The document summarizes background information on the UK's National Institute for Health and Care Excellence (NICE) taking into account additional factors like burden of illness and wider societal benefits in its appraisal of new health technologies. It discusses NICE receiving over 900 comments on its proposals to incorporate these new factors and its recommendation against changes in the short term. The rest of the document outlines the agenda and issues to be discussed in the workshop, including evidence from stated preference studies on societal values regarding severity of illness, end of life treatments, and proportional shortfall.
1. Koonal Shah
ISPOR 17th Annual European Congress
Workshop W5
10 November 2014
Value-based assessment for NICE
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Some background
•In July 2013, the UK Department of Health asked NICE to take into account additional terms of reference in the appraisal of new health technologies
•Amongst other things, the terms asked NICE to:
•Include a simple system of weighting for burden of illness that appropriately reflects the differential value of treatments for the most serious conditions
•Encompass the differential valuation of treatments designed to extend life at the end of life in the current approach within a new system of burden of illness weights
•Include a proportionate system for taking account of wider societal benefits
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Some background (2)
•NICE developed a set of proposals to incorporate these new terms of reference into its appraisal methods
•e.g. proportional and absolute QALY loss values would be calculated as part of an appraisal and be used as the basis for assessing burden of illness and wider societal impact, respectively
•Proposals were outlined in a consultation paper, open to scrutiny by the public (including patients and patient groups), clinicians, economists and academics, industry and other interested groups
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Some background (3)
•NICE received more than 900 comments from 121 organisations and individuals
•No consensus emerged, with respondents particularly split on the ways in which the burden of illness and wider social impact criteria should be measured and valued
•NICE has recommended to its Board that no changes to the technology appraisal methodology should be made in the short term
•Supplementary policy for the appraisal of life-extending end of life treatments will be retained in its current form
•Nevertheless, many of the issues that we had planned to discuss in this workshop remain relevant
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Agenda for the workshop
•Summary of existing academic research (Koonal Shah, Office of Health Economics)
•Introduction to the consultation paper and demonstration of how to do the QALY shortfall calculations (Anthony Hatswell, BresMed)
•Discussion of some of the issues with the proposed methods (Jeanette Kusel, Costello Medical Consulting)
•Conclusion and summary of NICE’s position (Meindert Boysen, NICE)
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Role of stated preference research
•NICE’s position on social value judgements is that ‘‘advice from NICE to the NHS should embody values that are generally held by the population of the NHS’’ (Rawlins & Culyer, 2004)
•Empirical stated preference studies can provide meaningful information about societal values, as long as the methods used are scientifically defensible
•The public are taxpayers / potential users of the NHS
•In line with NHS’s policy objective of ensuring public involvement in health-care priority setting activities
•Can form part of an “empirical ethics” approach to allocating health care resources
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EEPRU study (Rowen et al.)
•Commissioned by the Department of Health; led by researchers at University of Sheffield
•Web-based discrete choice experiment (n=3,669)
•Design informed by several preparatory studies, including face-to-face interviews with cognitive debriefing
•Pairwise choice tasks with four attributes: life expectancy without treatment; quality of life without treatment; life expectancy gain; quality of life gain
•Respondents were asked which of two hypothetical patients groups they thought should be treated
•Four variants, each with a different level of life expectancy without the condition
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EEPRU study findings
•Authors conclude that:
“The social value of a QALY gain is not equal between recipients, but depends on the prospective burden of illness and whether they are end of life”
•Respondents tended to choose to treat the group with the larger QALY gain, but at a declining rate
•Results suggest some support for burden of illness, though the findings are not consistent across all
•Support for an end of life premium found, though the authors warn about the conceptual overlap between the burden of illness and end of life variables
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Linley & Hughes (2013)
•Web-based survey of 4,118 respondents in Great Britain
•Examined a number of prioritisation criteria relevant to the UK context
•Support reported for prioritising according to severity, unmet need and extent of wider societal benefits
•No support for an end of life premium
Source: Linley & Hughes (2013)
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Severity of illness
•Many studies report that people wish to give high priority to those whose pre-treatment health or prognosis without treatment is poorest (see Shah, 2009)
•Until recently most of the evidence had focused on quality of life
Source: Nord (1993)
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End of life
Study
Country
Sample size
Method
Mode of administration
Findings relevant to end of life
Abel Olsen (2013)
NOR
503
Pairwise choice
Web-based survey
No evidence in support of a premium for life-extending end of life treatments
Baker et al. (2011)
UK
40
Ranking exercise
Focus group
Evidence in support of a premium for life-extending end of life treatments
Linley & Hughes (2013)
UK
4,118
Budget allocation
Web-based survey
No evidence in support of a premium for life-extending end of life treatments
Pennington et al. (2013)
Multiple
17,657
WTP
Web-based survey
Evidence in support of a premium for life-extending end of life treatments
Pinto Prades et al. (2014)
SPA
813
WTP, PTO
CAPI
Evidence in support of a premium for end of life treatments, but QOL-improving end of life treatments were valued more highly than life-extending end of life treatments
Rowen et al. (2014)
UK
3,669
DCE
Web-based survey
Evidence in support of a premium for life-extending end of life treatments, but responses to the follow-up attitudinal questions cast doubt on this finding
Shah et al. (2014)
UK
50
Pairwise choice
Face-to-face interview
Some evidence in support of a premium for end of life treatments, but QOL-improving end of life treatments were valued more highly than life-extending end of life treatments
Shah et al. (2012)
UK
3,969
DCE
Web-based survey
No evidence in support of a premium for life-extending end of life treatments
Skedgel et al. (2014)
CAN
656
DCE
Web-based survey
No evidence in support of a premium for life-extending end of life treatments
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Proportional shortfall and the fair innings argument
•Williams (1997) argued for a fair innings approach to health care prioritisation, which involves reducing differences in lifetime health
•Public support for fair innings has been found in several empirical studies, most recently Abel Olsen (2013)
•Stolk et al. (2005) have proposed the concept of “proportional shortfall”, which produces results that lie somewhere in between goals regarding equity in lifetime and future health
•Evidence regarding proportional shortfall is limited
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Wider societal benefits
•Original DH attempt to account for wider societal benefits involved calculating the net resource contribution of different age, gender and condition (ICD-level) groups
•There is some evidence that people wish to give priority to treatments with substantial non-health benefits, such as reduced need for carers (e.g. Linley & Hughes, 2013)
•However, Claxton et al. (2010) warn of the pitfalls of ignoring the implications of fixed health care budget constraints
•Full consideration of wider societal benefits could lead to the rejection of medicines considered cost-effective by NICE from an NHS perspective (e.g. life-extending treatments for conditions associated with older people)
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Related research
•Claxton et al. (2013) conducted an econometric study on the link between health care expenditure and quality of life with the aim of producing an estimate of what the cost-per-QALY threshold should be
•Programme budget and mortality data at the PCT-level
•Their “best estimate” is ~£13,000 per QALY gained
•Study has been critiqued by OHE (Barnsley et al., 2013)
•Paulden et al. (2014) describe the potential inconsistencies if any value considerations applied to the beneficiaries of new technologies are not also applied to those who bear the opportunity cost