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MCDA OR WEIGHTED CEA BASED ON THE QALY? WHICH IS THE FUTURE FOR HTA DECISION MAKING?

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In this ISPOR session Chuck Phelps and Adrian Towse debated the case for and against using MCDA to support HTA decision making, as compared to weighting or augmenting a QALY based ICER approach. Chuck Phelps argued for use of MCDA, Adrian Towse for weighting the QALY. Nancy Devlin set the scene and moderated.

Author(s) and affiliation(s): Nancy Devlin, Director, Centre for Health Policy, University of Melbourne, Australia Adrian Towse, Emeritus Director, Office of Health Economics, London, UK Chuck Phelps, University of Rochester, Rochester, NY USA

Event: ISPOR 2019

Location: New Orleans, USA

Date: 21/05/2019

Published in: Health & Medicine
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MCDA OR WEIGHTED CEA BASED ON THE QALY? WHICH IS THE FUTURE FOR HTA DECISION MAKING?

  1. 1. Augmented CEA or MCDA? Prof. Nancy Devlin Director, Centre for Health Policy University of Melbourne IP11, ISPOR New Orleans, May 2019
  2. 2. Our starting point • HEOR%methods%and%evidence%generation%for%HTA%principally%focused%on% CEA%and%ICERs • But%even%in%cases%where%HTA%is%primarily%concerned%with%cost% effectiveness,%it%is%not%the%only%consideration • There’s%a%push%toward%more,%not%fewer,%criteria%eg. US%value%frameworks • Our%focus%in%this%session%is%how%HEOR%can/should%go%beyond%generating% evidence,%to%methods%to%assist%decision%makers%to%weigh%up%evidence%on% multiple%factors 2
  3. 3. 3 Why not rely on deliberation alone? • errors,'biases'&'heuristics' that'affect'individuals’' judgements'when'confronted'' with'complex'decision' problems • combined,'in'committee' deliberations,'with'group' dynamics,'differences'in' chairing'styles • can'materially'affect'the' decision'that'results'from' considering'a'given'body'of' evidence'
  4. 4. The role of judgement in HTA • We#need#judgements#to#be#made#(scientific;*social*value*judgements)*in*order*to*make* decisions*– there’s*no*way*around*that.**That’s*why*we*have*HTA*committees.* • But*we*also*want*a*process*where*those*judgements#are#made#in#a#consistent,# transparent#manner. • Accountability*to*taxpayers • Clear*‘signaling’*to*the*suppliers*of*health*technologies Saying*that*‘HTA*should*be*transparent’*is*a*value*judgement*– but*surely*a*fairly*weak* one?* 4
  5. 5. Time to move the debate along… (1)$Multiple$criteria$are$and$will$continue$to$be$used$in$HTA (2)$There$will$always$be$a$need$for$judgements$and$deliberation$in$HTA (3)$There$are$ways$of$improving$the$way$deliberation$and$judgements$are$ reflected$in$decision$making Two$principal$options: • ‘augment’$the$standard$CEA$approach • adopt$some$form$of$structured$decision$making$(MCDA) What$are$the$relative$merits$of$each?$ 5
  6. 6. What do we mean by augmented CEA? • Reflecting*considerations*other*than*health*care*costs*and*QALYs ICER*=*(∆ Costs/∆ QALY+)* 6 In*the*denominator:* weighting*the*QALYs*to* reflect*characteristics*of* those*experiencing*them In*the*numerator:* include*wider*costs*and* offBsetting*monetary* benefits*not*captured*in* the*QALYs* The*ratio:*Vary*the* threshold*used*to*judge* the*ICER*to*reflect*factors* not*captured*in*the* numerator*or*denominator
  7. 7. What do we mean by MCDA? Resources: ISPOR-taskforce-reports-on-MCDA:--Marsh-et-al--(2016)-and-Thokala-et-al-(2016) Hansen-&-Devlin-(2019)-MCDA-in-health-care-decision-making-Oxford-Encyclopedia-of-Economics-and-Finance 7 • MCDA-formally-incorporates-multiple-dimensions-of-value.- • DecisionOmakers-select-relevant-dimensions-of-value-and-use-their-judgement-to- specify-their-relative-importance-(“weights”).- • MCDA-models-combine-each-option’s-performance-on-chosen-dimensions-of-value- into-comprehensive-scores,-used-to-rank-options.- • Many-different-MCDA-approaches;--supported-by-software • Processes-to-elicit-decisionOmakers’-weights-vary-considerably-across-different- models.-
  8. 8. Our protagonists Adrian'Towse Arguing'the'case'for'augmented'ICERs Charles'Phelps Arguing'the'case'for'MCDA' &'plenty'of'time'for'discussion'and'debate' from'the'floor 8
  9. 9. 9 Thanks! email: nancy.devlin@unimelb.edu.au twitter: @HEU_unimelb @nancydevlin1
  10. 10. Multi&Criteria+Decision+Analysis+(MCDA) vs Cost&Effectiveness+Analysis+(CEA) Charles(E(Phelps,(PhD University(of(Rochester Rochester,(NY(USA(
  11. 11. What’s'good'about'CEA? • It#flows#directly#from#the#theory#of#maximizing#expected#utility#for#a# single#(representative)#individual.# • If#properly#used,#it#leads#to#efficient#use#of#resources#to#improve# health. • It#is#widely#used#and#generally#well=understood.#
  12. 12. What%Can’t%CEA%Do? • Deal%with%issues%of%equity,%fairness • Income%and%wealth%disparities • Racial%disparities • Regional%issues%(e.g.,%Northern%Territories%in%Canada) • Incorporate%things%that%can’t%be%measured%in%QALYS%or%$ • Fear%factor%from%dread%disease • Fit%with%local%health%system%or%beliefs
  13. 13. What%Does%CEA%Do%Poorly,%If%at%All? • Contagious*diseases • Herd*immunity • Microbial*resistance*to*antibiotics*(AMR) • Other*Externalities* • Scientific*spillovers • People*with*multiple*chronic*conditions • Add*or*multiply*QALY*adjustments? • The*“disabled”*issue*looms*large*in*US*policy
  14. 14. MCDA%Models%in%a%Nutshell • MCDA%formally%incorporates%multiple%dimensions%of%value.% • Decision8makers%select%relevant%dimensions%of%value%and%specify%their%relative% importance%(“weights”).% • Processes%to%elicit%decision8makers’%weights%vary%considerably%across%different% models.% • They%are%not%yet%perfected!% • MCDA%models%combine%each%candidate’s%performance%along%chosen%dimensions% of%value%into%comprehensive%scores%used%to%rank%candidates.% • Despite%differences%in%intellectual%heritage,%these%value%metrics%often%use%simple% linear%combinations%(using%decision8makers’%weights)%of%each%candidate’s% performance%(perhaps%mathematically%transformed)%on%each%value%dimension%
  15. 15. In#the#simplest#form Multi&attribute+Utility+Index+(MAUI)+for+candidate+j,+where+!" =+weight+ placed+by+decision+maker+on+attribute+i and+#"$ =+normalized+score+for+ candidate+j+on+attribute+i:+ %&'($ = Σ"!"#"$
  16. 16. HOW$MCDA$Can$Help • It#formally#brings#“other#issues”#into#the#model • Explicit,#transparent#to#others • Usable#at#different#levels#of#decision=making • System=wide#adoption#of#technologies • Coverage#in#health#insurance#plans • Decisions#by#individual#patients • Which#health#plan • Which#among#available#treatments
  17. 17. Virtues(of(MCDA • Transparency."How"“other"issues”"are"incorporated"into"decisions"is"wholly"transparent,"specified"by" the"weights"assigned"in"MCDA"models"to"each"attribute"in"the"decision"model." • “Flight1simulator”1testing."Allows"people"to"test"perceived"value"of"alternative"combinations"of" attributes"before"construction"or"purchase,"potentially"focusing"R&D"in"earlier"development"stages." • Guiding1data1improvement."Data"imperfections"always"exists."MCDA"models"help"focus"dataC improvement"efforts"on"those"data"that"mostCaffect"decisions,"thus"conserving"resources." • Decision1convergence.1In"some"(but"not"all)"settings,"MCDA’s"formal"structure"can"assist"decision" convergence,"since"goals"and"preferences"of"all"participants"are"clearly"visible"as"MCDA"weights." Obviously,"in"some"settings,"some"parties"may"prefer"otherwise,"but"MCDA"can"have"value"in"others." • Avoiding1cognitive1bias."The"emerging"field"of"“behavioral"economics”"has"cataloged"over"two"dozen" common"errors"in"human"decision"making,"many"involving"steps"necessary"to"make"“intuitive”" choices."MCDA’s"formal"structure"helps"to"bypass"most"of"these"cognitive"errors."
  18. 18. Barriers'to'Use • “MCDA&requires&too&much&data.”& • Response:(Yes,%MCDA%requires%more%data.%%But%the%problems,%not%the%MCDA%models,% create%this%complexity. • “It’s&too&easy&to&‘game’&the&results.”& • Response:(You%can%“get%any%outcome%you%want”%by%changing%the%weights%in%the% MCDA%model,%but%MCDA%models%actually%make%it%more%difficult%to%do%this,%not%less% so,%since%the%decision%structures%(weights%on%attributes)%are%wholly%visible. • “The&meaning&of&the&resulting&‘index’&is&unclear.”& • Response:(Each%MDCA%model%has%its%own%scoring%system,%since%users%determine%their% own%weights.%Thus%“my”%index%is%not%comparable%to%“your”%index,%but%the%weights% are%visible,%so%the%meanings%can%be%easily%inferred.% • “MCDA&models&are&too&complicated&to&use.”& • Response:((Different%models%differ%hugely%in%demands%on%users.%%Working%to% maximize%usability%is%key%to%further%expansion%of%MCDA%use.% • “You&can’t&use&MCDA&in&situations&with&a&budget&constraint.”& • Response:&&No%longer%true.%%Several%approaches%allow%calculating%cost/value%ratios% just%as%done%with%ICERs%in%CEA.%
  19. 19. Next%steps%to%expand%use • Reduce&&user&complexity • How&many&decisions&must&“decision&makers”&make? • Models&differ&by&factors&of&K&or&N • Decision&making&in&groups • Voting&methods • Increased&data&availability • Increased&pool&of&skilled&“users” • Familiarity&breeds&acceptability
  20. 20. Who$can$(and$should)$do$what? • Consensus'on'key'attributes • Differs'by'disease • Patient'viewpoint'essential • Medical'specialty'groups?''Patient'advocacy'groups?'…..'????' • Expanded'data'gathering • FDA'rules • Build'from'Expanded'and'Augmented'CEA • Improved'software'for'groups'and'individuals'(ease'of'use) • Trained'personnel' • MPH,'Public'Policy,'Systems'Engineering,'Business,'… • Build'into'routine'clinical'settings'where'appropriate • Must'be'simple'patientKcentric'decision'tools
  21. 21. Improving*CEA*also*has*value • MCDA%models%“must”%include%QALYS% • or%other%health%outcome%measures • In%most%cases,%should%be%first%ranked%attribute • In%many%cases,%probably%a%significant%proportion%of%the%weight • Opportunities%exist%to%expand%QALY%concept • Incorporating%uncertainty%of%outcomes%(variance,%skewness,…) • Ongoing%work%by%Lakdawalla and%Phelps%addresses%these%issues% • Possibly%also%“scientific%spillovers) • Requires%subjective%estimates%of%probabilities% • Others?
  22. 22. CONCLUSION:))The)Future)is)MCDA “On$the$plains$of$hesitation Bleach$the$bones$of$countless$millions$ Who,$at$the$dawn$of$victory,$sat$down$to$wait,$ And$waiting….$died.”$ (George$W.$Cecil.)$
  23. 23. Thank&you&for&your&attention!
  24. 24. ohe.org PRESENTATOINISPOR*NEW*ORLEANS The case for Weighted / Augmented CEA MCDA*OR*WEIGHTED*CEA*BASED*ON* THE*QALY?*WHICH*IS*THE*FUTURE* FOR*HTA*DECISION*MAKING? Adrian*Towse Emeritus*Director*and*Senior*Research*Fellow Visting*Professor,*London*School*of*Economics* 21ST MAY(2019
  25. 25. Issues ISPOR&NEW&ORLEANS IP11 25 21ST MAY(2019 ●The centrality of health gain ●Additional attributes of value ●The challenge of opportunity cost ●Who is the decision maker? Welfarist versus extra-welfarist approaches ●The UK experience of failed implementation of Value- based pricing ●Deliberation and weighted CEA ●Which is the general case? Is weighted CEA a specific example of MCDA, or vice versa?
  26. 26. The centrality of health gain ISPOR&NEW&ORLEANS IP11 26 21ST MAY(2019 ● Assume(it(is(measured(in(QALYs ● A(core(challenge(– can(we(have(things(that(the(health(system(or(an(intervention(provides(that(are(not(to(do( with(health?( ● The(answer(is(“yes”,(for(example(reassurance,(or(indeed(information(that(is(not(reassuring(but(enables(life( choices(to(be(made(– the(“value(of(knowing.”( ● But(I(would(argue(it(is(a(key(challenge.(If(it(is(not(an(attribute(that(can(be(expressed(as(a(multiple(of(health( gain(then(think(carefully. ● This(links(to(a(related(issue.(Even(if(this(“non(health”(attribute(exists(should(the(health(system(be(paying(for( it?([This(may(also(apply(to(attributes(that(can be(expressed(as(multiples(of(health(gain] ● Again(the(answer(can(be(“yes”(providing(we(are(clear(that(this(is(part(of(the(objectives(of(the(health( system(to(be(paid(for(in(premiums(P be(they(funded(via(taxes,(social(insurance(or(private(insurance.( ● If(we(have(these(elements(that(are(not(a(function(of(health(gain(and(so(cannot(be(in(a(weighted(QALY(then( we(can(have(an(estimate(of(Net(Monetary(Benefit((NMB)(or(Net(Health(Effects((NHE),(i.e.(we(can(convert( them(into($$(or(QALYs(if(we(have(the(relevant(“rate(of(exchange”,(MRS,(or(ʎ
  27. 27. Additional attributes of value ISPOR&NEW&ORLEANS IP11 27 21ST MAY(2019 Value Quality( adjusted- life(years-- (QALYs)- gained- Net-health- system- costs Productivity Adherence (improving- factors Value-of- knowing Fear-of- contagion Insurance- value Severity-of- disease Value-of- hope Real- option( value Equity Scientific- spillovers • Do(we(want(to( include(them? • Can(we( measure? • Can(we(avoid( double( counting? • How(do(we( aggregate?
  28. 28. The challenge of opportunity cost ISPOR&NEW&ORLEANS IP11 28 21ST MAY(2019 ●We(are(looking(at(value8for8money(or(cost8effectiveness,(so(what(are(we(comparing(our(value(to?( What(is(our(ʎ? ●If(we(start(from(a(Garber(and(Phelps(individual(utility(function1,(then(we(have(marginal(rates(of( substitution((MRS)(as(between(the(different(elements(of(expenditure(on(health((measured(in(QALYs)( and(other(goods(and(services. ● Additional(elements(of(treatment(value(can(be(measured(and(we(can(construct(a(NMB(or(NHE((latter( using(the(MRS(as(between(consumption(and(health). ●However,(we(have(third(party(payers(acting(on(behalf(of(their(enrollees /(citizens.(Two(issues: ●Heterogeneity(of(preferences.(However,(a(payer(could,(for(example,(take(the(preferences(of(their( median(enrollee /(citizen ●The(payer(may(not(wish(to,(or(regard(it(as(efficient,(to(adopt(the(preferences(of(their(enrollees /( citizens(for(two(reasons: " (i)(They(have(a(budget(constraint(and/or((ii)(they(have(a(different(social(welfare(function 1(Garber(and(Phelps,((Economic(foundations(of(cost8effectiveness(analysis.(J(Health(Econ.(1997(FebZ16(1):1831.
  29. 29. Who is the decision maker? Welfarist versus extra-welfarist approaches ISPOR&NEW&ORLEANS IP11 29 21ST MAY(2019 ●Are(we(constructing(a(CBA(for(a(“welfarist”(decision(maker?(We(are(using(a(“demand(side”( threshold ●Or(do(we(have(an(“extraFwelfarist”(health(plan((public(or(private)(decision(maker(s)(with(a( budget(constraint(that(does(not(allow(them(to(efficiently(adopt(all(treatments(with(a(positive( CBA.(We(are(using(a(“supply(side”(threshold.( ●By(implication,(if(ʎ(is(less(than(the(MRS((or(citizen(WTP(for(a(QALY),(then(there(is(a( divergence(as(between(short(and(long(run(preferences.( ●The(“extraFwelfarist”(health(plan(decision(maker(s)(can(use(one(or(more(of((i)(weights(based( on(citizen(preferences((ii)(weights(applied(to(∆QALY(effects((iii)(an(MCDA(or(deliberative( decision(making(process.( ●So(an(extraFwelfarist can((a)(weight(the(∆QALY(with(attributes((a)((that(seeks(to(take( account(of(opportunity(cost((oc)((wa/woc)(and((b)(express(nonFQALY(attributes(in(money(and( then(convert(into(QALYs(using(an(exchange(rate(of((ʎ(/(MRS)
  30. 30. The UK experience of failed implementation of Value- based pricing (VBP) ISPOR&NEW&ORLEANS IP11 30 21ST MAY(2019 ●In(2013(the(UK(Health(Minister(sought(to(introduce(VBP.((This(included: ●A(disease(severity(adjustment(to(the(∆QALY,(with(weights(anchored(around(the(average( disease(severity(of(patients(treated(in(the(health(plan ●A(calculation(of(the(net(social(impact(of(a(treatment,(converted(into(QALYs(using(an( exchange(rate(of((ʎ(/(MRS) ●Conversion(into(a(price(using(the(threshold(ʎ ●The(Minister(retreated(and(handed(it(the(“hot(potato”(of(VBP(to(NICE ●NICE(replaced((i)(the(disease(severity(adjustment(with(a(proportional(shortfall(adjustment( and((ii)(the(net(social(impact(calculation(with(an(absolute(shortfall(adjustment.(The(adjusted( ICER(would(be(compared(with(the(threshold(range(of(£20K(to(£30K.( ●Both(were(weighted(CEA(approaches(that(could(be(operationalised. ●They(were(abandoned(because(of(equity(concerns(and(loss(of(deliberation.(
  31. 31. Deliberation and weighted CEA ISPOR&NEW&ORLEANS IP11 31 21ST MAY(2019 ●The(weights(do(not(need(to(be(pre8set.(It(can(be(left(to(the(decision8makers(in( Committee(to(give(implicit(or(explicit(weights(to(the(factors(they(think(are( relevant.( ●This(can(be(applied(to(the(attributes(converted(into(QALYs(using(an(exchange( rate(of((ʎ(/(MRS).(They(can(be(downrated(or(uprated. ●The(decision8makers(in(Committee(act(as(extra8welfarists making(a(decision(in( line(with(their(social(welfare(function((SWF). ●It(may(be(that(the(SWF(is(formed in(deliberation.(Preferences(are(not(pre8set( but(related(to(both(the(nature(of(the(items(under(discussion(and(the(views( formed(in(the(deliberative(process.
  32. 32. Which is the general case? Is weighted CEA a specific example of MCDA, or vice versa? ISPOR&NEW&ORLEANS IP11 32 21ST MAY(2019 ●Weighted(CEA(is(a(specific(example(of(MCDA.(It(is,(arguably,(“clean”.(It(focuses(on( health(gain(and(enables(an(element(of(deliberation(in(setting(weights(for(decision( making.( ●Or(as(the(UK(VBP(example(shows,(it(can(be(algorithmic(and(set(a(drug(price.( ●The(challenge(for(Chuck(is,(therefore,(what(do(other(forms(of(MCDA(bring(to(the( party?
  33. 33. To#keep#up#with#the#latest#news#and#research,#subscribe#to#our#blog.# OHE’s#publications#may#be#downloaded#free#of#charge#from#our#website. ohe.org OHE Southside 105#Victoria#Street## London#SW1E 6QT## United Kingdom Telephone +44#(0)20#7747 8850 FOLLOW%US To#enquire#about#additional#information#and#analyses,## please contact: Adrian%Towse Emeritus%Director%and%Senior%Research%Fellow,%OHE Visting%Professor,%London%School%of%Economics atowse@ohe.org

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