Lessons learned from IDeAl — 33 recommendations from the IDeAl-net about design and analysis of small population clinical trials-Reference-Cited by-同舟云学术

Lessons learned from IDeAl — 33 recommendations from the IDeAl-net about design and analysis of small population clinical trials

Published:2018-05-11 Issue:1 Volume:13 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

Hilgers Ralf-Dieter^ORCID,Bogdan Malgorzata,Burman Carl-Fredrik,Dette Holger,Karlsson Mats,König Franz,Male Christoph,Mentré France,Molenberghs Geert,Senn Stephen

Funder

FP7 Health

Publisher

Springer Science and Business Media LLC

Subject

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

Link

http://link.springer.com/content/pdf/10.1186/s13023-018-0820-8.pdf

Reference93 articles.

1. CHMP. Guideline on clinical trials in small populations. 2007. [Cited: February 1,2013.] www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003615.pdf .

2. Austin CP, et al. Future of rare diseases research 2017-2027: an IRDiRC perspective. Clin Transl Sci. 2018;11(1):21–7. https://doi.org/10.1111/cts.12500 .

3. Hilgers RD, Roes KC, Stallard N. Directions for new developments on statistical design and analysis of small population group trials. Orphanet J Rare Dis. 2016;11(1):78. https://doi.org/10.1186/s13023-016-0464-5 .

4. Senn SJ. A note regarding meta-analysis of sequential trials with stopping for efficacy. Pharm Stat. 2014;13(6):371–5. https://doi.org/10.1002/pst.1639 .

5. Bauer P, König F. Adaptive paediatric investigation plans, a small step to improve regulatory decision making in drug development for children. Pharm Stat. 2016:384–6. https://doi.org/10.1002/pst.1762 .

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