I discussed 'randomized withdrawal design and delayed start design in rare disease clinical trials'. The presentation slides are included in the link below.
In rare disease clinical trials, we should think about more when designing the clinical trial and should
go beyond the typical, conventional RCT (randomized, controlled trials).
In the presentation, the following materials were referenced:
Randomized Withdrawal Design or Randomized Discontinuation Trial:
- Kopec et al (1993) Randomized discontinuation trials: utility and efficiency. Journal of Clinical Epidemiology
- FDA (2012) Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products
- Biaggioni (2015) Randomized Withdrawal Study of Patients With Symptomatic Neurogenic Orthostatic Hypotension Responsive to Droxidopa
- Cardiovascular and Renal Drug Advisory Committee Meeting January 14, 2014
- Hughes RAC et al (2008) Intravenous immune globulin (10% caprylate-chromatography purified) for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy (ICE study): a randomised placebo-controlled trials. Lancet Neurology 2008
- Deng et al (2012) Challenges of clinical trial design when there is lack of clinical equipoise: use of a response-conditional crossover design. Journal of Neurology
Delayed Start Analysis
- Chapman et al (2015) Intravenous augmentation treatment and lung density in severe α1 Antitrypsin Deficiency (RAPID): a randomized, double-blind, placebo-controlled trial. The Lancet
- Liu-Seifert et al (2015) Delayed-start analysis: Mild Alzheimer’s disease patients in solanezumab trials, 3.5 years.
- Olanow et al (2009) A Double-Blind, Delayed-Start Trial of Rasagiline in Parkinson’s Disease NEJM
- October 17, 2011 Meeting of the Peripheral and Central Nervous System Drugs Advisory Committee