The use of social value judgements (SVJs) in health technology assessments is controversial but not new. The latest example of their use raises questions about how they are incorporated consistently into health technology appraisal.
The National Institute for Health and Care Excellence (NICE) recently published a final appraisal document regarding the use of dinutuximab beta as an option for treating high-risk neuroblastoma, a rare type of cancer, primarily affecting children. Neuroblastoma develops from specialised nerve cells left behind after a baby’s development in the womb. Patients with high-risk neuroblastoma tend to have a very poor long-term prognosis, and existing treatments are painful and can have long-lasting side effects.
Modelled trials indicate that, compared to the current treatment option of isotretinoin, dinutuximab beta increases survival (by between three and five years) and the length of time before the disease progresses. Despite these benefits, the incremental cost-effectiveness ratio (ICER) estimates presented were greater than £40,000 per quality-adjusted life year (QALY) gained – above the range usually considered a cost-effective use of NHS resources.
Nevertheless, NICE has recommended the use of dinutuximab beta (under certain circumstances). In addition to the cost-effectiveness evidence, the appraisal committee took into account “the uncaptured health-related benefits, the rarity and severity of the disease, and the potential lifetime benefit for children with neuroblastoma” (p.2). The guidance document indicates that the committee also noted the impact that the disease can have on carers and family members. This can include health-related effects such as anxiety and stress, and non-health-related effects such as disruption to working life.
NICE’s decision is likely to raise eyebrows amongst health economics commentators. One blog post noted that rarity and severity had tipped the balance in this appraisal, provocatively questioning whether this represented the “thin end of the wedge”. Yet NICE has been applying such social value judgements (SVJs) to its decision-making for many years. A paper in Health Economics, Policy and Law published in 2013 – a collaboration between OHE, University of York, and King’s College London – described several case-specific SVJs as reported in NICE guidance. This included the appraisal of riluzole for motor neurone disease in 2001, where consideration of the “severity and relatively short lifespan” of affected patients led to the technology being recommended, despite the ICER estimates being higher than the range normally considered acceptable.
Indeed, riluzole was one of several examples of how ‘special circumstances’ are sometimes considered in appraisals of high-ICER technologies, as highlighted by Sir Michael Rawlins (former OHE Research and Policy Committee member and then-Chair of NICE) and colleagues in an article on NICE’s approach to decision-making. The special circumstances were: severity, end of life, stakeholder persuasion, significant innovation, disadvantaged population and children. At least two of these circumstances can be argued to have applied in the case of dinutuximab beta.
The draft guidance document states that the committee was prepared “to be flexible in its decision-making given the rarity and severity of the disease” (p.20). This has the potential to be controversial. While there is widespread evidence of societal support for prioritising treatment of the severely ill, the evidence on preferences for prioritising treatments for rare diseases is less clear-cut. NICE’s own Citizens Council (a panel of members of the public, established to provide advice about the social values that NICE should adopt in preparing its guidance) judged that rarity alone is insufficient for accepting high ICERs – factors such as severity, the extent of health gain, and whether the condition is life-threatening should also be considered.
A potential issue with NICE’s approach is that it relies on a deliberative process, and it is not always clear whether factors such as severity are being considered consistently across different appraisals. It has been contended that structured decision-making techniques such as multi-criteria decision analysis may be helpful, as they involve a systematic approach to selecting and attaching weights to different decision criteria.
It is worth noting that NICE’s policy on the appraisal of life-extending end of life treatments – the only policy which asks appraisal committees to depart systematically from the reference case position that all QALYs should be valued equally – was not applicable in this appraisal. While dinutuximab beta met the ‘substantial extension to life’ criterion, the life expectancy for patients without the new treatment was estimated to be 31 to 34 years, so it did not meet the ‘short life expectancy’ criterion.
NICE’s approach to applying SVJs continues to develop over time, and this appraisal highlights many of the factors and trade-offs that its appraisal committees are expected to consider when judging whether a new treatment represents value for money for the NHS.
Shah, K.K., Tsuchiya, A. and Wailoo, A.J., 2018. Valuing health at the end of life: A review of stated preference studies in the social sciences literature. Social Science & Medicine, 204, pp.39-50. DOI | RePEc
Towse, A. and Garau, M., 2018. Appraising ultra-orphan drugs: is cost-per QALY appropriate? A review of the evidence. OHE Consulting Report. London: Office of Health Economics. RePEc
Posted in Health Technology Assessment, NICE | Tagged Commentary