Orphan drugs (ODs) manufacturers are faced with several barriers which make the commercial success of their products challenging. The investment required for the research and development of ODs is typically similar as for non-orphan drugs, however, the number of patients who require treatment is lower. ODs often carry price tags much higher than non-orphan counterparts, generating decision makers’ concern over their potential budget impact. Furthermore, ODs may not always be able to follow the conventional assessment criteria used in health technology appraisals (HTAs). This is especially the case in HTAs using cost-effectiveness analysis, which are primarily based on quantifying the direct health benefits of new drugs and have traditionally failed to capture the holistic value of ODs for rare diseases, such as the intrinsic value of providing new treatments for rare and often severe diseases.
With the goal of exploring how decision makers in England value disease rarity, we undertook an analysis to determine if there is any correlation between disease rarity and pricing outcomes of ODs. In this PharmaTimes article, we specifically focused on ODs for chronic conditions as their potential for significant and long-term budget impact typically attracts attention from decision makers.