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

Autor: Douglas CMW; Department of Science, Technology and Society, 307 Bethune College, York University, 4700 Keele Street, Toronto, ON, M3J 1P3, Canada. cd512@yorku.ca., Aith F; University of São Paulo Public Health School, Health Law Research Center of the University of São Paulo, Av. Dr. Arnaldo, 715, São Paulo, Brazil., Boon W; Copernicus Institute of Sustainable Development, Universiteit Utrecht, Princetonlaan 8a, 3584 CB, Utrecht, The Netherlands., de Neiva Borba M; São Camilo Medical School, School of Public Health, University of São Paulo, Av. Dr. Arnaldo, 715, São Paulo, Brazil., Doganova L; Mines ParisTech, Université PSL in Paris, 60 Boulevard Saint Michel, 75272, Paris Cedex 06, France., Grunebaum S; Department of Science and Technology Studies, 307 Bethune College, York University, 4700 Keele Street, Toronto, ON, M3J 1P3, Canada., Hagendijk R; Faculty of Social and Behavioural Sciences, International School of Social Sciences and Humanities, University of Amsterdam, Spui 2, 1012 WX, Amsterdam, The Netherlands., Lynd L; Faculty of Pharmaceutical Sciences, University of British Columbia, 2405 Wesbrook Mall, Vancouver, BC, V6T 1Z3, Canada., Mallard A; Center for Social Innovation, Université PSL in Paris, Mines ParisTech60 Boulevard Saint Michel, 75272, Paris Cedex 06, France., Mohamed FA; Faculty of Health Policy and Equity, York University, 4700 Keele Street, Toronto, ON, M3J 1P3, Canada., Moors E; Innovation and Sustainability, Copernicus Institute of Sustainable Development, Universiteit Utrecht, Princetonlaan 8a, 3584 CB, Utrecht, The Netherlands., Oliveira CC; Public Health at the Sergio Arouca National School of Public Health (ENSP/Fiocruz), Av. Brazil, 4365 - Manguinhos, Rio de Janeiro, Brazil., Paterson F; Mines ParisTech, Université PSL in Paris, 60 Boulevard Saint Michel, 75272, Paris Cedex 06, France., Scanga V; Osgoode Hall Law School of York University, 4700 Keele Street, Toronto, ON, M3J 1P3, Canada., Soares J; The Federal University of Sao Paulo (UNIFESP), School of Public Health at the University of São Paulo, Av. Dr. Arnaldo, 715, São Paulo, Brazil., Raberharisoa V; Mines ParisTech, Université PSL in Paris, 60 Boulevard Saint Michel, 75272, Paris Cedex 06, France., Kleinhout-Vliek T; Geosciences, Innovation Studies, Innovation and Sustainability Institute, Universiteit Utrecht, Princetonlaan 8a, 3584 CB, Utrecht, The Netherlands.
Jazyk: angličtina
Zdroj: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2022 Sep 05; Vol. 17 (1), pp. 344. Date of Electronic Publication: 2022 Sep 05.
DOI: 10.1186/s13023-022-02476-6
Abstrakt: 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. 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. 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. 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. 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.
(© 2022. The Author(s).)
Databáze: MEDLINE
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