Health technology assessment (HTA) and economic evaluation: efficiency or fairness first.

The economic evaluation which supports Health Technology Assessment (HTA) should inform policy makers of the value to society conferred by a given allocation of resources. However, neither the theory nor practise of economic evaluation satisfactorily reflect social values. Both are primarily concerned with efficiency, commonly conceptualised as the maximisation of utility or quality adjusted life years (QALYs). The focus is upon the service and the benefits obtained from it. This has resulted in an evaluation methodology which discriminates against groups and treatments which the population would like to prioritise. This includes high cost treatments for patients with rare diseases. In contrast with prevailing methods, there is increasing evidence that the public would prefer a fairness-focused framework in which the service was removed from centre stage and replaced by the patient. However methods for achieving fairness are ad hoc and under-developed. The article initially reviews the theory of economic evaluation and argues that its focus upon individual utility and efficiency as defined by the theory omits potentially important social values. Some empirical evidence relating to population values is presented and four studies by the first author are reviewed. These indicate that when people adopt the social perspective of a citizen they have a preference for sharing the health budget in a way which does not exclude patients who require services that are not cost effective, such as orphan medicinal products (OMP's) and treatments for patients with ultra-rare diseases (URD's).