Table 1.
Terms combined in PubMed search | |
• |
(“Rare diseases” OR “orphan*”) AND (“Epidemiologic Methods” OR “Research Design” OR “Clinical Trials as Topic”) |
• |
Rare disease* |
• |
Clinical trial* |
• |
Clinical research |
• |
Withdrawal |
• |
Winner* |
• |
Loser* |
• |
Sequential |
• |
Adaptive |
• |
Delayed start |
• |
Early escape |
• |
N-of-1 |
• |
Randomi* |
• |
Placebo phase |
• |
Three stage |
Journals (years) hand-searched: | |
• |
Statistics in Medicine (1990–2010) |
• |
Orphanet Journal of Rare Diseases (2006–2010) |
• |
Controlled Clinical Trials (2000–2004) |
• |
Contemporary Clinical Trials (2005–2010) |
• |
BMC Medical Research Methodology (2001–2011) |
• |
Journal of Biopharmaceutical Statistics (1991–2012) |
• |
Biometrical Journal (1990 – 2012) |
• |
Statistica Sinica (1991–2012) |
• |
Journal of Statistical Planning and Inference (1990 – 2012) |
• |
Journal of the American Statistical Association (1990–2012) |
• | American Journal of Biostatistics (2010 – 2012) |
* = truncation symbol used in PubMed search.