Table 2. Characteristics of Pivotal Studies and Their Primary Effectiveness End Points Supporting US Food and Drug Administration (FDA) Clearance of Therapeutic Medical Devices Through the De Novo Pathway Between 2011 and 2019.
| Characteristic | Pivotal studies, No. (%) |
|---|---|
| Total No. | 54 |
| Total patients, median (IQR) | 112.5 (73.5-187.0) |
| Patients treated, median (IQR) | 89.0 (49.0-118.5) |
| Study arms | |
| Multiarmed | 33 (61) |
| Single armed | 21 (39) |
| Randomized | 31 (57) |
| Blinding | |
| Blinded | 23 (43) |
| Open label | 23 (43) |
| Not specified | 8 (15) |
| Comparator type | |
| Active | 14 (26) |
| Sham | 19 (35) |
| Historical | 2 (4) |
| Pre–post comparison | 8 (15) |
| None | 11 (20) |
| Enrollment centers | |
| Multicenter | 41 (76) |
| Single center | 3 (6) |
| Not specified | 10 (19) |
| Pivotal premarket effectiveness end points | |
| Total No. | 60a |
| End point type | |
| Clinical outcome | 36 (60) |
| Clinical scale | 17 (28) |
| Surrogate marker | 7 (12) |
| Duration of longest primary end point follow-up, median (IQR), mo | |
| Overall | 1 (0-6) |
| Nonimplantable | 1 (0-3) |
| Implantable | 10 (4-12) |
| Primary end points met | |
| Yes | 40 (67) |
| No | 18 (30)b |
| End point success criteria unspecified | 2 (3) |
Abbreviation: IQR, interquartile range.
a
Does not include 5 pivotal studies supporting 5 devices without a primary effectiveness end point.
b
One pivotal study failed to meet 2 primary effectiveness end points.