Koonal K. Shah Office of Health Economics, UK Tony Culyer, Richard Cookson University of York, UK

1. Departing from the health maximisation approachSocial value judgements made by NICE’s advisory committees Koonal K. Shah Office of Health Economics, UK Tony Culyer, Richard Cookson University of York, UK

2. Background • NICE is a decision maker, responsible for providing advice on the use of health care resources in England and Wales • NICE’s advisory bodies are required to take account of clinical and cost effectiveness when reaching decisions about a given intervention • Decisions are guided largely by an approach of maximising health benefit per unit cost • Approximate threshold of £20,000-30,000 per QALY gained

3. Social value judgements • Scientific evidence is not always of good quality and is hardly ever complete • NICE’s advisory bodies therefore need to use their experience to make value judgements beyond the existing evidence Scientific evidence Value judgements + Scientific Social

4. Social value judgements (cont..) • NICE’s position on social value judgements is informed by: • General principles of bioethics • Bioethical considerations of resource allocation and priority setting • Empirical studies of societal attitudes to resource allocation, priority setting and rationing • Deliberations of its ‘Citizens’ Council’

5. Deliberative decision-making process Algorithmic process Deliberative process • Formulaic methods for combining information • ‘Equity weighting’ • Scope for advisory bodies to use their own judgement

6. Sunitinib for the first-line treatment of advanced and/or metastatic renal cell carcinoma (RCC) • Poor prognosis – 5yr survival rate for metastatic RCC is ~10% • No reliable cures for advanced and/or metastatic RCC exist • Sunitinib appears to offer benefits compared with standard immunotherapy in terms of overall survival, progression-free survival and tumour response • Assessment Group ICER: £54,366 per QALY gained SVJ issue: should NICE give greater weight to health gains for patients with terminal illness and short remaining life span?

7. Sunitinib for RCC (cont..) • NICE issued supplementary advice on the appraisal of end-of-life treatments to its advisory committees in Jan 2009 • Committees should consider the impact of giving greater weight to health gains achieved in the later stages of disease when the following criteria are met: • Indicated for patients with short life expectancy • Offers an extension to life • No alternative treatments are available • Licensed for small patient populations • Following application of this advice, the Committee recommended the use of sunitinib

8. Pemetrexed for the treatment of malignant pleural mesothelioma (MPM) • Poor prognosis: median survival is 9-13 months • Treatment aims to maintain quality of life • ~90% of cases of MPM are linked to asbestos exposure • The use of asbestos was banned in the UK in 1999 • Assessment Group ICER: £34,500-37,700 per QALY gained SVJ issue: should NICE give greater weight to health gains accruing to individuals whose condition is partly caused by a third party organisation?

9. Pemetrexed for MPM (cont..) • Cause of disease: the Committee noted in its deliberations that MPM is a rare and aggressive disease caused by occupational exposure to asbestos • Pemetrexed was recommended even though its base case ICER exceeded the range normally considered acceptable • Appeal: it was argued that NICE should assess treatments as they present, irrespective of the circumstances which led to them contracting the condition • The Committee responded that matters relating to the cause of the disease were not determinative to its recommendation

10. Donepezil for the treatment of moderate Alzheimer’s disease • People with Alzheimer’s disease gradually lose the ability to carry out routine daily activities and require the aid of carers • Carers’ quality of life can often be adversely affected by the burden of providing care • Manufacturer model ICER: £31,550 per QALY gained • Assessment Group ICER: £45,000 per QALY gained SVJ issue: how should NICE take into account outcomes for informal carers?

11. Donepezil for Alzheimer’s disease (cont..) • Impact on carers: the Committee considered that an additional utility benefit could be applied to the base case to account for the positive impact on the quality of life of carers • NICE methodology states that evaluations should be conducted from the perspective of the NHS & PSS decision-maker, so the Committee concluded that carer costs should be excluded from the model • Donepezil was recommended even though its base case ICER exceeded the range normally considered acceptable

12. Some thoughts • When formulating guidance, NICE takes into account not only clinical and cost effectiveness information but also a range of social, ethical and practical considerations… • …but the case of end-of-life treatments is the only officially recommended departure from health maximisation approach • There may be a tension between explicit departure from health maximisation versus implicit departure by relaxing scientific value judgements in order to bring the ICER estimate down to the range considered acceptable