Clinical trials and rare diseases

Advances in Experimental Medicine and Biology

Volumn 686, Issue , 2010, Pages 173-190

  Gerß, Joachim Werner Otto ^a   Köpcke, Wolfgang ^b  

^a UNIVERSITY OF MÜNSTER   (Germany)

^b NONE

Author keywords

Design; Methodological approaches; Rare disease; Small clinical trial; Statistical analysis

Indexed keywords

ARTICLE; BAYES THEOREM; CLINICAL TRIAL (TOPIC); CONTROL GROUP; DATA ANALYSIS; EXTERNAL VALIDITY; HUMAN; INTERNAL VALIDITY; LONGITUDINAL STUDY; MEDICAL RESEARCH; META ANALYSIS (TOPIC); METHODOLOGY; OUTCOME ASSESSMENT; PREDICTION; PRIORITY JOURNAL; RANDOMIZATION; RARE DISEASE; RISK ASSESSMENT; SAMPLE SIZE; STATISTICAL ANALYSIS; BIOSTATISTICS; CONTROLLED CLINICAL TRIAL (TOPIC); LABORATORY DIAGNOSIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); REPRODUCIBILITY; REVIEW; STATISTICAL MODEL; STATISTICS;

BAYES THEOREM; BIOSTATISTICS; CLINICAL TRIALS AS TOPIC; CONTROLLED CLINICAL TRIALS AS TOPIC; FALSE POSITIVE REACTIONS; HUMANS; META-ANALYSIS AS TOPIC; MODELS, STATISTICAL; RANDOM ALLOCATION; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RARE DISEASES; REPRODUCIBILITY OF RESULTS;

EID: 79952065109     PISSN: 00652598     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-90-481-9485-8_11     Document Type: Article

References (17)

1. Chow S-C, Chang M (2006) Adaptive design methods in clinical trials. Chapman & Hall/CRC, Boca Raton
2. Committee forMedicinal Products for Human Use (CHMP) (2006) Guideline on clinical trials in small populations (CHMP/EWP/83561/2005). European Medicines Agency, London
3. Cook TD, DeMets DL (2008) Introduction to statistical methods for clinical trials. Chapman & Hall/CRC, Boca Raton
4. Cytel Inc. (2005) StatXact® 7 PROCs For SAS® Users. Statistical Software for Exact Nonparametric Inference, User Manual. Cytel Statistical Software & Services, Cambridge
5. Dahmen G, Ziegler A (2004) Generalized estimating equations in controlled clinical trials: hypotheses testing. Biometr J 46(2):214-232 (Pubitemid 41449121)
6. Evans CH Jr, Ildstad ST (eds) (2001) Small clinical trials: issues and challenges. National Academy Press, Washington, DC
7. Fayers PM, Ashby D, Parmar MKB (1997) Tutorial in biostatistics: Bayesian data monitoring in clinical trials. Stat Med 16(12):1413-1430 (Pubitemid 27292273)
8. Finkelstein MO, Levin B, Robbins H (1996) Clinical and prophylactic trials with assured new treatment for those at greater risk. Part I. Introduction. Am J Public Health 86:691-695 (Pubitemid 26146927)
9. Finkelstein MO, Levin B, Robbins H (1996) Clinical and prophylactic trials with assured new treatment for those at greater risk. Part II. Examples. Am J Public Health 86:696-705 (Pubitemid 26145483)
10. Giles FJ, Kantarjian HM, Cortes JE et al (2003) Adaptive randomized study of idarubicin and cytarabine versus troxacitabine and cytarabine versus troxacitabine and idarubicin in untreated patients 50 years or older with adverse karyotype acute myeloid leukemia. J Clin Oncol 21(9):1722-1727 (Pubitemid 46638583)
11. Hart A (2001) Making sense of statistics in healthcare. Radcliffe Medical Press, Abingdon
12. Jennison C, Turnbull BW (2000) Group sequential methods: applications to clinical trials. Chapman & Hall/CRC, Boca Raton
13. Molenberghs G, Verbeke G (2005) Models for discrete longitudinal data. Springer, New York
14. Rochon J (1998) Application of GEE procedures for sample size calculations in repeated measures experiments. Stat Med 17(14):1643-1658 (Pubitemid 28367844)
15. Schulze R (2004) Meta-analysis: a comparison of approaches. Hogrefe & Huber, Göttingen
16. Spiegelhalter DJ, Abrams KR, Myles JP (2004) Bayesian approaches to clinical trials and health-care evaluation. Wiley, New York
17. Verbeke G, Molenberghs G (2000) Linear mixed models for longitudinal data. Springer, New York