The complexity of funding rare disease research: an IRDiRC assessment of the landscape
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.
Authors
- Christopher McMaster (ORCID: https://orcid.org/0000-0003-2432-5451)
- Daniel Scherman (ORCID: https://orcid.org/0000-0003-1207-1298)
- Persefoni Kritikou
- Samantha Parker
- Anneliene Hechtelt Jonker (ORCID: https://orcid.org/0000-0001-5883-7610)
- Samuel Wiafe (ORCID: https://orcid.org/0000-0003-2439-7631)
- Susan R. Kahn (ORCID: https://orcid.org/0000-0002-5667-8916)
- Naohiko Aketa (ORCID: https://orcid.org/0000-0002-0188-1255)
- Adam L. Hartman (ORCID: https://orcid.org/0000-0001-5672-3409)
- Simon Frost
- Christina Kyriakopoulou
- Mary Catherine V. Letinturier (ORCID: https://orcid.org/0000-0003-4387-6312)
- Alı́cia Granados (ORCID: https://orcid.org/0000-0003-0431-400X)
- Lucía Monaco (ORCID: https://orcid.org/0000-0001-5620-1790)
- Dimitrios Athanassiou
- Anthony R. Haight
- Jida el Hajjar
- Nivedita Valentine
- Sukhun Kang
Institutions
- National Institute of Neurological Disorders and Stroke (US)
Publication Details
- Journal
- Rare Disease and Orphan Drugs Journal
- Published
- 2024-09-09
- DOI
- https://doi.org/10.20517/rdodj.2024.18
- Citations
- 7
- Primary Topic
- Health Systems, Economic Evaluations, Quality of Life
- Type
- article
- Field-Weighted Citation Impact
- 7.90
Funders
- Parent Project Muscular Dystrophy
- Alexion Pharmaceuticals
- Ultragenyx Pharmaceutical
- European Commission
- Keio University
- Fondazione Telethon