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The patient’s view on rare disease trial design – a qualitative study

Abstract Background Clinical trials in rare diseases are more challenging than trials in frequent diseases. Small numbers of eligible trial participants, often complicated by heterogeneity among rare disease patients, hamper the design and conduct of a ‘classical’ Randomized Controlled Trial. Theref...

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Bibliografiske detaljer
Principais autores: C. M. W. Gaasterland, M. C. Jansen – van der Weide, M. J. du Prie – Olthof, M. Donk, M. M. Kaatee, R. Kaczmarek, C. Lavery, K. Leeson-Beevers, N. O’Neill, O. Timmis, V. van Nederveen, E. Vroom, J. H. van der Lee
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2019-02-01
Serier:Orphanet Journal of Rare Diseases
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Online adgang:http://link.springer.com/article/10.1186/s13023-019-1002-z
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