Please use this identifier to cite or link to this item:
https://www.arca.fiocruz.br/handle/icict/55430
Type
ArticleCopyright
Open access
Collections
- ENSP - Artigos de Periódicos [2082]
Metadata
Show full item record
SOCIAL PHARMACEUTICAL INNOVATION AND ALTERNATIVE FORMS OF RESEARCH, DEVELOPMENT AND DEPLOYMENT FOR DRUGS FOR RARE DISEASES
Orphan Drugs
Rare Diseases
Therapeutic Research and Development
Social Innovation
Policy
Patient Organisations
Author
Douglas, Conor M. W.
Aith, Fernando
Boon, Wouter
Borba, Marina de Neiva
Doganova, Liliana
Grunebaum, Shir
Hagendijk, Rob
Lynd, Larry
Mallard, Alexandre
Mohamed, Faisal Ali
Moors, Ellen
Cordovil-Oliveira, Claudio
Paterson, Florence
Scanga, Vanessa
Soares, Julino
Raberharisoa, Vololona
Kleinhout-Vliek, Tineke
Aith, Fernando
Boon, Wouter
Borba, Marina de Neiva
Doganova, Liliana
Grunebaum, Shir
Hagendijk, Rob
Lynd, Larry
Mallard, Alexandre
Mohamed, Faisal Ali
Moors, Ellen
Cordovil-Oliveira, Claudio
Paterson, Florence
Scanga, Vanessa
Soares, Julino
Raberharisoa, Vololona
Kleinhout-Vliek, Tineke
Affilliation
York University. Department of Science, Technology and Society. Toronto, Canada.
Universidade de São Paulo. Faculdade de Saúde Pública. Centro de Pesquisa em Direito Sanitário. São Paulo, SP, Brasil.
Universiteit Utrecht. Copernicus Institute of Sustainable Development. Netherlands.
Universidade de São Paulo. Faculdade de Medicina São Camilo. São Paulo, SP, Brasil.
Université PSL. Mines ParisTech. Paris, France.
York University. Department of Science and Technology Studies. Toronto, Canada.
University of Amsterdam. Faculty of Social and Behavioural Sciences. International School of Social Sciences and Humanities. Amsterdam, The Netherlands.
University of British Columbia. Faculty of Pharmaceutical Sciences. Vancouver, Canada.
Université PSL in Paris. Center for Social Innovation. Paris, France.
York University. Faculty of Health Policy and Equity. Toronto, Canada.
Universiteit Utrecht. Copernicus Institute of Sustainable Development. Utrecht, The Netherlands.
Fundação Oswaldo Cruz. Escola Nacional de Saúde Pública Sergio Arouca. Rio de Janeiro, RJ, Brasil.
Université PSL. Mines ParisTech. Paris, France.
York University. Hall Law School. Toronto, Canada.
Universidade Federal de São Paulo. Faculdade de Saúde Pública. São Paulo, SP, Brasil
Université PSL. Mines ParisTech. Paris, France.
Universiteit Utrecht. Geosciences, Innovation Studies, Innovation and Sustainability Institute. Utrecht, The Netherlands.
Universidade de São Paulo. Faculdade de Saúde Pública. Centro de Pesquisa em Direito Sanitário. São Paulo, SP, Brasil.
Universiteit Utrecht. Copernicus Institute of Sustainable Development. Netherlands.
Universidade de São Paulo. Faculdade de Medicina São Camilo. São Paulo, SP, Brasil.
Université PSL. Mines ParisTech. Paris, France.
York University. Department of Science and Technology Studies. Toronto, Canada.
University of Amsterdam. Faculty of Social and Behavioural Sciences. International School of Social Sciences and Humanities. Amsterdam, The Netherlands.
University of British Columbia. Faculty of Pharmaceutical Sciences. Vancouver, Canada.
Université PSL in Paris. Center for Social Innovation. Paris, France.
York University. Faculty of Health Policy and Equity. Toronto, Canada.
Universiteit Utrecht. Copernicus Institute of Sustainable Development. Utrecht, The Netherlands.
Fundação Oswaldo Cruz. Escola Nacional de Saúde Pública Sergio Arouca. Rio de Janeiro, RJ, Brasil.
Université PSL. Mines ParisTech. Paris, France.
York University. Hall Law School. Toronto, Canada.
Universidade Federal de São Paulo. Faculdade de Saúde Pública. São Paulo, SP, Brasil
Université PSL. Mines ParisTech. Paris, France.
Universiteit Utrecht. Geosciences, Innovation Studies, Innovation and Sustainability Institute. Utrecht, The Netherlands.
Abstract
Rare diseases are associated with difculties 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 expe‑
rienced diferently globally (i.e., higher vs. lower and middle income countries), many are also expressed transnation‑
ally, which suggests systemic issues. Pharmaceutical innovation is highly regulated and institutionalized, leading to
frmly established innovation pathways. While deviating from these innovation pathways is difcult, we take the posi‑
tion 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 signifcant number and variety of new types of initiatives in the rare diseases feld 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
feld’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.
Keywords
Social Pharmaceutical InnovationOrphan Drugs
Rare Diseases
Therapeutic Research and Development
Social Innovation
Policy
Patient Organisations
Share