Social Justice Research

, Volume 32, Issue 3, pp 277–317 | Cite as

(In)-Equality in the Allocation of R&D Resources for Rare Diseases

  • Setti Raïs AliEmail author
  • Sandy Tubeuf


This paper analyses the allocation of R&D investments within rare diseases and identifies the characteristics of rare diseases that appear to lead R&D resources. Rare diseases affect less than 1 in 2000 citizens. With over 7000 recognised 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 along with academic publications per rare disease from bibliographic databases. We test the existence of inequalities in R&D investments within rare diseases and identify the disease characteristics that appear to lead R&D investments 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 investments appear to be concentrated in more profitable research areas with potentially larger sample size and adult population.


R&D Inequality FOSD Rare diseases Orphan drug 

JEL Classification

I11 I14 I18 L65 



Authors are grateful to Christine Le Clainche, Clémence Thébaut, Aki Tsuchiya, the EuHEA 2018 conference participants and colleagues from the Economics School Louvain seminar for their useful comments and suggestions. We also received detailed and useful reports from two reviewers and the editors of this special issue that substantially helped us improve the paper. Any remaining errors are our responsibility.


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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2019

Authors and Affiliations

  1. 1.Hospinnomics (Paris School of Economics/Paris Public Hospitals), Fondation Imagine for Rare DiseasesParis 1 Panthéon-SorbonneParisFrance
  2. 2.Université Catholique de LouvainLouvain-la-NeuveBelgium
  3. 3.University of LeedsLeedsUK

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