Clinical trial designs for rare diseases: Studies developed and discussed by the International Rare Cancers Initiative
European Journal of Cancer2014Vol. 51(3), pp. 271–281
Citations Over TimeTop 10% of 2014 papers
Jan Bogaerts, Matthew R. Sydes, Nicola Keat, Andrea McConnell, Al B. Benson, Alan L. Ho, Arnaud Roth, Catherine Fortpied, Cathy Eng, Clare Peckitt, Corneel Coens, Curtis A. Pettaway, Dirk Arnold, Emma Hall, Ernie Marshall, Francesco Sclafani, Helen Hatcher, Helena Earl, Isabelle Ray‐Coquard, James Paul, Jean‐Yves Blay, Jeremy Whelan, K. Panageas, Keith Wheatley, Kevin J. Harrington, Lisa Licitra, Lucinda Billingham, Martee L. Hensley, Martin G. McCabe, Poulam M. Patel, Richard D. Carvajal, Richard H. Wilson, Rob Glynne‐Jones, Rob McWilliams, Serge Leyvraz, Sheela Rao, Steve Nicholson, Virginia L. Filiaci, Anastassia Negrouk, Denis Lacombe, Elisabeth Dupont, Iris Pauporté, John J. Welch, Kate Law, Ted Trimble, Michel Seymour
Abstract
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.
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