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The role of patient organizations on research and development: evidence from rare diseases

Gentilini, Arianna and Miraldo, Marisa (2023) The role of patient organizations on research and development: evidence from rare diseases. Social Science & Medicine, 338. ISSN 0277-9536

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Identification Number: 10.1016/j.socscimed.2023.116332


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, 1,893 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.

Item Type: Article
Official URL:
Additional Information: © 2023 The Author(s)
Divisions: Methodology
Subjects: R Medicine > RA Public aspects of medicine > RA0421 Public health. Hygiene. Preventive Medicine
H Social Sciences
Date Deposited: 20 Oct 2023 10:18
Last Modified: 16 May 2024 04:16

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