The complexity of funding rare disease research: an IRDiRC assessment of the landscape

Lucia Monaco; Mary Catherine V. Letinturier; Naohiko Aketa; Dimitrios Athanassiou; Jida el Hajjar; Simon Frost; Alicia Granados; Anthony R. Haight; Anneliene H. Jonker; Susan R. Kahn; Sukhun Kang; Persefoni Kritikou; Christina Kyriakopoulou; Christopher McMaster; Samantha Parker; Daniel Scherman; Nivedita Valentine; Samuel Wiafe; Adam L. Hartman

doi:10.20517/rdodj.2024.18

Rare Disease and Orphan Drugs Journal ›› 2024, Vol. 3 ›› Issue (4) :27 DOI: 10.20517/rdodj.2024.18

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The complexity of funding rare disease research: an IRDiRC assessment of the landscape

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¹Independent Researcher, Milano 20144, Italy.

²IRDiRC Scientific Secretariat, French National Institute of Health and Medical Research (Inserm), Ivry-sur-Seine 94200, France.

³Clinical and Translational Research Center, Keio University Hospital, Shinjuku-ku, Tokyo 160-0016, Japan.

⁴World Duchenne Organization, Veenendaal 3905 PG, The Netherlands.

⁵Rare Diseases Greece, Athens, Argyoupolis 16451, Greece.

⁶Canadian Organization of Rare Disorders, Toronto, ON M5S 1S4, Canada.

⁷Tiber Capital Group, Palm Beach, FL 33480, USA.

⁸Sanofi Global, Barcelona 08016, Spain.

⁹Sparx Biopharmaceuticals Corp., Mount Prospect, IL 60056, USA.

¹⁰Children’s Cancer Therapy Development Institute, Hillsboro, OR 97006, USA.

¹¹Health Technology and Services Research, Faculty, Department of Behavioural, Management and Social Sciences, TechMed Centre, University of Twente, Enschede 7522NH, The Netherlands.

¹²Duchenne Parent Project, Veenendaal 3905 PG, The Netherlands.

¹³SRK Consulting, Brookline, MA 02446, USA.

¹⁴Department of Technology Management, University of California Santa Barbara, Santa Barbara, CA 93106, USA.

¹⁵Rare Disease Consulting, Athens 15235, Greece.

¹⁶30 Unit of Health Innovations and Ecosystems, Directorate General for Research and Innovation, European Commission, Brussels 1049, Belgium.

¹⁷Canadian Institutes for Health Research, Halifax, NS B3H 4H7, Canada.

¹⁸ITALFARMACO, Cinisello Balsamo 20092, Italy.

¹⁹Foundation for Rare Diseases/Fondation Maladies Rares, Université Paris Cité, CNRS, Inserm, UTCBS, Paris 75006, France.

²⁰Pharmanovia, Basildon SS14 3FR, UK.

²¹Rare Disease Ghana Initiative, Accra GE155, Ghana.

²²Division of Clinical Research, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Rockville, MD 20852, USA.

Correspondence to: Dr. Adam L. Hartman, Division of Clinical Research, National Institute of Neurological Disorders and Stroke, National Institutes of Health, 6001 Executive Blvd, Rockville, MD 20852, USA. E-mail: adam.hartman@nih.gov

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Abstract

The complexity of the funding landscape for rare diseases (RD) research is due to many factors. Each type of funder has strategic goals guiding its investments. The International Rare Diseases Research Consortium (IRDiRC) convened RD experts to explore contributing factors and investment principles in RD funding. This report includes the Task Force’s findings. Similar to IRDiRC’s previous work on motivating factors for company investments in RD research, the current Task Force found that return on investment (ROI) was a guiding principle. However, within the broader RD funding landscape, the definition of ROI varied between types of funders. While they shared funding requirements (e.g., scientific quality, health economics), funders employed both major investment and venture-type instruments, demonstrating the ongoing need for flexibility in supporting RD funding. These observations warrant further analysis of the interactions and partnerships among all actors of RD research and the sustainability of RD research funding.

Keywords

Rare disease research funding / rare disease advocacy groups / rare disease regulatory issues / venture philanthropy / venture capital / public-private partnerships

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Lucia Monaco, Mary Catherine V. Letinturier, Naohiko Aketa, Dimitrios Athanassiou, Jida el Hajjar, Simon Frost, Alicia Granados, Anthony R. Haight, Anneliene H. Jonker, Susan R. Kahn, Sukhun Kang, Persefoni Kritikou, Christina Kyriakopoulou, Christopher McMaster, Samantha Parker, Daniel Scherman, Nivedita Valentine, Samuel Wiafe, Adam L. Hartman. The complexity of funding rare disease research: an IRDiRC assessment of the landscape. Rare Disease and Orphan Drugs Journal, 2024, 3(4): 27 DOI:10.20517/rdodj.2024.18

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