Participant-funded clinical trials on rare diseases
Por:
Dal-Ré R, Palau F, Guillén-Navarro E and Ayuso C
Publicada:
1 oct 2020
Ahead of Print:
1 jun 2020
Categoría:
Pediatrics, perinatology and child health
Resumen:
The development of medicines for certain rare diseases can be frustrated by lack of funding. In certain cases the patients themselves, or their relatives, occasionally fund the clinical trial in which they will be treated with the investigational medicine. There are 3 models of self-funded research: 2 of them, "pay to try" and "pay to participate", have already been put into practice. The third, the "plutocratic" proposal, which has been recently put forward is still a theoretical model. In this work the scientific, social and ethical benefits and risks of the 2 clinical research models, "pay to participate" and the "plutocratic" proposal, are reviewed. Patient-funded clinical trials are frequently performed through crowdfunding. The most controversial aspects of this funding modality are also addressed in this article from several perspectives. Finally, a future scenario that would allow the launching of self-funded clinical trials in Spain by the "plutocratic" proposal is proposed. (C) 2020 Asociacion Espanola de Pediatria. Published by Elsevier Espana, S.L.U.
Filiaciones:
Dal-Ré R:
Unidad de Epidemiología, Instituto de Investigación Sanitaria, Hospital Universitario Fundación Jiménez Díaz, IIS-UAM, Universidad Autónoma de Madrid, Madrid, España
Palau F:
CIBER de Enfermedades Raras (CIBERER), ISCIII, Madrid, España
Institut Clínic de Medicina i Dermatologia, Hospital Clínic, Barcelona, España
Unidad de Pediatría, Facultat de Medicina i Ciències de la Salut, Universitat de Barcelona, Barcelona, España
Servicio de Medicina Genética y Molecular, Instituto Pediátrico de Enfermedades Raras (IPER), Hospital Sant Joan de Déu, Institut de Recerca Sant Joan de Déu, Esplugues de Llobregat, Barcelona, España
Guillén-Navarro E:
CIBER de Enfermedades Raras (CIBERER), ISCIII, Madrid, España
Sección de Genética Médica, Servicio de Pediatría, Hospital Clínico Universitario Virgen de la Arrixaca, Instituto Murciano de Investigación Biosanitaria (IMIB-Arrixaca), Facultad de Medicina, Universidad de Murcia, Murcia, España
Ayuso C:
CIBER de Enfermedades Raras (CIBERER), ISCIII, Madrid, España
Departamento de Genética y Genómica, Instituto de Investigación Sanitaria, Hospital Universitario Fundación Jiménez Díaz, IIS-UAM, Universidad Autónoma de Madrid, Madrid, España
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