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

Abstract

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).

Keywords: Economic evaluation; communitarianism; cost/QALY; sharing; social preferences; ultra-rare diseases.

Figure 1.

Results from sharing survey 1 (n = 501). Source: Richardson et al. with permission from Elsevier [70]

Figure 2.

Visual aid for sharing study 4^(1)(2). (1) The shaded area indicates the ‘health’ obtained by the two groups. The vertical axis measures utility and the horizontal axis the number of patients affected. In this figure the 5 high cost patients have full health; the 600 low cost patients have a utility of 0.67. These could be changed by moving the slider at the base of the figure. The figure is to scale. The blue area therefore measures QALYs, although it was referred to as ‘health’ by the avatar.(2) Illness A was experienced by patients in Group 2; Illness B by those in Group 1.Source: Richardson et al [72]

Figure 3.

Results from sharing survey 4: QoL purchased for high cost patients A, ^(1). (1) By price of insurance and size of the low cost group B, whose QoL falls to meet the budget.Source: Richardson et al [72]