Pharmacogenetics
The allocation of resources
19 Both public and private providers of healthcare operate on limited budgets. In addition to the traditional requirements of quality, efficacy and safety for the regulatory approval of new medicines, public policy in many countries is developing the requirement to assess medicines for their cost-effectiveness. That is to say, the question is not simply whether the medicine has its intended effects and is safe when it is administered, but also whether the cost of the medicine represents good value for money, given the health benefits that it is expected to produce.
20 There are a number of approaches that could be taken by such bodies in determining whether to approve the use of a particular medicine. One approach would be to rely solely on the criterion of cost-effectiveness: to maximise the amount of benefit for the population as a whole, for any given level of expenditure. As has often been pointed out, however, such an approach risks ignoring considerations of justice or equity.5 On this view, it is not the total increase in health which is important, but the fair distribution of that benefit among the members of a population. Unless such considerations are set alongside those of costeffectiveness, those suffering from rare conditions may be overlooked in the allocation of resources because their numbers are not large enough to count against the more prevalent conditions. In liberal democratic societies there is a widespread sense of justice which includes the belief that everyone is owed a certain minimum entitlement, no matter how small the minority to which they might belong. These entitlements include access to health services relevant to the illnesses from which they are suffering. Hence, it may well be right to allocate resources to the treatment of those suffering from a rare condition, even if this means that these resources are less productive of overall benefit. We endorse the approach taken by the National Institute of Clinical Excellence (NICE) of reviewing cases on an individual basis, not applying thresholds, and incorporating considerations of both equity and cost-effectiveness (paragraph 4.21).
Footnotes5 Campbell A (2003) Nice or nasty? Threats to justice from an emphasis upon effectiveness, in International Perspectives in Equity and Health: As Seen from the UK, A Oliver, Editor (London: Nuffield Trust), pp. 35-9; Daniels N (1985) Just Health Care (Cambridge: Cambridge University Press); Doyal L and Gough I (1991) A Theory of Human Need (Basingstoke: Macmillan).