小瓢虫传媒

(In)-equality of opportunity in the allocation of R&D resources for rare diseases

Sandy Tubeuf, UCLouvain an University of Leeds

This paper investigates the existence of inequalities of opportunity in the allocation of R&D resources within rare diseases and identifies the characteristics of rare diseases that appear to lead R&D investments. Rare diseases affect less than 1 in 2,000 citizens. With over 7,000 recognized rare diseases and 350 million people affected worldwide, rare diseases are not so rare when considered collectively. Rare diseases are generally underserved by drug development because pharmaceutical industries consider R&D investments in rare diseases too costly and risky in comparison with the low expected returns due to the small population involved. We use data on rare diseases research from Orphanet and academic publications from MEDLINE and test the existence of inequalities using dominance tools and bilateral tests. We show that rare diseases in children and with a smaller prevalence, such as ultra-rare diseases, are underserved by R&D. R&D efforts appear to be concentrated in more profitable research areas with potentially larger sample size for trials design and adult population.

(Joint paper with聽Setti Ra茂s Ali1, Paris I)