Table 3.
Drugs with patient-reported outcome measure findings in Food and Drug Administration medical review documents
| Brand namea | PRO measureb | Classification (Symptom, function, or both) | Clinical trials that used PRO measures | Trial design | PRO endpoint status | Analytical methods of PRO datab |
|---|---|---|---|---|---|---|
| Faslodex (Fulvestrant) | FACT-B TOI | Both | Trial #20 | Phase 3, Randomized, Open label, Active-controlled trial | Secondary | Time to deterioration in QoL |
| Faslodex (Fulvestrant) | FACT-B TOI | Both | Trial #21 | Phase 3, Randomized, Double-blind, Active-controlled trial | Secondary | Time to deterioration in QoL, Change in TOI |
| Tykerb (Lapatinib) | FACT-B, EQ-5D | Both | EGF 100151 | Phase 3, Randomized, Open label, Active-Controlled trial | Secondary | Change in QoL |
| Tykerb (Lapatinib) | FACT-B | Both | EGF 20008 | Phase 2, Open label, Uncontrolled, Two-cohort trial | Secondary | Change in QoL |
| Ixempra (Ixabepilone) | FBSI | Symptom | Study 046 | Phase 3, Randomized, Open label, Active-controlled trial | Secondary | Changes in symptoms during therapy |
| Perjeta (Pertuzumab) | FACT-B TOI | Both | CLEOPATRA | Phase 3, Randomized, Double-blind, Placebo-controlled trial | Secondary | Time to symptom progression |
| Kadcyla (Ado-trastuzumab emtansine) | FACT-B TOI | Both | EMILIA | Phase 3, Randomized, Open label, Active-controlled trial | Secondary | Time to symptom progression |
| Ibrance (Palbociclib) | mBPI-sf | Symptom | PALOMA-1 | Phase 1/2, Randomized, Open label, Active-controlled trial | Secondary | Changes in symptoms during therapy (pain) |
| Kisqali (Ribociclib) | EORTC QLQ-C30, EORTC QLQ-BR23, EQ-5D-5 L | Both | MONALEESA-2 | Phase 3, Randomized, Double-blind, Placebo-controlled trial | Secondary | Time to deterioration in QoL, Change in QoL |
| Nerlynx (Neratinib) | EQ-5D, FACT-B | Both | Study 3004 | Phase 3, Randomized, Double-blind, Placebo-controlled trial | Exploratory | Change in QoL |
| Verzenio (Abemaciclib) | mBPI-sf, EORTC QLQ-C30, EORTC QLQ-BR23, EQ-5D-5 L | Symptom (mBPI-sf), Both (rest) | MONARCH 2 | Phase 3, Randomized, Double-blind, Placebo-controlled trial | Secondary | Time to symptom progression, Change in QoL and symptom |
| Verzenio (Abemaciclib) | mBPI-sf, EORTC QLQ-C30 | Symptom (mBPI-sf), Both (rest) | MONARCH 1 | Phase 2, Open label, Single arm trial | Secondary | Change in QoL and symptom |
| Talzenna (Talazoparib) | EORTC QLQ-C30, EORTC QLQ-BR23 | Both | EMBRACA | Phase 3, Randomized, Open label, Active-controlled trial | Exploratory | Time to deterioration in QoL and symptom |
| Piqray (Alpelisib) | BPI-sf, EORTC QLQ-C30, EQ-5D-5 L | Symptom (BPI-sf), Both (rest) | SOLAR-1 | Phase 3, Randomized, Double-blind, Placebo-controlled trial | Secondary | Time to deterioration in QoL, symptom, and functioning |
aReview documents of Halaven and Enhertu did not include information about PRO measures and/or endpoints
bExplanation of abbreviations: BPI-sf Brief Pain Inventory short form, EORTC QLQ-BR23 European Organization for Research and Treatment of Cancer, Breast Cancer-Specific Quality of Life Questionnaire, EORTC QLQ-C30 European Organization for Research and Treatment of Cancer Quality of Life Questionnaire – Core Questionnaire, EQ-5D EuroQol 5 Dimensions, EQ-5D-5 L The 5-level EuroQol 5 Dimensions, FACT-B TOI Functional Assessment of Cancer Therapy-Breast Trial Outcome Index, FBSI Functional Assessment of Cancer Therapy-Breast Symptom Index, mBPI-sf Modified version of the Brief Pain Inventory short form, QoL Quality of Life, TOI Treatment Outcome Index