Improving estimate of cost/effectiveness of drugs for rare diseases

Abstract

Background/Aim. Incremental cost/effectiveness ratio (ICER) of many drugs for rare diseases is often much higher that the accepted cost/effectiveness threshold for reimbursement, primarily due to their extremely high prices, raising the question of their availability. The aim of this article was to review necessary adjustments of methods used for cost/effectiveness analysis of drugs for rare diseases. Methods. This article is a narrative review of methods for adjusting cost/effectiveness analysis of drugs for rare diseases in order to get more realistic estimate of ICER threshold, which is essential information for decision-makers. Results. Inputs in cost/effectiveness analysis of a drug for rare diseases should be adjusted by changing discount rates, estimating utilities in a more precise way, excluding treatment-unrelated costs, calculating local C/E threshold, and most importantly, by negotiating drug price until the C/E threshold is not surpassed. With intensified adjusted cost/effectiveness research within the area, many uncertainties will be ended, and real-life value of many of the drugs for rare diseases will be known, influencing pricing in a sustainable direction. Conclusion. With the adjustments, the true cost/effectiveness of a drug for rare disease will be approached, enabling evidence-based and completely transparent reimbursement decisions.