Nermina Ferizovic | Health Economics Expert

In order to gain a positive reimbursement decision, manufacturers are required to highlight the cost-effectiveness of their proposed intervention to reimbursement agencies. That is, interventions are assessed against the cost per unit of health benefit they provide. In many countries, this health benefit is defined by the quality-adjusted life year (QALY) where the quality of life over a particular time period is calculated. However, it is also possible that these interventions are assessed using more ‘natural’ units of health benefits. For instance, in the case of cardiac interventions, this may be the reduction in blood pressure. The cost per unit health benefit is then commonly compared against an arbitrary monetary threshold and if the intervention falls below this threshold, the treatment is granted a positive reimbursement decision.

In rare diseases and in oncology, additional criteria may be used by reimbursement bodies,  such as in conditions where there is a high burden of disease or the treatment provides end-of-life benefits. As such, exceptions are made to the rule described above where interventions are assessed against an arbitrary threshold.

However, in cases where exceptions are made, it can be argued that this reduces the value or the impact of the methodologies used, namely the cost per unit health benefit. Thus, what value claiming that a methodology should be used and that it applies to all interventions when exceptions are made? On the other hand, it could be argued that assessing interventions for their cost-effectiveness only does not allow their full benefit to be highlighted. Examples of this are rife in the rare disease space where there is often a lack of understanding of the disease natural history and small patient numbers in which to collect data. This may be because it is not clear which disease milestones should be used as outcomes or current quality of life tools do not capture the full benefit of treatment. This means that neither the QALY nor any natural units capture the full benefit of the treatment leading to the ‘incorrect’ cost-effectiveness being calculated. This often leads to non-cost-effective interventions and increased difficulty in gaining reimbursement. However, these treatments may provide immense benefit to patients which simply cannot be quantified. Whist rare diseases offer good examples here, problems such as these are not confined to rare diseases. 

As such, is it appropriate that interventions be assessed using cost-effectiveness? If not, which other economic methods should be used? Or should economics accept that its methods are limited by the ‘known’ science and account for this in analyses?