Orphan drugs and the NHS

Editor—When it comes to disease and illness, of course we should not value rarity.¹ It certainly does not matter to those with rare disorders that they have a rare disease. What matters is how much the treatments cost.

The real question is how much we should value cost and cost effectiveness. Cost clearly matters to some degree, and we should be aware of whether NHS resources are being used effectively, but is cost effectiveness everything?

McCabe et al consider two groups of people with similar diseases (J and K).¹ Disease J is rare and treatment expensive, disease K is common and treatment cheaper. They say that to argue for funding treatment for those with J is to value the health gain of the few over that of the many. But why is health gain the important factor for a fair outcome? Is it fair that people are judged only in terms of how cost effective their health gain is?

What matters might be that the people themselves are treated equally (rather than their potential for health gain). Perhaps it matters that people's healthcare needs are met, that they have equal access to care, or that they are given the same chance to lead normal lives.

It helps when the example is constructed so that there are no differences between the two conditions and only two are being considered. If two such diseases with identical patients existed the authors might be correct. But funding decisions are more often made in the context of a range of services with broader considerations.

Those who think that the question of funding for expensive treatments (as opposed to rare ones) is difficult are not convinced that justice in health care is simply to be understood in terms of health gains.