Koonal gave a seminar at the Manchester Centre for Health Economics at the University of Manchester. His presentation covered a review of the literature on societal support for an end of life premium and some of the challenges associated with attempting to measure the 'overall' preference of society. Author(s) and affiliation(s): Koonal Shah, Office of Health Economics Event: Manchester Centre for Health Economics seminar series Location: University of Manchester, Manchester, UK Date: 02/04/2019

1. Koonal Shah, Office of Health Economics
MCHE seminar • 4 February 2019
End of life treatments, societal
values, and selecting a measure
of ‘overall’ preference

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Outline
• End of life premium: some background
• Review of the stated preference literature
• Challenges in categorising studies and
determining the ‘overall’ preference

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• Common assumption: principal objective of
health care is to maximise population health
using available resources (QALY-maximisation)
• Entails the QALY is a QALY principle
Background

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QALY = QALY

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Rejection of new cancer treatments
Recommendation of the Richards (2008) review:
NICE should demonstrate greater flexibility and assess
“what measures could be taken to make available drugs
used near the end of life that do not meet the cost-
effectiveness criteria applied to all drugs” (p.4)

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• Criteria that need to be satisfied for NICE’s
supplementary end of life policy to apply:
• If met, NICE’s appraisal committee will consider
assigning additional weight to the QALY benefits
generated by the treatment
NICE end of life criteria
C2
The treatment is indicated for patients with a short life
expectancy, normally less than 24 months
There is sufficient evidence to indicate that the treatment
offers an extension to life, normally of at least an additional
three months, compared to current NHS treatment
The treatment is licensed, or otherwise indicated, for small
patient populationsC3
C1

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Rationale for NICE’s policy
“The Institute recognises that the public, generally, places special
value on treatments that prolong life – even for a few months –
at the end of life, as long as that extension of life is of reasonable
quality (at least pain-free if not disability-free).” (p.348)

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• In Scotland, a ‘New Medicines Fund’ has been introduced
to increase access to medicines for patients with rare or
end of life conditions
• Followed calls from the Scottish Government for the
Scottish Medicines Consortium to revise its appraisal
methods to take better account of orphan and end of life
treatments
Elsewhere in the UK…

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• Placing additional weight on survival benefits in patients
with short remaining life expectancy could be considered a
valid representation of society's preferences
• But the NICE consultation revealed concerns that there is
little scientific evidence to support this premise
• Two (unpublished) reviews undertaken in 2011 did not
identify many relevant studies
Evidence for an end of life premium

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Case for examining societal preferences
Adaptation of logic presented by McNamara (HESG 2019):
• The relative priority to be assigned to end of life patients
is ultimately a matter of judgement, upon which
reasonable people may, and do, reasonably disagree
• In matters of value upon which reasonable disagreement
exists, democracy is capable of providing a procedurally
justifiable set of values which can be reasonably utilised
in social decision making
So…
• The relative priority assigned to end of life patients
should be democratically defined according to the views
of the public

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And if you don’t buy that…
• Review undertaken by the Department of Health to
assess the potential impact of the Cancer Drugs Fund
(CDF) noted the need for robust evidence to support the
weighting of health gains accruing to severely ill or end of
life patients
• Two former chairs of the SMC claimed (referring to
NICE’s end of life policy, the CDF and Scotland’s New
Medicines Fund): [there] has been tacit acceptance that
the changes made match the view of UK society, yet
there has been no exploration of whether that is, in fact,
the case” (Webb and Paterson, 2016)
• Consistent with NICE’s own position on citizen
involvement (see next slide)

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Extract from NICE’s SVJ guide
“The NHS is funded from general taxation, and it is right
that UK citizens have the opportunity to be involved in the
decisions about how the NHS’s limited resources should be
allocated.”
NICE, 2008. Social value judgements: Principles for the development of NICE guidance (p.10)

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Review of the literature
Research question:
Do members of the general public wish to
place greater weight on a unit of health
gain for end of life patients than on that for
other types of patients?
Short version:
Is there support for an ‘end of life
premium’?

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Review of the literature

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Inclusion criteria
1. Publication: Article must be published in English in a peer-reviewed
source.
2. Empirical data: Article must review, present or analyse empirical data.
3. Priority-setting context: Article must relate to a health care priority-
setting or resource allocation context. Articles reporting preferences
from an individual or ‘own health’ perspective (rather than a social
decision maker perspective) can be included as long as they clearly
seek to inform health care priority-setting policies.
4. Stated preference data: Article must report preferences that were
elicited in a hypothetical, stated context using a choice-based approach
involving trade-offs.
5. End of life: Article must inform the topic of placing greater weight on a
unit of health gain for end of life patients (i.e. patients with short life
expectancy) than on that for other types of patients.
6. Original research: Article must present original research and must not
be solely a review of the literature.

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Summary of included studies (n=23)
Authors (date) Country N Method Mode Summary of primary study objective(s)
Abel Olsen (2013) NOR 503 Choice Internet survey To test for support for end of life prioritisation and the fair innings
approach
Baker et al. (2010) UK 587 DCE CAPI To test for support for multiple prioritisation criteria
Dolan & Cookson (2000) UK 60 Choice Focus group Qualitative examination of support for multiple prioritisation criteria
Chim et al. (2017) AUS 3,080 Budget
allocation
Internet survey To test for support for multiple prioritisation criteria
Dolan & Shaw (2004) UK 23 Choice Focus group To test for support for multiple prioritisation criteria
Dolan and Tsuchiya
(2005)
UK 100 Choice;
ranking
Self-completion survey To compare support for prioritisation according to age vs.
prioritisation according to severity/life expectancy
Kwon et al. (2017) KOR 330 AHP Personal interview To test for support for multiple prioritisation criteria
Lim et al. (2012) KOR 800 DCE Internet survey To test for support for multiple prioritisation criteria
Linley & Hughes (2013) UK 4,118 Budget
allocation
Internet survey To test for support for multiple prioritisation criteria
McHugh et al. (2015) UK 61 Q method Personal interview Qualitative examination of societal perspectives in relation to end of
life prioritisation
Pennington et al. (2015) Multiple 17,657 WTP Internet survey To compare WTP for different types of QALY gain
Pinto-Prades et al.
(2014)
SPA 813 WTP; PTO CAPI To test for support for end of life prioritisation and to compare
support for life extensions vs. quality of life improvements
Richardson et al. (2012) AUS 544 Other Internet survey and
self-completion survey
To test a technique for measuring support for health-maximisation
and health sharing
Rowen et al. (2015) UK 3,669 DCE Internet survey To test for support for multiple prioritisation criteria
Shah et al. (2014) UK 50 Choice Personal interview To test for support for end of life prioritisation
Shah et al. (2015) UK 3,969 DCE Internet survey To test for support for end of life prioritisation
Shiroiwa et al. (2010) Multiple 5,620 WTP Internet survey To obtain the monetary value of a QALY (in six countries)
Shiroiwa et al. (2013) JPN 2,283 WTP Internet survey To obtain the monetary value of a QALY
Skedgel et al. (2015) CAN 656 DCE Internet survey To test for support for multiple prioritisation criteria
Stahl et al. (2008) USA 623 Choice Internet survey To test for support for multiple prioritisation criteria
Stolk et al. (2005) NLD 65 Choice Personal interview To test for support for multiple approaches to priority-setting
Wouters et al. (2017) NLD 46 Q method Personal interview and
focus group
Qualitative examination of societal perspectives in relation to end of
life prioritisation

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Summary of findings
Freq. %
Overall finding: end of life premium
- Evidence consistent with an end of life premium
- Evidence not consistent with an end of life premium
- Mixed or inconclusive evidence
8
11
4
35%
48%
17%
Overall finding: quality of life-improving vs. life-extending end of life treatments
- Quality of life improvement preferred
- Life extension preferred
- Mixed or inconclusive evidence
- Not examined / reported
2
1
2
18
9%
4%
9%
78%

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Selected key variables
Variable Evidence consistent with
an end of life premium
Evidence not consistent
with an end of life
premium
Country
- UK
- Europe (non-UK)
- Rest of the world
2
2
4
5
3
3
Method
- DCE or other choice exercise
- Willingness-to-pay
- Other
4
3
2
6
1
4
Possible to express indifference?
- Yes
- No or not reported
6
2
5
6
Visual aids used?
- Yes
- No or not reported
5
3
3
9
Note: some larger studies comprised multiple sub-studies employing different methods – these are
counted multiple times if separate results were reported for each sub-study

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• Overall findings were summarised using three categories:
• Consistent with an end of life premium
• Not consistent with an end of life premium
• Mixed or inconclusive evidence
• Involved subjective judgement
• Rare in stated preference studies to observe a unanimous
preference – there is usually a split in opinion
• Method for determining ‘overall’ preference depends on
the preference elicitation technique used
Difficulty in categorising (1)

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• Majoritarian decision rules are common in politics and
policy making, with most elections/referenda in modern
western democracies being decided by majority rule
• However, such approaches are criticised for failing to
achieve outcomes that represent the views of all sections
of society in a representative manner
• Is the minority view held by a sufficiently large number of
respondents (or held sufficiently strongly) so as to
conclude that the evidence is inconclusive overall?
Difficulty in categorising (2)

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• Hypothetical example of a study that would be
problematic to categorise based on a majority rule:
• Slight (but statistically significant) majority of respondents express
weak support for an end of life premium
• Sizeable minority of respondents strongly disfavour an end of life
premium
• Most studies did not examine strength of preferences at
the individual respondent level
• Normative basis for specifying a measure of average or
overall preference in social choices is unclear
Difficulty in categorising (3)

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• Several researchers reported evidence of heterogeneous
preferences within their own individual studies,
controlling for sample, objective and methodology
• Reflects diversity of the population and range of opinions
within society
• Is it sufficient simply to report a single representative
preference (e.g. that of the median respondent) or is it
important to account for the heterogeneity of views?
Preference heterogeneity

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Extract from my DCE study
“analysis of choice frequencies at the individual respondent
level showed that some respondents appeared to support a
QALY-maximisation type objective throughout; a small
minority always sought to treat those who are worse off
without treatment; but the majority seemed to advocate a
mixture of the two approaches. These heterogeneous
preferences do not appear to be well predicted by
respondents’ observable characteristics.”

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MRC-funded research on societal viewpoints
Some of this material has been taken from
a panel session at the 2018 HTAi Meeting

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i. To identify and describe societal perspectives on the (relative) value of end of life
technologies by eliciting the views of both members of the public and experts in
relevant fields
ii. To develop methods to investigate the distribution of those views, including their
association with other characteristics, in a nationally representative sample of
the UK general public.
Phase 1
Phase 2
Methodological, quantitative
Empirical, qualitative
Q methodology
Survey methods
5,000 online questionnaire respondents
50 experts; 250 public; face to face Q sorts
GCU-led studies

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• Allows unexpected viewpoints to emerge
• Examines strength of preference at the individual level
Q study (phase 1)
Example statements:
• To extend life in a way that is
beneficial to the patient is morally
the right thing to do.
• It is not worthwhile devoting more
and more NHS money to someone
who is going to die soon anyway.
• Treatments that are very costly in
relation to their health benefits
should be withheld.

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Q study (phase 1) - findings
V3: “Valuing wider benefits and opportunity
cost – the quality of life and death”
V1: “A population perspective – value for
money, no special cases”
V2: “Life is precious – valuing life-extension
and patient choice”

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Q-to-survey (phase 2)
Viewpoint Number of
respondents
%
1 1808 37
2 2416 49
3 456 9
MIXED 231 5
TOTAL 4911 100

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Token Brexit reference
Applied to health care
priority setting research,
one perspective might
come out as the most
preferred using one system
of voting/aggregation, but
another perspective would
come out as more
preferred using a different
system

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Can we find a normative basis?

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Possible criteria
Assuming that social choices should be informed by
evidence of people’s preferences, it might be argued that
the method of calculating an average should obey some of
the properties of the democratic systems within which
publicly-funded health care systems are contained:
• It should respect the majority view
• It should give every person’s preferences an equal
weight in the final outcome
• It should ensure representativeness, and therefore
minimise the number whose preferences are
excluded from counting in the voting

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Characteristics of three principal approaches

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Some questions and points for discussion
• From a health economics perspective, is there a case for
applying an end of life premium?
• Are there alternative approaches that would be preferable
to the current binary cut-offs underpinning the NICE
criteria?
• Which methods are best equipped to address these kinds
of questions about social value?
• How should we assess and report the overall preferences
of society?

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To enquire about additional information and analyses, please contact
Koonal Shah at kshah@ohe.org
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