Abstrakt
There is an increasing need to manage cost-effectiveness issues of novel or relatively expensive technologies that are currently in use or being proposed for the treatment of cystic fibrosis (CF) (e.g. cystic fibrosis transmembrane conductance regulator (CFTR) modulation therapy). Health-economic evaluations of rising pharmacotherapeutic costs, as the major driver of overall cost, have to be part of the cost analysis of chronic and progressive (rare) diseases like CF that may require lifelong therapy.
Total costs include not only direct healthcare costs but also the cost of lost productivity by both patients and family caregivers. When considering the results of cost-effectiveness analysis of new technologies associated with the management of CF, it is unreasonable to expect that the incremental cost-effectiveness ratio to be less than the generally applied thresholds (willingness to pay) for other common diseases.
Therefore, when assessing CF and other rare diseases, such analyses should include complex health technology assessment approaches, which evaluate comparative treatment effectiveness (novel and established), as well as wider social benefits and ethical aspects.