Last week saw coverage by the media of NICE recommendation of two new drugs to treat breast cancer – palbociclib and ribociclib. These are a new class of small molecule drugs, which inhibit DNA synthesis and hence inhibit tumour growth. Both are tablets, and are indicated for use in previously untreated breast cancer, before it has progressed; the data show a significant increase in progression-free survival of around 10 months – that is, a significant delay in patients needing chemotherapy, with its associated decrease in quality of life.
One of the arguments put forward in support of these drugs is that this extra time before disease progression is highly valued by patients, who are able to lead an almost normal life during this period – and that this value is under-estimated in the appraisal. The additional time gained in a relatively ‘well’ state of health is accounted for in the cost effectiveness analysis, but does not attract a weighting to reflect a particularly high value attached to this extra time. NICE acknowledged that current measures of health are a point-in-time snapshot, and do not capture the value of avoiding future events.
It is not unreasonable that these months of relative ‘wellness’ in a disease with inevitable progression, should be highly valued. It is also not the first time we have encountered this argument; our exploration of patient and clinician input into funding decisions for Scotland identified the same rationale for placing a high value on delaying disease progression – both in cancer examples, and other progressive diseases such as polycystic kidney disease (delay of dialysis) and Duchenne muscular dystrophy (extending ambulant life). Further, there is a precedent for valuing health improvement differently in certain phases of a disease: the End of Life considerations, largely applied to cancer drugs, which weight the last months of life as having particular value.
The question of the extent of preference for extending a period of wellness, is an empirical one, although not trivial to do, as we have seen with the equivocal results of studies to establish the added value of life extension at the end of life. Beyond the challenges of study design, though, the idea of attaching special value to the pre-progression state raises a series of broader questions. Clearly, the same logic would apply as much to other progressive diseases as to cancer; for example, in early Alzheimer’s where patients may be asymptomatic. Irrespective of the disease, it would seem that the same argument could apply anywhere along the disease pathway – if the time at the end of life, and before first progression, are uniquely valuable, is the same not true of a second (or subsequent) stage of progression? In which case, we would be giving special value to health gain in progressive disease overall – again, perhaps not unreasonable, but yet to be validated empirically.
A further major question is the perennial one: whose values count? Currently, our valuations of health states represent society’s view, and are obtained from studies among the general public. This reflects the public’s role as the funder of healthcare through taxation, and as potential beneficiaries of (as yet unknown) care, and is consistent with theories of social justice proposed by philosophers such as Rawls; he proposes the notion of a ‘veil of ignorance’ from which members of society can judge what is ‘fair’ independently of vested interest. The approach does, however, assume that the public are well-informed. It may be that, having not experienced a diagnosis of a progressive disease, members of the public do not attach the same high value to a pre-progression state as patients do – specifically, that members of the public are not prepared to sacrifice other health in order to prioritise such treatments. To resolve such conflicts of preferences, studies of social values may need to incorporate a fuller exposition of patients’ experience, to move us towards a consensus value that is fair to all. CASMI would welcome the opportunity to develop a substantive research project on this much-debated topic – perhaps in conjunction with a consortium of interested patient organisations?
– Dr Liz Morrell