Bayesian methods for the design and interpretation of clinical trials in very rare diseases

Author:

Hampson Lisa V.1,Whitehead John1,Eleftheriou Despina2,Brogan Paul2

Affiliation:

1. Medical and Pharmaceutical Statistics Research Unit, Department of Mathematics and Statistics; Lancaster University; Lancaster, LA1 4YF U.K.

2. Department of Paediatric Rheumatology; UCL Institute of Child Health; 30 Guilford Street London WC1N 1EH U.K.

Funder

Medical Research Council

Arthritis Research UK

Publisher

Wiley

Subject

Statistics and Probability,Epidemiology

Reference38 articles.

1. European Medicines Agency Guideline on clinical trials in small populations 2006 http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003615.pdf

2. Clinical trials and rare diseases: a way out of a conundrum;Lilford;British Medical Journal,1995

3. Small sample sizes in clinical trials: a statistician's perspective;Billingham;Clinical Investigation,2012

4. Summarizing historical information on controls in clinical trials;Neuenschwander;Clinical Trials 2010

5. The network meta-analytic-predictive approach to non-inferiority trials;Schmidli;Statistical Methods in Medical Research,2011

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