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Available online at www.sciencedirect.com journal homepage: www.elsevier.com/locate/jval Using Multicriteria Approaches to Assess the Value of Health Care Charles E. Phelps, PhD 1, *, Guruprasad Madhavan, PhD 2 1 University of Rochester, Rochester, NY, USA; 2 The National Academies of Sciences, Engineering and Medicine, Washington, DC, USA ABSTRACT Practitioners of cost-utility analysis know that their models omit several important factors that often affect real-world decisions about health care options. Furthermore, cost-utility analyses typically reect only single perspectives (e.g., individual, business, and societal), further limiting the value for those with different perspectives (patients, providers, payers, producers, and plannersthe 5Ps). We discuss how models based on multicriteria analyses, which look at problems from many perspectives, can ll this void. Each of the 5Ps can use multicriteria analyses in different ways to aid their decisions. Each perspective may lead to different value measures and outcomes, whereas no single-metric approach (such as cost-utility analysis) can satisfy all these stakeholders. All stakeholders have unique ways to measure value, even if assessing the same health intervention. We illustrate the benets of this approach by comparing the value of ve different hypothetical treatment choices for ve hypothetical patients with cancer, each with different preference structures. Nine attributes describe each treatment option. We add a brief discussion regarding the use of these approaches in group-based decisions. We urge that methods to value health interventions embrace the multicriteria approaches that we discuss, because these approaches 1) increase transparency about the decision process, 2) allow ight simulator- type evaluation of alternative interventions before actual investment or deployment, 3) help focus efforts to improve data in an efcient manner, 4) at least in some cases help facilitate decision convergence among stakeholders with differing perspectives, and 5) help avoid potential cognitive errors known to impair intuitive judgments. Keywords: multicriteria analysis, priority setting, systems analysis, value modeling. Copyright & 2017, International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. Introduction Standard approaches for evaluating health interventions use cost-utility analysis, most commonly with a societal perspective. All health-improving benets and costs enter the cost-utility model (similar to the methods of cost-benet analysis), which follows directly from a utility-maximizing framework [1,2]. Cost- effectiveness and cost-benet analyses are closely related once a critical cutoff value is established for resource allocation in the cost-utility framework [3]. But this perspective seldom corre- sponds to the perspectives of most of the participants in the health enterprise: patients, providers, payers, producers, and plannersthe 5Ps. Each of these 5Ps almost certainly will have multiple objectives when selecting, advising about, providing coverage for, investing in, or supporting research about health care options. Each of these decision makers has multiple goals, and therefore no single-metric approach (such as cost-benet or cost-utility ratios) can accommodate any of their viewpoints. We need better, comprehensive frameworks to assess the value of health that can both accommodate different values and incorpo- rate multiple attributes associated with health options. Any approach using only a single attribute and a single perspective is almost certainly inadequate and incomplete. Others have acknowledged this challenge. For example, the International Society for Pharmaceutical Outcomes Research (ISPOR) Task Force on multicriteria decision analysis issued two reports in 2016 summarizing best practices for such approaches [4,5]. These reportsin addition to providing a state-of-the-art reviewdiscuss the uses of the multicriteria models in health care, from supporting decisions of individual patients to a universal health care system. As their summaries show, use of multicriteria analysis in the United States has primarily focused on decision support for individual patients rather than on technology assessment or insurance coverage decisions (e.g., [6]). Among the 5Ps, Patients are likely to focus on potential therapeutic benets, risks, and side-effects, as well as travel time, net costs and out of pocket expense as they evaluate treatment options and other alternatives, including watchful waiting, palliation, treatment holidays, and continuous monitor- ing. Providers may consider their understanding of patient preferences, but may also consider the consequences for their own nancial situation (including differential reimbursement 1098-3015$36.00 see front matter Copyright & 2017, International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. http://dx.doi.org/10.1016/j.jval.2016.11.011 Note: The views expressed in this article are those of the authors and not necessarily of the National Academies of Sciences, Engineering, and Medicine. E-mail: [email protected]. * Address correspondence to: Charles E. Phelps, 30250 South Highway 1, Gualala, CA 95445. VALUE IN HEALTH 20 (2017) 251 255

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Page 1: Using Multicriteria Approaches to Assess the Value of ... · PDF fileUsing Multicriteria Approaches to Assess the Value of Health Care Charles E. Phelps, ... Practitioners of cost-utility

Avai lable onl ine at www.sc iencedirect .com

journal homepage: www.elsevier .com/ locate / jva l

Using Multicriteria Approaches to Assess the Valueof Health CareCharles E. Phelps, PhD1,*, Guruprasad Madhavan, PhD2

1University of Rochester, Rochester, NY, USA; 2The National Academies of Sciences, Engineering and Medicine, Washington, DC, USA

A B S T R A C T

Practitioners of cost-utility analysis know that their models omitseveral important factors that often affect real-world decisions abouthealth care options. Furthermore, cost-utility analyses typically reflectonly single perspectives (e.g., individual, business, and societal),further limiting the value for those with different perspectives(patients, providers, payers, producers, and planners—the 5Ps). Wediscuss how models based on multicriteria analyses, which look atproblems from many perspectives, can fill this void. Each of the 5Pscan use multicriteria analyses in different ways to aid their decisions.Each perspective may lead to different value measures and outcomes,whereas no single-metric approach (such as cost-utility analysis) cansatisfy all these stakeholders. All stakeholders have unique ways tomeasure value, even if assessing the same health intervention. Weillustrate the benefits of this approach by comparing the value of fivedifferent hypothetical treatment choices for five hypothetical patients

with cancer, each with different preference structures. Nine attributesdescribe each treatment option. We add a brief discussion regardingthe use of these approaches in group-based decisions. We urge thatmethods to value health interventions embrace the multicriteriaapproaches that we discuss, because these approaches 1) increasetransparency about the decision process, 2) allow flight simulator-type evaluation of alternative interventions before actual investmentor deployment, 3) help focus efforts to improve data in an efficientmanner, 4) at least in some cases help facilitate decision convergenceamong stakeholders with differing perspectives, and 5) help avoidpotential cognitive errors known to impair intuitive judgments.Keywords: multicriteria analysis, priority setting, systems analysis,value modeling.

Copyright & 2017, International Society for Pharmacoeconomics andOutcomes Research (ISPOR). Published by Elsevier Inc.

Introduction

Standard approaches for evaluating health interventions usecost-utility analysis, most commonly with a societal perspective.All health-improving benefits and costs enter the cost-utilitymodel (similar to the methods of cost-benefit analysis), whichfollows directly from a utility-maximizing framework [1,2]. Cost-effectiveness and cost-benefit analyses are closely related once acritical cutoff value is established for resource allocation in thecost-utility framework [3]. But this perspective seldom corre-sponds to the perspectives of most of the participants in thehealth enterprise: patients, providers, payers, producers, andplanners—the 5Ps. Each of these 5Ps almost certainly will havemultiple objectives when selecting, advising about, providingcoverage for, investing in, or supporting research about healthcare options. Each of these decision makers has multiple goals,and therefore no single-metric approach (such as cost-benefit orcost-utility ratios) can accommodate any of their viewpoints. Weneed better, comprehensive frameworks to assess the value ofhealth that can both accommodate different values and incorpo-rate multiple attributes associated with health options. Any

approach using only a single attribute and a single perspectiveis almost certainly inadequate and incomplete.

Others have acknowledged this challenge. For example, theInternational Society for Pharmaceutical Outcomes Research(ISPOR) Task Force on multicriteria decision analysis issued tworeports in 2016 summarizing best practices for such approaches[4,5]. These reports—in addition to providing a state-of-the-artreview—discuss the uses of the multicriteria models in healthcare, from supporting decisions of individual patients to auniversal health care system. As their summaries show, use ofmulticriteria analysis in the United States has primarily focusedon decision support for individual patients rather than ontechnology assessment or insurance coverage decisions (e.g., [6]).

Among the 5Ps, Patients are likely to focus on potentialtherapeutic benefits, risks, and side-effects, as well as traveltime, net costs and out of pocket expense as they evaluatetreatment options and other alternatives, including watchfulwaiting, palliation, treatment holidays, and continuous monitor-ing. Providers may consider their understanding of patientpreferences, but may also consider the consequences for theirown financial situation (including differential reimbursement

1098-3015$36.00 – see front matter Copyright & 2017, International Society for Pharmacoeconomics and Outcomes Research (ISPOR).

Published by Elsevier Inc.

http://dx.doi.org/10.1016/j.jval.2016.11.011

Note: The views expressed in this article are those of the authors and not necessarily of the National Academies of Sciences,Engineering, and Medicine.

E-mail: [email protected].* Address correspondence to: Charles E. Phelps, 30250 South Highway 1, Gualala, CA 95445.

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and use of provider-owned facilities), and perhaps litigation risks.Payers (third-party insurers) may seek to minimize premiumcosts by bargaining with providers, perhaps choosing limitedpanels of preferred providers, and choosing which therapies toinclude as covered and at what co-payment rates (e.g., tierswithin prescription drug plans). Producers (e.g., manufacturersof drugs, vaccines, and devices) may each have their owninterests, ranging from profitability to perceived public good.Planners may focus on prioritizing research investments andpossibly (for elected policymakers) even on enhanced re-electionchances. Again, no single perspective can possibly representthese diverse interests.

Why Use Multifactorial Systems Analysis?

Multifactorial approaches approximate as well as expand theeconomic concept of a utility function with a goal of incorporat-ing relevant parameters to make the models useful for decisionsupport [7–11]. Each approach makes trade-offs between general-izability and usability. They also seek to consider competingattributes of various options, making the trade-offs explicit andtransparent to others, thus excelling in comparison with single-attribute models such as cost-effectiveness analysis [12].

Multicriteria approaches may lead to two potentially conflict-ing situations. First, all users can create their own priority lists onthe basis of their own values and preferences. Second, a con-sequence of the first, we may see the creation of potentiallydisparate lists of priorities. This is not a defect of multicriteriamodels, but rather represents the reality of the world: peoplewith different perspectives have different priorities.

Multicriteria approaches provide a number of direct benefitsrelated to decision making. First comes transparency. These modelsilluminate the key values driving priorities and choices [13].

Second, this approach allows the assessment of the implica-tions of adopting a particular option (and multiple variationsthereof) before actually spending resources to implement thechoice, similar to a flight simulator in aviation engineering. Thisadvance testing capability uniquely builds on the multicriteriaevaluation processes of these models. In appropriate settings, itcan also allow reverse engineering to improve the specificationsor characteristics of diagnostic or treatment interventions(e.g., considering trade-offs between cost, discomfort, radiationexposure, and accuracy in diagnostic protocols).

Third, multicriteria models can help guide efforts to system-atically improve data. Often, at least some of the data enteringthe model are imperfect. Using sensitivity analysis within themodel can show which variables importantly contribute to thefinal rankings and which do not matter, allowing users to focustheir efforts on improving those data that matter most [14].

Fourth, multicriteria models can potentially assist competinginterests in reaching negotiated decision convergence. Prices inmarkets perform a similar function by providing information tobuyers and sellers about how other participants in the marketvalue things. Similarly, values specified in multicriteria modelsprovide information that can assist various parties in negotiatingagreements by showing what is more important and less impor-tant to various stakeholders. Clearly, in some settings, variousbargaining parties may not wish to reveal such information,but in some other settings, it can pave the way to a mutuallyagreeable negotiated agreement, just as market prices help tofacilitate exchanges.

Fifth, multicriteria approaches can help bypass at least somecognitive biases that are now well understood to adversely affectdecisions. These models ask humans to do tasks for which theyare best suited (specifying values) and then ask computers to dowhat they do best (numerical synthesis of information). These

cognitive biases include those relating to framing of issues,anchoring, time inconsistency, estimation of probabilities,making choices in the presence of uncertainty, overconfidence,and many others [15].

These five considerations—transparency, advance testing ofalternatives, efficiency in data improvement, supporting decisionconvergence, and alleviation of cognitive biases in real-worlddecision making—all point to the value of using multicriteriamodels. Most previous assessments of multicriteria models haveprimarily emphasized the value of transparency in the decisionprocess, although we believe that the other considerations listedhere create similar benefits.

Present Limitations of Multicriteria Approaches

A key limitation of multicriteria models is the lack of consensus onthe best method to balance costs and benefits because benefitmeasures in these models purposefully include a wider array ofattributes than do standard narrow measures such as quality-adjusted life-years (QALYs). A recent ISPOR Task Force reportdiscussed three ways to approach these issues using multicriteriaanalysis [5]. The first uses direct inclusion of cost as a “negative”attribute with its own weight. The second involves finding otherhealth care interventions (with known costs) that could be elimi-nated and assessing the multicriteria value of those as a bench-mark. The third approach divides the multicriteria scores of eachoption by its cost (akin to using the ratio of cost per health benefit).The task force found none of these approaches perfectly persuasive,and urge further research on the matter. A fourth available alter-native assumes an exogenous investment budget, without suggest-ing how to set its level [16]. We suggest a new alternative here.

Cost-benefit analysis recommends adopting every alternativechoice with positive net present value. This method, however,requires analysts to assign a specific value to human life, a taskmany resist. To solve this concern, cost-utility analysis calculatesthe incremental cost-effectiveness ratio (ICER), and the decisionmaker chooses the critical cutoff value for acceptable ICERs.

We suggest a similar approach for use in multicriteria models.Suppose that in any multicriteria model, health-related measures(such as QALYs) accounted for a share S of the total value, and allother attributes accounted for a share of 1 � S. Then, if the ICERcutoff was $100,000/QALY, the measured scores for each choice inthe multicriteria model would be evaluated against a cutoff of$100,000/S. This would provide a direct method, comparable withthat of cost-utility analysis when using an ICER cutoff to guideresource allocation in the broader framework of multicriteriadecision analysis.

Other concerns about multicriteria models are specific toparticular applications. For example, the weight-setting protocolin the analytic hierarchy process allows internal inconsistenciesand rank-reversal with different decision options [11]. The swingweight approach in the multi-attribute utility theory requiresanalysts to create standardized 0 to 100 scales for each attribute,which interact with the weights in potentially unforeseenways [9]. These and other technical issues can and should beresolved to bring multicriteria models to their greatest potential.

Choices and Scenarios

To illustrate key features of the multicriteria systems approach,consider a patient faced with choosing among available cancertherapies. Clinicians would specify the quantitatively measurabletherapeutic and side-effect profiles of each option. Patients wouldspecify the levels of any subjectively determined attribute asso-ciated with each choice, for example, distaste or fear of a

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particular therapeutic modality (e.g., radiation, chemotherapy, ormolecular targeting) independent of quantitatively known bene-fits and risks.

As a specific example, we show the choice of five hypotheticalcancer therapies for a set of hypothetical individuals using multi-attribute utility theory, a specific approach within multicriteriasystems analysis.

This approach requires the decision maker (patient) to specifyweights for the selected attributes. A simple method to obtaingood weights simply asks the patient to list the desired attributesin rank order. Then, a formula—the rank order centroid method—estimates the average of all weights (summing to 1.0) consis-tent with the selected rankings [9]. This approximation has beenshown to provide highly reliable results when used in linearmulti-attribute models [10]. Our example uses rank order listsfrom the hypothetical patients to rank the importance of nineattributes in cancer therapy, which then create correspondingweights. The multi-attribute model combines the attribute per-formance measures of each therapy with the patients’ weights tocreate value scores on a 0 to 100 scale.

Treatments

Multi-attribute utility models require defining boundaries of theworst and best outcomes that can be imagined for the choices.Thus, for example, we set the best case for probability ofremission at 0.5 (rather than 1.0), under the premise that for thisdisease, higher probabilities of remission cannot be imaginedwith existing treatment approaches.

Table 1A reports the values of each of these five hypotheticaltherapies (T1–T5) on each of their nine attributes, showing (in thecolumn headings) the feasible range and (where appropriate)which score is the best. In our example, all these attributes arequantified, whereas in other settings, some of the attributesmight be subjectively determined, as previously discussed.

T1 has the highest probability of remission, but lower overallsurvival if the regimen fails. It is supported by a moderate-sizedrandomized controlled trial (RCT), has mid-range costs to patientand society, and is quite likely to cause pain, hair loss, andconsiderable nausea.

T2 is similar to T1, with a lower remission rate but potentiallylonger overall survival. It has stronger RCT evidence with higherexpected months of remission-free survival. Costs are the same.It has less pain associated with it, but a higher chance of nausea.

T3 is offered in an RCT. Preliminary evidence says it has highsurvival benefits and life extension, but is available to the patientonly with 50% probability, and so the probabilities of remissionand life extension reflect the expected value of the treatment andthe control therapy in the RCT, and the side-effect profiles are asimilar mix of the experimental and control therapies. T3 has ahigher societal cost, but is free to the patient as part of the RCT.

T4 has a low remission rate, but the highest expectedremission-free survival. It is opposite of T1. It gives the mostassurance for some additional survival, but little promise ofremission. It has relatively low cost, and has strong RCT evidencesupporting its outcomes. It has a side-effect profile that isunfavorable relative to other options.

T5 is palliative care only. It offers no remission or life expect-ancy gains, but has no side effects and removes all painassociated either with therapy or with the disease itself. It costslittle, and the data supporting its outcomes are solid.

Patients

We also created five hypothetical patients with the same primarycancer who have different preference profiles and attribute ranks.The weights associated with these rankings come from the

Table

1–Tre

atm

entperform

ance

and

patientpre

fere

nce

sbase

don

multip

leattribute

s.

Treatmen

t/patient

Prob

ability

ofremission

(0–0.5)

Expec

tedmon

ths

ofremission

-free

survival

(0–36

)

Prob

ability

ofhairloss

(0–1.0)

Prob

ability

ofnau

sea

(0–1.0)

Pain

scale

(0–10

)Total

cost

($1K

–$1

00K)

Patien

tco

st($0–

$10K

)Adva

ncing

know

ledge

(0or

1)

Qualityof

eviden

ceab

out

ben

efits

(0–5)

A.Treatmen

tperform

ance

on

nineattribute

scales

T1

0.4

120.8

0.3

250

,000

5,00

00

4T2

0.2

240.2

0.8

850

,000

5,00

00

5T3

0.2

120.3

0.4

580

,000

01

2T4

0.1

360.8

0.7

320

,000

2,00

00

5T5

00

00

102,00

01,00

00

4B.Pa

tien

trankings

ofattributes(andas

sociated

weigh

ts)

A1(0.314

)6(0.061

)4(0.111

)5(0.083

)7(0.042

)9(0.012

)2(0.203

)8(0.026

)3(0.148

)B

9(0.012

)1(0.314

)4(0.111

)3(0.148

)2(0.203

)6(0.061

)5(0.083

)7(0.042

)3(0.026

)C

4(0.111

)2(0.203

)7(0.042

)6(0.061

)5(0.083

)3(0.148

)8(0.026

)1(0.314

)9(0.012

)D

1(0.314

)2(0.203

)3(0.148

)4(0.111

)5(0.083

)9(0.012

)8(0.026

)7(0.042

)6(0.061

)E

6(0.061

)1(0.314

)8(0.026

)5(0.083

)4(0.111

)2(0.203

)7(0.042

)3(0.148

)9(0.012

)

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previously discussed rank order centroid method [9,10]. Table 1Bsummarizes each of these hypothetical patient’s preferences,showing the rank order in which they place each of the nineattributes of the therapies and (in parentheses) the associatedrank order centroid weights. In short, our five patients have thefollowing value structures.

Patient A places the greatest value on the chance of aremission, and then out-of-pocket cost, and wants certaintyabout the quality of the evidence, followed by concerns abouthair loss and nausea, freedom from pain, advancement of knowl-edge, and last, total societal cost.

Patient B places the greatest value on expected months ofremission-free survival, but has little concern about the prob-ability of remission. This patient has heightened concernsabout treatment side effects, modest concern for out-of-pocket costs and total costs, and little concern about advancingknowledge or the quality of data supporting the doctors’recommendations.

Patient C has a different view, committed to advancingsocietal knowledge, concerned about the total cost of care, butwishing to avoid side effects if possible. This patient has moder-ate interest in the probability of remission, and high interest inextending life expectancy, wishing primarily to survive until agranddaughter’s planned wedding.

Patient D is self-oriented, caring only about survival, remis-sion, pain, and side effects, and little at all about social or privatecost, advancing knowledge, or the quality of the data.

Patient E cares considerably about extending life expectancy,but is guilt-ridden about using resources that do not lead toremission, and so has great concern about the total cost oftreatment, and also wishes to contribute to human knowledgeabout curing this disease. Modestly concerned about side effects,this patient has full insurance coverage and hence minimalconcerns about out-of-pocket costs.

Table 2 highlights each hypothetical patient’s most preferredchoice of therapy. In some cases (e.g., patient A), the choices areclose in value, whereas in other cases (e.g., patient B) they differconsiderably. These scenarios illustrate our key point: preferencesmatter. Each of these hypothetical patient profiles could plausiblyrepresent a real person and their trade-off considerations.Although these profiles were constructed deliberately to makeour point, clearly the creation of preference weights that lead todifferent choices is not difficult. Indeed, our experience withusing multi-attribute models in the realm of global health forprioritizing new vaccine development has led us to expect thatdifferences in preference structure commonly lead to differentoutcomes in ranking of alternatives [17–19].

The values in Table 2 call to attention another feature ofmulti-attribute models: The scores of the different patients areunrelated to each other. Each patient’s preference structurecreates a different value scale, just as, for example, Fahrenheitand Celsius temperature scales differ. Furthermore, these scalesare not relative values. A value of 60 is not twice as good as a

value of 30; it is 30 points better, just as 801F is not twice aswarm as 401F, but is 401 warmer. These are interval, but notratio, scales.

Choosing by Groups

Although our hypothetical example has a single decision maker,the same conceptual approach remains valid in group decision-making processes. Groups would use one of several availablevoting methods to rank attributes in the model and to select(when necessary) appropriate values for any subjectively definedattributes. Each of these requires a collective choice, using avoting or grading process of some form, guidance for which touse is found in the social choice literature [20].

When might such issues appear? In health care, theoccasions may involve investment choices, personnel decisionsand related provision of care issues, or perhaps insurancecoverage decisions. Suppose a prepaid managed care plan oran accountable care organization wished to choose the bestcancer therapy for a particular condition. It might use samplesof real patients to help make a system-wide choice amongtherapeutic options. Many other resource planning decisionshave the same basic structure. The recent ISPOR Task Forcereports provide useful examples of these types of multicriteriaapplications in group settings [4,5].

On the basic research end of the spectrum, review panels ofgovernments or charitable foundations evaluate various grantproposals geared toward improving cancer therapy. Often in thecurrent processes, peer reviewers give a composite numericalscore to proposals under their consideration, with scores aver-aged to achieve final ranking recommendations. As an alterna-tive, they could evaluate each proposal on the basis of variousresearch attributes—uniqueness, likelihood of scientific break-through, public impact—and then use multicriteria models toprovide final scoring of research proposals.

Conclusions

Once various technical issues are resolved, we believe thatmulticriteria systems analysis models have much to favor themover alternative approaches used to guide health care choices,and possibly in broader program evaluation and comparisons. Fordecisions ranging from individual to societal, multicriteria mod-els provide a valuable extension over single-criterion modelssuch as cost-utility analysis. The participants in the health careenterprise (i.e., the 5Ps) commonly hold different perspectives,and so no single perspective can possibly meet all their needs.Using systems-based approaches improves transparency abouttheir decision processes, because all assumptions and values aremade specific. Often, these models can guide improvements inproduct design by varying attributes and costs of alternatives andmeasuring the value created by such variations. At least in somesettings, this transparency can facilitate decision convergenceamong otherwise competing viewpoints. These models also helpfocus efforts to improve data in an efficient manner usingsensitivity analysis. Finally, using these approaches also reducesrisks of cognitive error associated with intuitive judgments andchoices.

In our view, these gains in assisting decision making atvarious levels provide compelling reasons to shift from narrowanalytic techniques to systems approaches capable of incorpo-rating a wide array of factors that actually drive real decisions inhealth and medicine.

Table 2 – Treatment scores based on patient values.

Patient T1 T2 T3 T4 T5

A 58.6 56.0 58.5 50.3 54.9B 37.4 59.3 52.5 58.5 61.6C 32.0 39.1 59.4 42.8 36.4D 51.1 54.8 50.0 44.5 42.5E 37.1 49.5 48.8 59.8 46.7

Note. Figures in boldface represent the highest ranked treatmentfor each patient.

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