Table 1.
Summary characteristics of the pivotal trials used for MM approvals in the US.
| Trials (n = 17) | |
|---|---|
| Study design | |
| Randomized | 14 (82%) |
| Single arm | 3 (18%) |
| Number of therapies (n = 32) | |
| Monotherapy | 3 (10%)a |
| Doublet | 15 (47%)a |
| Triplet | 12 (38%)a |
| Quadruplet | 2 (6%)a |
| Blinding status | |
| Blinded | 1 (6%) |
| Open label | 16 (94%) |
| Line of therapy | |
| First | 3 (18%) |
| Second line and beyond | 12 (70%) |
| Maintenance | 2 (12%) |
| Number of PRO instruments | |
| 1 | 3 (18%) |
| 2 | 7 (41%) |
| 3 | 5 (29%) |
| 4 | 2 (12%) |
| Collection format | |
| Electronic | 7 (42%) |
| Paper | 5 (29%) |
| Not specified | 5 (29%) |
| Type of instrument name | |
| EORTC QLQ-C30 | 13 (76%) |
| EORTC QLQ-MY20 | 8 (47%) |
| MDASI-MM | 1 (6%) |
| BPI-SF | 2 (12%) |
| EQ-5D—5L | 7 (41%) |
| EQ-5D—3L | 4 (23%) |
| FACT/GOG-NTX | 3 (18%) |
| MRU | 1 (6%) |
| FACT-MM | 1 (6%) |
| Item burden | |
| Mean (SD) | 48 (13) |
| Median (range) | 42 (32, 75) |
EORTC QLQ-C30 European Organization for Research and Treatment of Cancer Quality of Life Questionnaire—core Questionnaire, EORTC QLQ-MY20 EORTC QLQ—Multiple Myeloma Module, MDASI-MM MD Anderson Symptom Inventory—Multiple Myeloma Module, BPI-SF Brief Pain Inventory- Short Form, EQ-5D-5L EQ-5D-5 Levels, EQ-5D-3L EQ5D-3 Levels, FACT/GOG-NTX Functional Assessment of Cancer Treatment Gynecologic Oncology Group—Neurotoxicity, MRU Medical Resource Utilization, FACT-MM Functional Assessment of Cancer Treatment—Multiple Myeloma.
aPercentage based on the total number of therapies.