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Article Dans Une Revue Orphanet Journal of Rare Diseases Année : 2022

Social pharmaceutical innovation and alternative forms of research, development and deployment for drugs for rare diseases

Conor Douglas
Fernando Aith
  • Fonction : Auteur
Wouter Boon
  • Fonction : Auteur
Marina de Neiva Borba
  • Fonction : Auteur
Shir Grunebaum
  • Fonction : Auteur
Rob Hagendijk
  • Fonction : Auteur
Larry Lynd
  • Fonction : Auteur
Faisal Ali Mohamed
  • Fonction : Auteur
Ellen Moors
  • Fonction : Auteur
Claudio Cordovil Oliveira
  • Fonction : Auteur
Vanessa Scanga
  • Fonction : Auteur
Julino Soares
  • Fonction : Auteur
Tineke Kleinhout-Vliek
  • Fonction : Auteur


Abstract Rare diseases are associated with difficulties in addressing unmet medical needs, lack of access to treatment, high prices, evidentiary mismatch, equity, etc. While challenges facing the development of drugs for rare diseases are experienced differently globally (i.e., higher vs. lower and middle income countries), many are also expressed transnationally, which suggests systemic issues. Pharmaceutical innovation is highly regulated and institutionalized, leading to firmly established innovation pathways. While deviating from these innovation pathways is difficult, we take the position that doing so is of critical importance. Innovation Challenges in Rare Diseases The reason is that the current model of pharmaceutical innovation alone will not deliver the quantity of products needed to address the unmet needs faced by rare disease patients, nor at a price point that is sustainable for healthcare systems. In light of the problems in rare diseases, we hold that re-thinking innovation is crucial and more room should be provided for alternative innovation pathways. Social Pharmaceutical Innovation (SPIN) We already observe a significant number and variety of new types of initiatives in the rare diseases field that propose or use alternative pharmaceutical innovation pathways which have in common that they involve a diverse set of societal stakeholders, explicitly address a higher societal goal, or both. Our position is that principles of social innovation can be drawn on in the framing and articulation of such alternative pathways, which we term here social pharmaceutical innovation (SPIN), and that it should be given more room for development. Interdisciplinary Research Approach As an interdisciplinary research team in the social sciences, public health and law, the cases of SPIN we investigate are spread transnationally, and include higher income as well as middle income countries. We do this to develop a better understanding of the social pharmaceutical innovation field’s breadth and to advance changes ranging from the bedside to system levels. Collaborations and Contributions We seek collaborations with those working in such projects (e.g., patients and patient organisations, researchers in rare diseases, industry, and policy makers). We aim to add comparative and evaluative value to social pharmaceutical innovation, and we seek to ignite further interest in these initiatives, thereby actively contributing to them as a part of our work.
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hal-03792528 , version 1 (12-12-2023)



Conor Douglas, Fernando Aith, Wouter Boon, Marina de Neiva Borba, Liliana Doganova, et al.. Social pharmaceutical innovation and alternative forms of research, development and deployment for drugs for rare diseases. Orphanet Journal of Rare Diseases, 2022, 17 (1), pp.344. ⟨10.1186/s13023-022-02476-6⟩. ⟨hal-03792528⟩
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