Some practical ethics
of cost-effectiveness
in public health care
systems
Tony Culyer
Institute 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 Efficiency
o Equity
•
Paretian welfare economics
o Preference-based
o 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
o
Health max
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
o
o
o
Is categorically good
Is instrumentally good
Creates expectations
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
Element of HTA
Reference case
Defining the decision problem
The scope developed by
the Institute
Comparator
Alternative therapies
routinely used in the NHS
Perspective on costs
NHS and PSS
Perspective on outcomes
All health effects on
individuals
Type of economic evaluation
Cost-effectiveness analysis
The reference case – specifics 2
Element of HTA
Reference case
Synthesis of evidence on
outcomes
Based on a systematic
review
Measure of health benefits
Quality-adjusted life-years
(QALYs)
Description of health
states
Health states described
using a standardised and
validated generic instrument
for calculation of QALYs
The reference case – specifics 3
Element of HTA
Reference case
Method of preference
elicitation for health state
valuation
Choice-based method, for
example, time trade- off, SG,
(not a rating scale)
Source of preference data
Representative sample of the
public
Discount rate
Annual rate of 3.5% on both
costs and health effects
Distributive equity
An additional QALY has the
same weight regardless of
the other characteristics of
the individuals receiving the
health benefit
The scoping phase
•
Defines framework for the subsequent appraisal
•
Inescapably entails explicit or implicit valuejudgements
•
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 wellconceived 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. lifeyears 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 process
Consultees
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 costeffectiveness 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.ca
E-mail: aculyer@iwh.on.ca
Tony’s phone: 416-927-2118
Tony’s web pages:
http://www-users.york.ac.uk/~ajc17