The patient’s view on rare disease trial design – a qualitative study-Reference-Cited by-同舟云学术

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

Published:2019-02-07 Issue:1 Volume:14 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

Gaasterland C. M. W.,van der Weide M. C. Jansen –,du Prie – Olthof M. J.,Donk M.,Kaatee M. M.,Kaczmarek R.,Lavery C.,Leeson-Beevers K.,O’Neill N.,Timmis O.,van Nederveen V.,Vroom E.,van der Lee J. H.

Funder

FP7 research program

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics (clinical),General Medicine

Link

http://link.springer.com/content/pdf/10.1186/s13023-019-1002-z.pdf

Reference28 articles.

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2. (EURORDIS) EOfRD. Rare Diseases: Understanding This Public Health Priority. 2005.

3. Angelis A, Tordrup D, Kanavos P. Socio-economic burden of rare diseases: a systematic review of cost of illness evidence. Health Policy. 2015;119(7):964–79.

4. Wilcken B. Rare diseases and the assessment of intervention: what sorts of clinical trials can we use? J Inherit Metab Dis. 2001;24(2):291–8.

5. Gagne JJ, Thompson L, O’Keefe K, Kesselheim AS. Innovative research methods for studying treatments for rare diseases: methodological review. BMJ. 2014;349:g6802.

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