presentation by paula lorgelly - beyond qalys: a quantum leap forward or a leap in the dark?

Beyond QALYs

A quantum leap forward or a leap in the dark?

Assoc Prof Paula Lorgelly

Deputy Director

Future of Value: Beyond QALYs

Professional background

• Experience in both UK and Australia health care systems

• (rigorous/rigid) HTA environments

• Member of PBAC ESC

• Cost effectiveness thresholds

• Societal willingness to pay or opportunity cost of benefit forgone

• Budget impact assessment/constraints

• So much I could discuss ….


Value judgements

• When assessing value for money, ask

• What constitutes benefit?

• How useful is it for informing decision making?

• Welfarist approach, monetary metric of benefit1

• Transport, energy, environment …

• But issues of applying contingent valuation methodology to measure benefits of health interventions

• Extra-welfarist approach, use non-utility information in decision making, target health instead of utility1


Health and QALYs

• Measure health or health related quality of life (HRQoL) using multi-attribute utility instruments (MAUIs)2

• EQ-5D, SF-36/6D, HUI, QWB

• Use societal preferences to convert reported health profiles into values which we then use to estimate QALYs given time in a health state

• QALYs offer a common metric for comparisons across diseases

• Also (established) cost effectiveness thresholds

• But the QALY criticisms are many3


Non health or beyond health

• Once the focus was on survival (overall or progression free) and life extension

• Then came an interest in quality of life

• Medical intervention today goes well beyond health

• Genetic tests which offer information/reassurance4

• End-of-life care, palliative care, ‘a good death’5

• Increasing role of caregivers/carers and informal care6

• Add to this the growing need to evaluate public health/social care (cross-sectional) interventions7


How do we assess value?

• Could use (and we do use) QALYs, but

• Most MAUIs focus on health related quality of life which is generally generic

• To go beyond QALYs one could have8,9

• Better measure of health, disease/condition-specific, EQ-5D bolt-ons10

• Broader measure of benefit

• Societal perspective, e.g. productivity losses and carer effects


Capability Approach

• Amartya Sen awarded the Nobel prize in economics, 199811

• When assessing quality of life, the object of the assessment should be people’s capabilities, intended as the real freedom that people have to live the life they value

• A crucial normative argument that quality of life should not be measured as opulence or utility and should not be assessed using people’s preferences or desires but should concern people’s capabilities

• the abilities to achieve those ‘beings and doings’ that people have reason to value in life12


Operationalising the CA

• Early work by Sen on the Human Development Index

• Wide adoption in development and gender studies

• Informed Tony Culyer’s contribution to the progress of extra-welfarism and the subsequent development of the QALY framework13

• Most recently operationalised as an outcome measure14

• ICECAP suite of instruments

• ASCOT

• OCAP, OCAP-18, OxCAP-MH

• Targeted instrument development


ICECAP suite of instruments15

• ICECAP-O for older people

• ICECAP-A for adults

• ICECAP-SCM for end of life care

• Additionally, Carers Experience Scale (CES)

ICECAP-A

ICECAP-SCM


ASCOT instruments16

• Social Care Related Quality of Life, SCRQoL

• Number of instruments for use in different settings

User SCRQoL Domain Definition

Control over daily life The service user can choose what to do and when to do it, having control over his/her daily life and activities

Personal cleanliness and comfort The service user feels he/she is personally clean and comfortable and looks presentable or, at best, is dressed and groomed in a way that reflects his/her personal preferences

Food and drink The service user feels he/she has a nutritious, varied and culturally appropriate diet with enough food and drink he/she enjoys at regular and timely intervals

Personal safety The service user feels safe and secure. This means being free from fear of abuse, falling or other physical harm and fear of being attacked or robbed

Social participation and involvement The service user is content with their social situation, where social situation is taken to mean the sustenance of meaningful relationships with friends and family, and feeling involved or part of a community, should this be important to the service user

Occupation The service user is sufficiently occupied in a range of meaningful activities whether it be formal employment, unpaid work, caring for others or leisure activities

Accommodation cleanliness and comfort The service user feels their home environment, including all the rooms, is clean and comfortable

Dignity The negative and positive psychological impact of support and care on the service user's personal sense of significance

ASCOT


Capability values

• Both ICECAP and ASCOT employ best worst scaling, BWS

• Authors argue choice approach elicits values for capability/social care states, not preferences

• ICECAP values range from 1 full capability to 0 no capability

• Not anchored on dead so cannot be used in a QALY

• ASCOT values range from 1 ideal SCRQoL to –0.19, where 0 is dead (used a time trade off approach)

• ASCOT can be used in a QALY context – social care QALYs


Capability in HTA





Example – ADHD

• Attention-deficit/hyperactivity disorder (ADHD) can be managed by helping children to develop concentration and social skills, but in some case it requires pharmacotherapy

• Lisdexamfetamine found to improve symptoms (ADHD-RS-IV) and result in weight loss

• Considerable non-health benefits, evidence suggests ADHD results in poor educational performance and increased criminal activity

• A capability instrument could provide an estimate of how treating/managing children with ADHD improves their wellbeing, beyond just health improvements


Example – diagnostic tests

• Diagnostic tests provide information primarily to inform a treatment strategy which offers health benefits

• But the information itself can be valuable to patients, it might change behaviour and offer empowerment

• If the diagnostic is a genetic test, may offer additional benefits to relatives, so by extension valuable to the patient’s overall wellbeing


Subjective Wellbeing

• Traditional welfarist view of the world where individuals are regarded as the best judge of their own conditions, and the aim of public policy is to maximise the sum of everybody’s happiness (or utility)18

• Different measures19

• experienced happiness

• evaluation of life, or life satisfaction

• However, evidence that life satisfaction and happiness are not measuring the same concept20

• None-the-less attractive as they are relatively easy to collect


OECD21


SWB in HTA





Examples – limited

• HTA applications are non-existent

• But we know Bhutan is happy!

• Wellbeing Valuation Approach, wellbeing is valued using data on individual’s SWB from large-scale surveys to assess how non-market goods or life events such as unemployment or illness impact on people’s life satisfaction22


Decision makers beyond QALYs

• NICE social care guidance, states that when measuring and valuing effects “‘QALYs or ‘social care QALYs’ with parallel evaluation based on capability measures where an intervention results in both capability and health or social care outcomes’’ can be used, and that the ‘‘ASCOT instruments may be used as measures of social care quality of life and ICECAP instruments may be used to measure capability’’

• Recent Dutch adoption of capability measures (ICECAP) in their HTA guidance


Quantum leap?

• Payne and Thompson neatly stated that the “availability of other approaches to value benefit is a necessary but not sufficient requirement for moving beyond the QALY. It is not controversial to suggest that the alternatives to the QALY should only be used if they offer sufficient improvements to the existing valuation metric’’22

• Limitations remain: What does the incremental cost per unit of capability/SWB mean?


To conclude

• Health gain is the most important driver (in the health care system)

• But capturing benefit of a therapy has to go beyond a simple measure of health gain

• Important to capture and evaluate the overall benefit for the patient and/or society


Questions?Comments?

Controversies?


References

1. Brouwer & Koopmanschap. On the economic foundations of CEA. Ladies and gentlemen, take your positions! Journal of Health Economics. 2000; 19: 439-59.

2. Richardson et al. Review and critique of health related multi attribute utility instruments. Monash University, Business and Economics, Centre for Health Economics, 2011.

3. Mehrez & Gafni. Quality-adjusted life years, utility theory, and healthy-years equivalents. Medical Decision Making. 1989; 9(2): 142-149.4. Grosse SD, Wordsworth S, Payne K. Economic methods for valuing the outcomes of genetic testing: beyond cost-effectiveness analysis.

Genetics in Medicine. 2008 Sep 1;10(9):648-54.5. Round (ed). Care at the End of Life: an economic perspective. Springer: 2016.6. Van Exel et al. The invisible hands made visible: recognizing the value of informal care in healthcare decision-making. Expert Review of

Pharmacoeconomics & Outcomes Research. 2008; 8(6): 557-61.7. Lorgelly et al. Outcome measurement in economic evaluations of public health interventions: a role for the capability approach?.

International Journal of Environmental Research and Public Health. 2010; 7(5): 2274-89.8. Payne K, J Thompson A. Economics of pharmacogenomics: rethinking beyond QALYs?. Current Pharmacogenomics and Personalized

Medicine (Formerly Current Pharmacogenomics). 2013 Sep 1;11(3):187-95.9. http://www.mrc.ac.uk/funding/how-we-fund-research/highlight-notices/improving-cross-sector-comparisons-beyond-qaly/10. Yang et al. An exploratory study to test the impact on three "bolt-On" items to the EQ-5D. Value in Health, 2015; 18(1): 52-60.11. Sen. 1985. Commodities and Capabilities, Elsevier: New York.12. Sen. 1993. Capability and well-being. In: Nussbaum, MC, Sen A (eds.), The Quality of Life. Claredon Press: Oxford.13. Coast, Smith, Lorgelly. Welfarism, extra-welfarism and capability: the spread of ideas in health economics. Social science & medicine. 2008

Oct 31;67(7):1190-8.14. Culyer. 1990. Commodities, characteristics of commodities, characteristics of people, utilities, and the quality of life, Routledge: London.15. Lorgelly. Choice of Outcome Measure in an Economic Evaluation: A Potential Role for the Capability Approach. PharmacoEconomics. 2015

Aug 1;33(8):849-55.16. http://www.birmingham.ac.uk/research/activity/mds/projects/HaPS/HE/ICECAP/index.aspx17. http://www.pssru.ac.uk/ascot/18. Stiglitz. 2009. Progress, what progress. OECD Observer 272.19. Dolan et al. 2008. Do we really know what makes us happy? A review of the economic literature on the factors associated with subjective

well-being. Journal of Economic Psychology 29: 94–12220. Kahneman & Deaton. 2010. High income improves evaluation of life but not emotional well-being. Proceedings of the National Academy of

Sciences 107: 16489–16493.21. OECD. 2013. OECD Guidelines on Measuring Subjective Well-being.22. Fujiwara. 2013. A General Method for Valuing Non-market Goods Using Wellbeing Data: Three-stage Wellbeing Valuation, Centre for

Economic Performance: London School of Economics and Political Science.23. Payne & Thompson. Economics of pharmacogenomics: rethinking beyond QALYs? Curr Pharmacogenomics Pers Med. 2013;11(3):187–95.

http://www.birmingham.ac.uk/research/activity/mds/projects/HaPS/HE/ICECAP/index.aspx

http://www.pssru.ac.uk/ascot/

presentation by paula lorgelly - beyond qalys: a quantum leap forward or a leap in the dark?

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