Critical appraisal of arguments for the delayed-start design proposed as alternative to the parallel-group randomized clinical trial design in the field of rare disease

Disease Modification in Emphysema Related to Alpha-1 Antitrypsin Deficiency

(2016) Joanna Chorostowska-Wynimko   COPD-Journal of Chronic Obstructive Pulmonary Disease

Intravenous augmentation treatment and lung density in severe α1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial

(2015) Kenneth R Chapman et al.   LANCET

Clinical Trials in Rare Disease

(2013) Erika F. Augustine et al.   JOURNAL OF CHILD NEUROLOGY

Experimental designs for small randomised clinical trials: an algorithm for choice

(2013) Catherine Cornu et al.   Orphanet Journal of Rare Diseases

A new proposal for randomized start design to investigate disease-modifying therapies for Alzheimer disease

(2011) Richard Y Zhang et al.   Clinical Trials

A framework for applying unfamiliar trial designs in studies of rare diseases

(2011) Samir Gupta et al.   JOURNAL OF CLINICAL EPIDEMIOLOGY

Survival distributions impact the power of randomized placebo-phase design and parallel groups randomized clinical trials

(2010) Lusine Abrahamyan et al.   JOURNAL OF CLINICAL EPIDEMIOLOGY

The Delayed-Start Study Design

(2009) Ralph B. D'Agostino   NEW ENGLAND JOURNAL OF MEDICINE

Are delayed-start design trials to show neuroprotection in Parkinson's disease fundamentally flawed?

(2008) Carl E. Clarke   MOVEMENT DISORDERS