Do ultra-orphan medicinal products warrant ultra-high prices? A review

Eline Picavet,¹ David Cassiman,² Steven Simoens<sup>1

1</sup>Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium; ²Department of Hepatology, University Hospital Leuven, Leuven, Belgium

Abstract: Ultra-orphan medicinal products (ultra-OMPs) are intended for the treatment, prevention, or diagnosis of ultra-rare diseases, ie, life-threatening or chronically debilitating diseases that affect less than one per 50,000 individuals. Recently, high prices for ultra-OMPs have given rise to debate on the sustainability and justification of these prices. The aim of this article is to review the international scientific literature on the pricing of ultra-OMPs and to provide an overview of the current knowledge on the drivers of ultra-OMP pricing. The pricing process of ultra-OMPs is a complex and nontransparent issue. Evidence in the literature seems to indicate that ultra-OMPs are priced according to rarity and what the manufacturer believes the market will bear. Additionally, there appears to be a trend between the price of an ultra-OMP and the number of available alternatives. Patients, third-party payers, and pharmaceutical companies could benefit from more transparent pricing strategies. With a view to containing health care costs, it is likely that cost-sharing strategies, such as performance-based risk sharing arrangements, will become increasingly more important. However, it is vital that any measures for price control are consistent with the intended goals of the incentives to promote the development of new OMPs. Ideally, a balance must be struck between attaining affordable prices for ultra-OMPs and securing a realistic return on investment for the pharmaceutical industry.

Keywords: ultra-orphan medicinal product, ultra-rare disease, pricing