Here in the UK, NICE sometimes advises against the provision of particular drugs, by the NHS, on the grounds that evidence does not indicate them to be cost-effective. In some cases it appears that these ‘rejections’ are the result of insufficient data rather than comprehensive data against the use of the drug. On occasion the Department of Health has followed such decisions with a trial-in-practice patient access scheme; what are known as Risk Sharing Schemes. These allow for the provision of the drug, following an agreement with the manufacturer, and the possibility for evidence development.
One well-publicised risk sharing scheme is the Multiple Sclerosis Risk Sharing Scheme. The outcomes of this trial-in-practice remain uncertain, but the scheme has been described as a “costly failure“. In a study of participant data, a recent article demonstrated that the likelihood of individuals being offered treatment, as a part of the MS Risk Sharing Scheme, was positively related to their socio-economic status. This raises questions about the value of the results from a ‘trial-in-practice’ such as this.
Rationing and deprivation
That individuals’ deprivation levels or economic status can be a determinant of prescribing decisions is a well-documented phenomenon. Evidence exists in relation to treatment for colorectal cancer, glaucoma, lung cancer and the prescription of statins and antidementia drugs. Health economists often suppose that the most deprived individuals are also those most in need of health care, but the evidence from all the studies mentioned above highlights deprivation level as being a negative predictor of the levels of care received. In some cases there is good reason for this; those who are more deprived can sometimes tend to present later when care would be less effective, and there may also be relevant issues surrounding health literacy. However, in other cases such an explanation is not so obvious.
Experimental evidence
Trudy Owens and co’s study shows that risk sharing schemes can demonstrate similar characteristics, which I believe to be a point of concern. I would suggest that such schemes as the MS Risk Sharing Scheme can only be justified if they seek to produce experimental evidence of the cost-effectiveness of the intervention. Without this they will be little more than a back door means of provision for drugs that have not been demonstrated to be cost-effective. It seems obvious to me, therefore, that this research and experimental evidence, and the schemes themselves, must conform to a good study design. One would not tolerate a study that allowed practitioners to pick and choose individuals for treatment based on their subjective expectation of success. While this may contribute to the manufacturer’s aims of finding a cost-effective use of their drug it is hardly good science. If such a drug were accepted on to formularies, would doctors continue to (inadvertently or otherwise) discriminate based on deprivation status? Quite possibly, but we’d certainly highlight this as a problematic issue and it would be one reinforced by the poor quality trial-in-practice of the risk sharing scheme.
While some papers offer advice on the design and administration of risk sharing and evidence development schemes, there appear to be no studies addressing the problems caused by discrimination and rationing. It seems to me that there is a substantial gap in the research if we are to prevent risk sharing schemes becoming publicly-funded bad science.
Do you see value in risk sharing schemes? Are they likely to be more representative of practice than randomised trials? Is deprivation a reasonable basis for rationing?
