Some practical ethics of cost-effectivenessin public health care systems

Tony CulyerInstitute for Work & Health

©2004 Institute for Work & Health

Ethical building blocks – the context

• Publicly funded provision

• Ownership of providers is another matter

• Equal terms of access

• Cheap terms of access

• Funding conditional on performance

Building blocks – ethical points of departure

• The “authority”o Efficiencyo Equity

• Paretian welfare economicso Preference-basedo Distributionally neutral/silent

Building blocks – ethical points of departure

Extra-welfarism• Health care → health → flourishing• Consistent with moral authoritarianism• Eclectic costs and benefits• Cardinality and interpersonal

comparisons allowed• Distributive equity allowed

Context – NHS in England and Wales

• Public funding for a wide range of services

• Mixed public/private provision

• Moral authority (?)o Health maxo Distributive justice

The National Institute for Clinical Excellence (NICE)

• To identify cost-effective technologies and make recommendations for their use in the NHS

• To create authoritative clinical guidelines to support cost-effective clinical practice in all health care settings

NICE – some characteristics

• ‘Technology’ – a broad concept

• Guidance not binding on NHS but critical for Clinical Governance

• Statutory obligation to fund clinical decisions based on NICE guidance

• Transparency

• Participatory

NICE – examples of openness (1)

• Open Board meetings (around England & Wales)

• Minutes published before confirmation

• Partners Council

• Citizens Council

• Consultative exercises in appraisals process

NICE – examples of openness (2)

• Membership of Technology Appraisals panels

• Appeals procedure

• Consultative process about process

• Liaisons with Royal Colleges and National Collaborating Centres

• Joint working with NHS R&D and the National Coordinating Centre for HTA

Ethical issues in NICE’s technology appraisals

Guide to the Methods of Technology Appraisal (NICE 2004)

• The context• The Reference Case• The scoping phase• Perspective• Outcome measurement (what costs and what benefits?

How measured?)• Distribution of costs and benefits (QALY bias and QALY

weights)

The Context

Transparency:

o Is categorically goodo Is instrumentally goodo Creates expectationso Is, however, a hostage to

fortune

The ‘reference case’

The Institute has to make decisions across different technologies and disease areas. It is, therefore, important that analyses of clinical and cost effectiveness undertaken to inform the appraisal adopt a consistent approach. To facilitate this, the Institute has defined a ‘reference case’ that specifies the methods considered by the Institute to be the most appropriate for the Appraisal Committee’s purpose and consistent with an NHS objective of maximising health gain from limited resources. … This does not preclude additional analyses being presented where one or more aspects of methods differ from the reference case. However, these must be justified and clearly distinguished from the reference case.

(NICE 2004, p.19)

Three implications:

• The scope is constrained by the maximand (“health gain”) which might have ethical justification via the routes already discussed (or, indeed, others);

• It is constrained by a need for consistency in analytic methods (or as much as is necessary for its Appraisal Committee to be able to function effectively);

• It is pragmatic, not only in the two respects just mentioned (bullets above) but also in that exceptions to the reference case are permitted.

Some personal meta-moral principles

• Never let the perfect become the enemy of the merely good (Voltaire, roughly)

and

• The highest form of treason: to do the right thing for the wrong reason. (Eliot, exactly)

and

• If economists could manage to get themselves thought of as humble, competent people, on a level with dentists, that would be splendid! (Keynes, exactly)

The reference case – specifics 1

Reference case

The scope developed by the Institute

Alternative therapies routinely used in the NHS

NHS and PSS

All health effects on individuals

Cost-effectiveness analysis

Element of HTA

Defining the decision problem

Comparator

Perspective on costs Perspective on outcomes

Type of economic evaluation

The reference case – specifics 2

Element of HTA

Synthesis of evidence on outcomes

Measure of health benefits

Description of health states

Reference case

Based on a systematic review

Quality-adjusted life-years (QALYs)

Health states described using a standardised and validated generic instrument for calculation of QALYs

The reference case – specifics 3

Reference case

Choice-based method, for example, time trade- off, SG, (not a rating scale) Representative sample of the public

Annual rate of 3.5% on both costs and health effects

An additional QALY has the same weight regardless of the other characteristics of the individuals receiving the health benefit

Element of HTA

Method of preference elicitation for health state valuation

Source of preference data

Discount rate

Distributive equity

The scoping phase

• Defines framework for the subsequent appraisal

• Inescapably entails explicit or implicit value-judgements

• Precedes every NICE technology appraisal

• Determines the specific questions to be addressed

• Involves consultation and response to it

The scope specifies (1):

• the clinical problem and the population(s) and any relevant subgroups for whom treatment with the technology is being appraised (choices here may be determined by both scientific judgements and ethical ones – like the personal and social significance attached to particular clinical ‘problems’);

• the technology and the setting for its use (for example, hospital or community);

The scope specifies (2):

• the relevant comparator technologies (it is easy to ‘rig’ a comparison of technologies by suitable biased choices, or to render an analysis fairly useless by using evidence from trials using only a placebo comparator);

• the principal health outcome measures appropriate for the analysis (pragmatism is likely to rule here but choice of outcome measure – the denominator in an incremental cost-effectiveness ratio – is a critically important social value-judgement);

The scope specifies (3):

• the measures of costs to be assessed (again, interpreted as opportunity cost, this raises the same ethical issues as the choice of outcome measure, since cost is essentially “health gain forgone”);

• the time horizon over which benefits and costs will be assessed (both the period and the rate of discount entail value-judgements);

The scope specifies (4):

• other considerations, for example, identification of patient subgroups for whom the technology might potentially be particularly cost effective (this implies that some patients ought to receive more favourable consideration than others - clearly value-judgmental);

• special considerations and issues that are likely to affect the appraisal

• the extent of the evidence (while this may involve scientific value judgements – “is this research well-conceived and conducted?” – it is less likely to involve social value-judgements)

Perspective

The scope is constrained to adopt a specific perspective:

the perspective on outcomes should be all direct health effects whether for patients or, where relevant, other individuals (principally carers). The perspective adopted on costs should be that of the National Health Service and the Personal Social Services. If the inclusion of a wider set of costs or outcomes is expected to influence the results significantly, such analyses should be presented in addition to the reference case analysis.

Pragmatism

The procedure is pragmatic in two particular ways:

(a) it locates the task as one of optimizing (possibly sub-optimizing) within a frame defined by a budget and a mission statement

(b) it has a caveat enabling other elements to be adduced when judgment (ultimately that of NICE’s Board) suggests that it may be desirable

The outcome measure

• Preferred measure: Quality-Adjusted Life-Year (QALY) in EQ-5D version

• EQ-5D, a 3-level, 5-dimensional index

• Dimensions are: Mobility, Self-care, Usual activity, Pain/discomfort, Anxiety/depression

• Scoring on a three-point scale (1- no problem, 2 - some problem, 3 - extreme problem)

• 245 health states (35 plus perfect health and dead) on an interval scale

Why the QALY (1)?

• there was a general agreement amongst colleagues that we needed an outcome measure that related as closely as possible to the Secretaries of State’s charge to NICE regarding “health” (we effectively recognized his “authority”);

• I had a personal stake in the QALY from its earliest inception at York in 1971 and subsequent development mainly by Alan Williams (which is no moral reason for promoting it, of course, merely the open admission of a bias!);

•

Why the QALY (2)?

• It was sufficiently close to common outcome measures used by clinicians in research (e.g. life-years or 5-year survival rates) to be a familiar starting point;

• It was an index rather than a profile;• It was generic – applicable to a wide range of

technologies, thus facilitating comparison of relative cost-effectiveness;

Its theoretical properties and their consequences were (or were becoming) well-understood (properties such as constant proportional trade-off, risk neutrality over life-years, additive independence in health states);

Why the QALY (3)?

• The trade-offs embodied in it were derived from a representative sample of the UK population;

• The ethical arguments adduced against it did not seem persuasive – particularly when it was compared with practical alternatives (let alone impractical ones!);

• It had the attractive attribute of identifying, in a classically reductionist way, a set of value-judgmental issues for resolution (once one had, of course, accepted the essentially pragmatically crude utilitarian nature of the construct) - some of these come up later;

• It was simple and low-cost.

Appraisals Committees (3 of them) • Statisticians • General Practitioners • Patient advocates • Public Health/Investigation/Consultant Physicians • Health Economists • Clinical Pharmacists/Pharmacologists • Nurses • Consultant Surgeon • NHS Management • Association of British Healthcare Industries Representative • Psychiatrist • Professions Allied to Medicine • Paediatrician

Others in appraisals processConsultees

Manufacturer(s) or sponsor(s) of the technology; national professional organizations; national patient organizations; the Department of Health and the Welsh Assembly Government; relevant NHS organizations in England and Wales. Consultees participate in consultation on the draft scope, the Assessment Report and the Appraisal Consultation Document. Consultee organizations representing patient/carer-givers and professionals nominate clinical specialists and patient experts to present their personal views to the Committee. Consultees may appeal against recommendations.

Commentators

Organizations that engage in the appraisal process but that are not asked to prepare a submission dossier: manufacturers of comparator technologies; NHS Quality Improvement Scotland; the relevant National Collaborating Centre; other related research groups such as the Medical Research Council; various other groups such as the NHS Purchasing & Supplies Agency, the British Medical Association. No right of appeal.

Assessment Group

An independent academic group which prepares a review of the clinical and cost-effectiveness of the technology(ies) based on a systematic review of the literature and a review of manufacturer and sponsor submissions to the Institute.

Distributional equity

Recall the egalitarian proposition:

An additional QALY has the same weight regardless of the other characteristics of the

individuals receiving the health benefit

Aristotelian principles

• Horizontal equity – the equal treatment of those who are equal in an ethically relevant sense

• Vertical equity – the unequal treatment of those who are unequal in an ethically relevant sense.

What might some of these ethically relevant characteristics be?

Possible candidates for (un)equal weighting (1)

• their pre- or post-treatment level of health (e.g. an incremental QALY is as socially valued when it accrues to someone with a lifetime of chronic incapacity as to someone who has hitherto been in abundant health)

• their current level of health (e.g. an incremental QALY is as socially valued when it accrues to someone who is currently very ill as to someone receiving preventive care who is perfectly fit)

• the size of the increment in health they may derive (e.g. the tenth incremental QALY someone receives is a socially valued as the first)

Possible candidates for (un)equal weighting (2)

• age (e.g. a QALY for an aged person is as socially valued as one for a youngster)

• gender• economic productivity• lifestyle (e.g. a QALY for someone whose

reckless style of life has brought about or exacerbated their ill-health is a socially valued as one for an impeccably clean-living type)

Equality…?

• Of what? (environments, inputs, outputs/outcomes, processes, characteristics of people?)

• Of whom? (what sorts of people with what sorts of pasts, presents and futures?)

• For what? (for its own sake, to achieve more fundamental equalities?)

To wind up

• the usefulness of adopting an extra-welfarist perspective on the ethical issues involved in appraising health care technologies (whether by cost-effectiveness analysis or anything else) – its provision of a systematic mode of thinking through the myriad issues that need to be resolved

• the flexibility of the E-W approach – enabling many considerations to be adduced (or excluded) according to the “rulings” of the ethical “authority” (or, more realistically, one’s guesses as to what these rulings would be!)

And …

• The explicitness with which the key issues are brought into the light for debate and decision

• The empiricist approach to finding out what values are held in the community as a basis for embodying consensus (if it exists!) in the analysis

• The openness with which problems are identified and shared, and resolution is sought

Web site: www.iwh.on.caE-mail: aculyer@iwh.on.caTony’s phone: 416-927-2118Tony’s web pages: http://www-users.york.ac.uk/~ajc17