| Title: |
How social pharmaceutical innovations are addressing problems of availability, accessibility and affordability of drugs for rare diseases |
| Authors: |
Douglas, Conor M. W.; Kleinhout - Vliek, Tineke; Hagendijk, Rob; Rabeharisoa, Vololona; Boon, Wouter; Aith, Fernando; Oliveira, Claudio Cordovil; Grunebaum, Shir; Moors, Ellen; Innovation and Transition; Dep of Sustainable Development; Innovation and Sustainability |
| Publication Year: |
2025 |
| Subject Terms: |
Orphan drugs; Patient organisations; Policy; Rare diseases; Social innovation; Social pharmaceutical innovation; Therapeutic research and development; Genetics(clinical); Pharmacology (medical) |
| Description: |
Background The current organization of the pharmaceutical innovation system poses three major challenges for rare disease patients in terms of availability, accessibility and affordability of treatments. While some changes have emerged in the European Union to address some of these challenges, their impacts are not experienced uniformly across member states nor around the world. We have observed niche initiatives that are actively working to address those challenges within their local contexts. In a position paper in this journal, we characterized such initiatives as “social pharmaceutical innovation” (or SPIN): novel collaborations involving diverse sets of actors that break with conventional pharmaceutical innovation practices to develop interventions that address unmet societal needs of rare disease patients and that are not primarily market driven. Results Here we report on 15 cases of SPIN across Brazil, Canada, France and the Netherlands that we studied through semi-structured qualitative interviews (n = 151) with players involved in those cases. Our findings show how SPIN initiatives are reconfiguring pharmaceutical innovation networks to include a wider range of actors in redistributed and differentiated roles within innovation processes. Further, we find that SPINs are associated with changes in the ways data is gathered (often in clinical contexts rather than in conventional trials), and how evidence is assembled to improve access to the treatments. Finally, we demonstrate how SPINs are providing new routes for patients to access treatments for rare diseases, often at more affordable prices. Conclusions While promising, SPINs are not perfect solutions for rare disease patients or the broader challenges to the pharmaceutical innovation system. SPINs are specific solutions adapted to the particulars of local framing, institutions, national policy and care contexts of rare diseases, and should be developed as such. Our findings support these recommendations for SPIN: use local knowledge and expertise in ... |
| Document Type: |
article in journal/newspaper |
| File Description: |
application/pdf |
| Language: |
English |
| ISSN: |
1750-1172 |
| Relation: |
https://dspace.library.uu.nl/handle/1874/479873 |
| Availability: |
https://dspace.library.uu.nl/handle/1874/479873 |
| Rights: |
info:eu-repo/semantics/OpenAccess |
| Accession Number: |
edsbas.2E3A693D |
| Database: |
BASE |