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

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Abstract

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.

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Notes

  1. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ L 18, 22.1.2000, p.1), last amended by Regulation (EC) No 596/2009 (OJ L 188, 18.07.2009, p. 14).

  2. See: https://ncats.nih.gov/.

  3. See https://www.nice.org.uk/news/feature/changes-to-nice-drug-appraisals-what-you-need-to-know.

  4. Human rights and health: http://www.who.int/news-room/fact-sheets/detail/human-rights-and-health.

  5. European Commission memo: http://europa.eu/rapid/press-release_MEMO-14-141_en.htm.

  6. See: https://www.orpha.net/consor/cgi-bin/index.php.

  7. The Cramer’s V statistics indicates how strongly two categorical variables are associated (Sheskin, 2003). The statistics is ranging between 0 and 1, the maximum value indicating perfect relationship.

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Acknowledgements

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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Authors and Affiliations

  1. Hospinnomics (Paris School of Economics/Paris Public Hospitals), Fondation Imagine for Rare Diseases, Paris 1 Panthéon-Sorbonne, 1, Parvis Notre-Dame, 75004, Paris, France

    Setti Raïs Ali

  2. Université Catholique de Louvain, Louvain-la-Neuve, Belgium

    Sandy Tubeuf

  3. University of Leeds, Leeds, UK

    Sandy Tubeuf

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  1. Setti Raïs Ali
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Correspondence to Setti Raïs Ali.

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Raïs Ali, S., Tubeuf, S. (In)-Equality in the Allocation of R&D Resources for Rare Diseases. Soc Just Res 32, 277–317 (2019). https://doi.org/10.1007/s11211-019-00332-w

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