Figure 3.

Use of novel clinical trial designs. (a) Number of trials where a novel clinical trial design was used in an approved US Food and Drug Administration (FDA) submission. (b) Number of approved submissions that used surrogate end points. (c) Number of approved submissions that included studies with patient‐reported outcomes and/or real‐world data. Source data: ClinicalTrials.gov,6 FDA,7 Bhatt et al.,11 Zang et al.,12 TrialTrove,13 Bateman et al.,14 Yelland et al.,15 Albertson et al.,16 and Batten Disease News.17