Clinical trial designs for rare diseases: studies developed and discussed by the International Rare Cancers Initiative.

Détails

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Etat: Serval
Version: Final published version
ID Serval
serval:BIB_45CB57E34A2D
Type
Article: article d'un périodique ou d'un magazine.
Sous-type
Synthèse (review): revue aussi complète que possible des connaissances sur un sujet, rédigée à partir de l'analyse exhaustive des travaux publiés.
Collection
Publications
Titre
Clinical trial designs for rare diseases: studies developed and discussed by the International Rare Cancers Initiative.
Périodique
European Journal of Cancer
Auteur(s)
Bogaerts J., Sydes M.R., Keat N., McConnell A., Benson A., Ho A., Roth A., Fortpied C., Eng C., Peckitt C., Coens C., Pettaway C., Arnold D., Hall E., Marshall E., Sclafani F., Hatcher H., Earl H., Ray-Coquard I., Paul J., Blay J.Y., Whelan J., Panageas K., Wheatley K., Harrington K., Licitra L., Billingham L., Hensley M., McCabe M., Patel P.M., Carvajal R., Wilson R., Glynne-Jones R., McWilliams R., Leyvraz S., Rao S., Nicholson S., Filiaci V., Negrouk A., Lacombe D., Dupont E., Pauporté I., Welch J.J., Law K., Trimble T., Seymour M.
ISSN
1879-0852 (Electronic)
ISSN-L
0959-8049
Statut éditorial
Publié
Date de publication
2015
Peer-reviewed
Oui
Volume
51
Numéro
3
Pages
271-281
Langue
anglais
Notes
Publication types: Journal Article ; Research Support, Non-U.S. Gov't Publication Status: ppublish Document Type: Review
Résumé
BACKGROUND: The past three decades have seen rapid improvements in the diagnosis and treatment of most cancers and the most important contributor has been research. Progress in rare cancers has been slower, not least because of the challenges of undertaking research.
SETTINGS: The International Rare Cancers Initiative (IRCI) is a partnership which aims to stimulate and facilitate the development of international clinical trials for patients with rare cancers. It is focused on interventional--usually randomized--clinical trials with the clear goal of improving outcomes for patients. The key challenges are organisational and methodological. A multi-disciplinary workshop to review the methods used in ICRI portfolio trials was held in Amsterdam in September 2013. Other as-yet unrealised methods were also discussed.
RESULTS: The IRCI trials are each presented to exemplify possible approaches to designing credible trials in rare cancers. Researchers may consider these for use in future trials and understand the choices made for each design.
INTERPRETATION: Trials can be designed using a wide array of possibilities. There is no 'one size fits all' solution. In order to make progress in the rare diseases, decisions to change practice will have to be based on less direct evidence from clinical trials than in more common diseases.
Pubmed
Web of science
Open Access
Oui
Création de la notice
10/04/2015 19:25
Dernière modification de la notice
08/05/2019 17:54
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