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Page 1: STOCHASTIC LEAGUE TABLES: COMMUNICATING COST-EFFECTIVENESS ... · STOCHASTIC LEAGUE TABLES: COMMUNICATING COST-EFFECTIVENESS ... intervention would be included in the optimal mix

HEALTH ECONOMICS

Health Econ. 10: 473–477 (2001)DOI: 10.1002/hec.614

HEALTH ECONOMICS LETTERS

STOCHASTIC LEAGUE TABLES:COMMUNICATING COST-EFFECTIVENESS

RESULTS TO DECISION-MAKERS

RAYMOND C.W. HUTUBESSYa,*, ROB M.P.M. BALTUSSENa, DAVID B. EVANSa,JAN J. BARENDREGTb AND CHRISTOPHER J.L. MURRAYa

a Global Programme on E�idence for Health Policy (GPE), World Health Organization (WHO),Gene�a, Switzerland

b Department of Public Health, Erasmus Uni�ersity, Rotterdam, Netherlands

SUMMARY

The presentation of the results of uncertainty analysis in cost-effectiveness analysis (CEA) in the literature has beenrelatively academic with little attention paid to the question of how decision-makers should interpret theinformation particularly when confidence intervals overlap. This question is especially relevant to sectoral CEAproviding information on the costs and effects of a wide range of interventions.

This paper introduces stochastic league tables to inform decision-makers about the probability that a specificintervention would be included in the optimal mix of interventions for various levels of resource availability, takinginto account the uncertainty surrounding costs and effectiveness. This information helps decision-makers decide onthe relative attractiveness of different intervention mixes, and also on the implications for trading gains inefficiency for gains in other goals such as reducing health inequalities and increasing health system responsiveness.Copyright © 2001 John Wiley & Sons, Ltd.

KEY WORDS — cost-effectiveness analysis; decision-making analysis; uncertainty analysis

INTRODUCTION

Uncertainty in cost-effectiveness analysis (CEA)has received a lot of attention in recent years,leading to the development of a range of ap-proaches, such as non-parametric bootstrapping[1], the construction of confidence planes [2],mathematical techniques [3], probabilistic sensitiv-ity analyses using Monte Carlo simulations [4],and the net health benefit approach [5]. Thesetechniques all present study results in terms ofsome type of uncertainty interval. However, littleor no attention is paid to the question of how

decision-makers should interpret the results whereuncertainty intervals overlap.

This absence of guidance to decision-makers isexacerbated in sectoral CEA based on the implicitor explicit use of cost-effectiveness league tables[6,7]. Sectoral analysis requires that interventionsare ranked on the basis of their cost-effectivenessratios. In deterministic analysis, decision-makersare assumed to work down the list, starting withthe most cost-effective, and to stop funding inter-ventions when the resources run out. The additionof uncertainty to this analysis is more realistic,but uncertainty intervals of many of the ratiosmay overlap and the decision-maker is left with

* Correspondence to: Global Programme on Evidence for Health Policy (GPE), World Health Organization (WHO), 20 AvenueAppia, CH-1211 Geneva, Switzerland. Tel.: +41 22 7913253; fax: +41 22 7914328; e-mail: [email protected]

Copyright © 2001 John Wiley & Sons, Ltd.Recei�ed 21 No�ember 2000

Accepted 6 February 2001

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R.C.W. HUTUBESSY ET AL.474

no guidance in the literature. It is simply assumedthat no decision about which intervention is moreefficient can be made. Yet decision-makers mustand do make decisions about which interventionsto encourage even when uncertainty is high (e.g.with overlapping confidence intervals).

We propose a new approach to presenting deci-sion-makers with the results of CEA includinguncertainty through the construction of a‘stochastic league table’. This informs decision-makers about the probability that a specific inter-vention would be included in the optimal mix ofinterventions for various levels or resourceavailability, taking into account the uncertaintysurrounding its total costs and effectiveness. Eachintervention should be thought of as a nationalprogramme or policy, which can only be pur-chased at one point [8]. Although the argument ispresented with reference to the generalizedmethod for sectoral CEA, which we recently pro-posed, allowing decision-makers to assess the effi-ciency of the current mix of interventions as wellas the relative attractiveness of changes to thismix should new resources become available [8], itis applicable to any form of sectoral analysis.

THE ANALYTICAL FRAMEWORK

The construction of stochastic league tables re-quires four steps (a software program,MCLeague, is being developed to carry out thisprocess). Firstly, using Monte Carlo simulations,

random draws are taken from estimated distribu-tions of total costs and effects for the interven-tions under study. These distributions are a prioridefined by the analyst and may take differentforms, for example normal, log-normal, and uni-form distributions [4]. Table 1 presents the hypo-thetical costs and effect data first presented inMurray et al. [8]. To reflect uncertainty, costs arehere assumed to be log-normally distributed, withan S.D. of 20, and effects are assumed to benormally distributed with an S.D. of 2. The co-variance is assumed to be zero. The conclusionsare not dependent on these assumptions. Randomdraws are taken from these distributions for allinterventions.

The second step is to determine the optimal mixof interventions for given levels of resourceavailability following the procedure for choosingbetween mutually exclusive and independent in-terventions outlined in Murray et al. [8]. The mostefficient intervention in the set of mutually exclu-sive interventions is evaluated according to itsaverage cost-effectiveness ratio (versus doingnothing), while the cost-effectiveness of others inthe mutually exclusive set are evaluated incremen-tal to the most efficient intervention.

Thirdly, this process is repeated a large numberof times (here 10000) to provide 10000 estimatesof the optimal mix of interventions. If P equalsthe number of times that an intervention is in-cluded in the optimal mix, P/10000 is the proba-bility that the intervention is included. Hence, P isthe proportion of samples from the estimated

Table 1. Costs, effects and cost-effectiveness of three independent sets of mutuallyexclusive alternatives

Interventions Total costs Total effects Cost-effectivenessa

S.D.MeanS.D.Mean

—2120120A120A2 25.4140 25.5

A3 —2320170190 20 7 2 33.3A4100B1 20 12 2 —120B2 20 17 2 7.1

10.022020150B350 20 22 2 2.3C1

C2 70 20 24.5 2 8.0120 11.122920C3170 20C4 31 2 25.0

a Cost-effectiveness ratios after exclusion of dominated interventions.

Copyright © 2001 John Wiley & Sons, Ltd. Health Econ. 10: 473–477 (2001)

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STOCHASTIC LEAGUE TABLES 475

distribution for which the intervention is esti-mated to be optimal based on the sample averageand incremental cost-effectiveness ratios. In ourexample, for resources equal to 50, C1 is included4323 times, a 43% probability of being included(Table 2). P for C2 equals 1406, a probability ofinclusion of 14%. In the remaining cases (43% ofall random draws), costs of each possible optionoverrun the available resources and no interven-tion can be funded fully. This explains why theprobabilities do not add up to 100%.

The fourth step involves repeating this proce-dure for various levels of resource availability toreveal the ‘resource expansion path’, showing theprobability that each intervention will be includedat different levels of resource availability (Table2). Decision-makers can use this information toprioritize interventions should more resources be-come available for health care. The probabilitythat a more expensive alternative will be includedincreases with the level of resource availability.For example, the probability C2 is included in-creases from 14% to 47% when resources increasefrom 50 to 100. In our example, no intervention isincluded in the optimal mix with certainty—evenat high levels of resource availability—because ofthe relative large standard deviations assumed forcosts and effects.

The degree of uncertainty in costs and effects ofan intervention can have a large impact on itsprobability of inclusion in the optimal mix. If wechange the standard deviation of the cost distribu-

tion for intervention A2 from 20 to 70, its proba-bility of inclusion at a level of resourceavailability of 300 increases from 5% to 22%(Table 3). This is because intervention costs noware sometimes very low thereby rendering theintervention relatively cost-effective (with re-sources �600, its probability of inclusion de-creases because it now has to compete with themore cost-effective interventions A3 and A4which can be afforded). The general conclusion isthat the higher the uncertainty in costs and ef-fects, the more equal the probabilities of inclusionof interventions will be, other things equal. This istrue both within the same mutual exclusive set aswell as between independent sets of interventions.

In Table 2, the numbers in bold represent inter-ventions that would be selected in a traditionalleague table based on the cost-effectiveness ratioscalculated in Table 1. These interventions wouldalso be chosen by the stochastic league tablebecause of their higher probabilities of inclusion.However, the stochastic league table provides ad-ditional information to the decision-maker. Withresources of 200, a traditional league table wouldchoose intervention C2 whereas our stochasticleague table shows almost identical probabilitiesof inclusion of C1 and C2 in the optimal mix ofinterventions. This information provides decision-makers with more information than simply pre-senting the confidence intervals for all CERs. Forexample, it allows decision-makers to better eval-uate the impact of trading off the efficiency goal

Table 2. Stochastic league table presenting the probability of inclusion (%) of threeindependent sets of mutual exclusive interventions in the optimal mix of interventionsat different levels of resource availabilitya

Resource availabilityIntervention

50 100 150 200 300 400 600 800

A1 0 000 21000A2 2928255110

40 0 0 0 0 1 4A3670 0A4 0 0 0 11 67

00 3 14 11 2 0 0B115152126351810B2

85787236 85400B30C1 43 40 30 36 8 3 0

C2 14 47 25 37 17 10 0 0130 2324334516C3 38

0 0 8 11 31 54 76C4 76

a Numbers in bold represent interventions that would be listed in a traditional league table.

Copyright © 2001 John Wiley & Sons, Ltd. Health Econ. 10: 473–477 (2001)

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R.C.W. HUTUBESSY ET AL.476

Table 3. As Table 2, with standard deviation for costs of intervention A2 increasedfrom 20 to 70a

Intervention Resource availability

50 100 150 200 300 400 600 800

A1 0 0 0 0 1 2 1 1A2 7 9 14 12 22 37 28 28A3 0 0 0 0 0 1 5 4A4 0 0 0 0 1 9 67 67B1 0 3 15 10 1 1 0 0B2 0 1 19 35 28 18 15 14B3 0 0 3 37 71 81 85 86C1 43 40 30 39 11 3 0 0C2 15 47 30 33 17 7 1 1C3 0 13 34 17 45 30 23 24C4 0 0 6 11 28 60 77 75

a Numbers in bold represent interventions that would be listed in a traditional league table.

against other objectives such as reducing healthinequalities in their selection of interventions [9].In general, the more interventions (belonging tothe same mutually exclusive set) differ regardingtheir probabilities of inclusion in the optimal mix,the more efficiency decision-makers give up ifthey choose to over-ride the results in favour ofother goals in their choice of interventions— thestochastic league table in our example informsdecision-makers that they are not likely to losemuch in terms of efficiency if they decide to selectC1 rather than C2 for equity reasons. This impor-tant information is not revealed in deterministicleague tables or in the traditional approach touncertainty in CEA.

Another advantage concerns the informationprovided in the expansion path, illustrated inTable 2. With resources of 200, there is little tochoose between B2 and B3 but preference wouldbe given to B2. However, if the decision-makerfelt that additional resources would become avail-able in the near future, and that the costs ofswitching from B2 to B3 might be substantial, itwould be sensible for them to choose B3. Again,this type of information is not provided in thestandard approach to uncertainty.

Stochastic league tables may also show thatinterventions that would otherwise have beenruled out by dominance in traditional league ta-bles might well be included in some draws. In ourexample, intervention B1 will never be eligible forselection in a deterministic league table because it

is (weakly) dominated by B2. However, takinginto account uncertainty the stochastic leaguetable (Table 2) shows that B1 has a low butnon-zero probability of being included in the opti-mal mix. Whether decision-makers will actuallyselect such interventions depends on the probabil-ity of inclusion compared to other mutually exclu-sive alternatives, and the trade-off betweenefficiency and other objectives of health systems.

Figure 1 depicts an alternative way of visualiz-ing the information of Table 2. The vertical axisshows the probability of being chosen at the levelof resource availability on the horizontal axis. Thelogic is the same as that described for the interpre-tation of the tables.

Figure 1. Probability of inclusion (%) of three independentsets of mutually exclusive interventions

Copyright © 2001 John Wiley & Sons, Ltd. Health Econ. 10: 473–477 (2001)

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STOCHASTIC LEAGUE TABLES 477

DISCUSSION

The stochastic league table developed in this pa-per is a new way of presenting uncertainty aroundcosts and effects to decision-makers. It providesadditional information beyond that offered by thetraditional treatment of uncertainty in CEA, pre-senting the probability that each intervention isincluded in the optimal mix for given levels ofresource availability. The most likely optimal mixwill be the one that contributes the most to maxi-mizing population health for that level of re-sources. Decision-makers can then decide theextent to which they should trade-off gains inefficiency for gains in other goals of the healthsystem.

Stochastic league tables are conceptually differ-ent from the recently suggested portfolio ap-proach, borrowed from financial economics andcharacterizing health care resources allocation asa risky investment problem [10]. This approachprovides the optimal intervention mix given deci-sion-makers’ explicit preferences concerning riskand return. Our stochastic league table providesthe probability of an intervention being chosen inthe optimal mix, given uncertainty. Risk-neutraldecision-makers would choose the most likelycombination of interventions.

A drawback to our framework (and to theportfolio approach for that matter) is that distri-butions of costs and effects are assumed to beindependent, e.g. no joint distributions are de-fined. Moreover, the definition of the distribu-tions is left to the analyst, who may have verylittle information about the actual distribution,but whose choice is likely to have a large effectson the results. It is technically possible to includecovariance between costs and outcomes in theanalysis, but this requires more information aboutcovariances than is usually available. Alterna-tively, where empirical data on patient’ costs andeffects are available, our framework could employthe technique of non-parametric bootstrapping inwhich samples are drawn with replacement fromthe original data. This approach has the advan-tage that is does not rely on parametric assump-

tions concerning the underlying distribution andthat covariances between costs and effects can beeasily incorporated [1]. The development ofstochastic league tables is an important step for-ward in the interpretation of uncertainty at thedecision-making level.

ACKNOWLEDGEMENTS

The views expressed are those of the authors and not necessar-ily those of the Organization they represent.

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Copyright © 2001 John Wiley & Sons, Ltd. Health Econ. 10: 473–477 (2001)