Table 2 Types of dose-finding studies and analyses performed in rare genetic disease drug development programs
From: Dose-finding studies in drug development for rare genetic diseases
Type of study or analysis | All drug development programs (n = 40) | No dedicated dose-finding study (n = 19) |
|---|---|---|
Dedicated dose-finding study | 21 (53%) | Not applicable |
Healthy subject study | 23 (58%) | 11 (58%) |
Study in different patient populations* | 10 (25%) | 4 (21%) |
Population pharmacokinetic analysis | 31 (78%) | 13 (68%) |
Exposure–response analysis | 28 (70%) | 11 (58%) |