatment effect would be an estimated $305 (or 26%) less if the misspecified outcome model had been chosen.     

Habit formation in an endogenous growth model with pollution abatement activities

This paper studies how habit formation in consumption affects pollution abatement activities and the steady-state growth rate, in the context of an endogenous growth model in which agents derive disutility from the habit stock and pollution. The paper also examines how the effect of technological change in abatement on the optimal growth rate is enhanced or weakened by habit formation. We show that if agents persist in and care about their habits, sustained growth is only possible with rapid technological progress.      

Malmquist productivity index decompositions: a unifying framework

In two widely cited but unpublished working papers, Simar and Wilson (1998 Shephard, R. 1970. Theory of Cost and Production Functions, New Jersey: Princeton University Press.  [Google Scholar]) and Zofío and Lovell (1998 Wheelock, D and Wilson, PW. 1999. Technical progress, inefficiency, and productivity change in U.S. banking, 1984–1993. Journal of Money, Credit and Banking, 31: 212–34. [Crossref], [Web of Science ®] , [Google Scholar]) proposed an alternative decomposition of the Malmquist Productivity Index, which retained what seemed to be the strongholds of previous proposals with regard to the contribution of technological and efficiency change to productivity change. Namely, a technical change term with regard to the best practice variable returns to scale (VRS) technology, which is to be found in Ray and Desli (1997 Ray, S. Sept. 2001. “On an extended decomposition of the malmquist productivity index”. In Paper Presented at the Seventh European Workshop on Efficiency and Productivity, Sept., 25–29. Oviedo: Universidad de Oviedo.  [Google Scholar]) and a scale efficiency change term that illustrates a firm's situation with regard to optimal scale (benchmark technology), Färe et al . (1994 Färe, R, Grosskopf, S and Norris, M. 1997. Productivity growth, technical progress, and efficiency change in industrialized countries: reply. American Economic Review, 87: 1040–43. [Web of Science ®] , [Google Scholar]). Attaining this objective required the introduction of an additional term in the Malmquist Productivity Index decomposition, which would reflect the scale bias of technical change. It is our objective to provide economic rationale for this term within a theory of production context, the existing decompositions and recent articles that further elaborate on this issue. The ideas are illustrated using productivity trends in 17 OECD countries.     

Economic evaluation of tocilizumab combination in the treatment of moderate-to-severe rheumatoid arthritis in Italy

Abstract

Objective:

This study was designed to evaluate the cost utility of tocilizumab in rheumatoid arthritis (RA) patients, with inadequate responses to traditional disease-modifying anti-rheumatic drugs (tDMARDs) from a payer’s perspective in Italy.

Methods:

An individual patient simulation model was used to project lifetime medical costs (payer’s perspective) and quality-adjusted life-years (QALYs). Treatment sequences starting with tocilizumab or the most commonly prescribed biologics (etanercept, adalimumab, or infliximab) were compared. The addition of tocilizumab to standard of care, without the replacement of anti-tumor necrosis factor (TNF)-α treatments, was also evaluated. Patient characteristics, treatment efficacy, and quality-of-life data were based on three phase 3 tocilizumab clinical trials (TOcilizumab Pivotal Trial in Methotrexate Inadequate respONders [OPTION], Tocilizumab in cOmbination With traditional DMARD therapy [TOWARD], and TociLIzumab Safety and THE Prevention of Structural Joint Damage [LITHE]). Mixed-treatment comparison was used to estimate response probabilities. Resource utilization, treatment acquisition, administration, and monitoring costs were estimated using Italian secondary sources. Uncertainty in model parameters was evaluated by probabilistic sensitivity analysis.

Results:

Replacement of anti-TNF-α treatments with tocilizumab reduced total costs over a patient’s lifetime (base-case analysis: tocilizumab sequence, €141,100 vs standard of care sequence, €143,500). Patients receiving tocilizumab realized more QALYs than patients receiving standard of care (9.8881 vs 9.3502 QALYs). Therefore, according to the base-case analysis, the tocilizumab sequence dominated the standard of care. In a sensitivity analysis, the model base-case result was robust to input changes. When tocilizumab was added to standard of care, without replacing anti-TNF-α treatments, the incremental cost-effectiveness ratio was €17,100 per QALY.

Conclusion:

The analysis demonstrates that, in Italy, replacing another biologic DMARD with tocilizumab or adding tocilizumab to the current standard of care is a cost-effective strategy in the treatment of RA patients with inadequate responses to tDMARDs.     

Is transcatheter aortic valve implantation (TAVI) a cost-effective treatment in patients who are ineligible for surgical aortic valve replacement? A systematic review of economic evaluations

Objectives:

Health Technology Assessment (HTA) agencies often undertake a review of economic evaluations of an intervention during an appraisal in order to identify published estimates of cost-effectiveness, to elicit comparisons with the results of their own model, and to support local reimbursement decision-making. The aim of this research is to determine whether Transcatheter Aortic Valve Implantation (TAVI) compared to medical management (MM) is cost-effective in patients ineligible for surgical aortic valve replacement (SAVR), across different jurisdictions and country-specific evaluations.

Methods:

A systematic review of the literature from 2007–2012 was performed in the MEDLINE, MEDLINE in-process, EMBASE, and UK NHS EED databases according to standard methods, supplemented by a search of published HTA models. All identified publications were reviewed independently by two health economists. The British Medical Journal (BMJ) 35-point checklist for economic evaluations was used to assess study reporting. To compare results, incremental cost effectiveness ratios (ICERs) were converted to 2012 dollars using purchasing power parity (PPP) techniques.

Results:

Six studies were identified representing five reimbursement jurisdictions (England/Wales, Scotland, the US, Canada, and Belgium) and different modeling techniques. The identified economic evaluations represent different willingness-to-pay thresholds, discount rates, medical costs, and healthcare systems. In addition, the model structures, time horizons, and cycle lengths varied. When adjusting for differences in currencies, the ICERs ranged from $27K–$65K per QALY gained.

Conclusions:

Despite notable differences in modeling approach, under the thresholds defined by using either the local threshold value or that recommended by the World Health Organization (WHO) threshold value, each study showed that TAVI was likely to be a cost-effective intervention for patients ineligible for SAVR.     

Targeted erlotinib for first-line treatment of advanced non-small cell lung cancer: a budget impact analysis

Objectives:

A recent phase III trial showed that patients with advanced non-small cell lung cancer (NSCLC) whose tumors harbor specific EGFR mutations significantly benefit from first-line treatment with erlotinib compared to chemotherapy. This study sought to estimate the budget impact if coverage for EGFR testing and erlotinib as first-line therapy were provided in a hypothetical 500,000-member managed care plan.

Methods:

The budget impact model was developed from a US health plan perspective to evaluate administration of the EGFR test and treatment with erlotinib for EGFR-positive patients, compared to non-targeted treatment with chemotherapy. The eligible patient population was estimated from age-stratified SEER incidence data. Clinical data were derived from key randomized controlled trials. Costs related to drug, administration, and adverse events were included. Sensitivity analyses were conducted to assess uncertainty.

Results:

In a plan of 500,000 members, it was estimated there would be 91 newly diagnosed advanced NSCLC patients annually; 11 are expected to be EGFR-positive. Based on the testing and treatment assumptions, it was estimated that 3 patients in Scenario 1 and 6 patients in Scenario 2 receive erlotinib. Overall health plan expenditures would increase by $0.013 per member per month (PMPM). This increase is largely attributable to erlotinib drug costs, in part due to lengthened progression-free survival and treatment periods experienced in erlotinib-treated patients. EGFR testing contributes slightly, whereas adverse event costs mitigate the budget impact. The budget impact did not exceed $0.019 PMPM in sensitivity analyses.

Conclusions:

Coverage for targeted first-line erlotinib therapy in NSCLC likely results in a small budget impact for US health plans. The estimated impact may vary by plan, or if second-line or maintenance therapy, dose changes/interruptions, or impact on patients’ quality-of-life were included.