The role of patient organisations in research and development

Evidence from rare diseases

Bibliographic Data

ID4599208
AuthorsArianna Gentilini (0000-0002-6943-6158, London School of Economics and Political Science, corresponding author), Marisa Miraldo (0000-0002-5772-7740, Imperial College London)
Year2023
Volume338
Pages116332
Publication date2023-12-01
Peer ReviewedYes
Open AccessYes
TypeARTICLE
VenueSocial Science & Medicine (JOURNAL)
Journal identifiersISSN: 0277-9536 • E-ISSN: 1873-5347
PublisherElsevier BV (PUBLISHER)
DOI10.1016/j.socscimed.2023.116332
PMID37866173
OpenAlexW4387709715
LanguageEN
Citations received5
References cited51

Patient organisations play an increasingly crucial role in the pharmaceutical sector, yet their impact on innovation remains unexplored. We estimate the impact of patient organisations on R&D activity in the context of rare diseases in Europe using a proprietary dataset that maps clinical trials from discovery to phase III across 29 countries, 1893 indications, and 30 years (1990-2019). By applying difference-in-differences and event study methodologies to a panel of 1,646,910 unique R&D observations, we find that country-indication pairs with at least one operating patient organisation have a higher rate of R&D activity compared to those without, with stronger effect in more prevalent rare diseases compared to ultra-rare conditions. We observe a lag in effects from patient organisation introduction, suggesting it takes approximately five years for these organisations to affect R&D activity. Overall, our work suggests that patient organisations play an important role in steering R&D efforts in rare diseases. Further research is needed to better understand mechanisms driving this effect and the potential impact of patient organisations on existing health inequities

Economic growth · Economics · Political science · Innovation Policy and RD · Intellectual Property and Patents · Medicine · Pharmaceutical Economics and Policy