Table 6 Categories Used to Review Articles Identified Through Search on Rare Disease Methods
From: An overview of the impact of rare disease characteristics on research methodology
Broad Categories | Research Designs within each category |
|---|---|
Randomized Designs | Parallel-group RCT; Crossover RCT; N-of-1 Trials; Ranking and Selection RCT; Sequential RCT; Adaptive RCT; Internal Pilot; Randomized Placebo Phase; Stepped Wedge; “Early Escape” in RCT; Randomized Withdrawal; Three-stage trial; Boundaries Design; Factorial Design; Phase II Multicenter Open Label; Phase III: Intent to Treat - Randomized, double-blind, Placebo Controlled; RCT with patients grouped by etiology; Phase I/II Proof of Principle; Repeated measurement designs; Single multi-arm trial where a series of research arms are assessed in parallel against a common control |
Nonrandomized Controlled Trials | Risk-based allocation; Delayed Start |
Observational Designs | Prospective inception cohort; case-control studies; cohorts with historic controls/ Natural History Studies; Pre-post designs; Case reports/ case series |
Analytic Methods | Bayesian Analysis; Propensity Scores; Instrumental Variables |
Other | Meta-Analysis |