Table 2. Characteristics of US Food and Drug Administration–Approved Drugs.
Drug | Drug class | Patient enrollment | Approval indication | ASCO-VF category (score)a |
---|---|---|---|---|
Afatinib | Targeted | 7894 | Locally advanced or metastatic NSCLC with EGFR variant | Low value (33.6) |
Blinatumomab | Immunotherapy | 1582 | Philadelphia chromosome–relapsed or refractory B-cell precursor ALL | Low value (29.0) |
Elotuzumab | Immunotherapy | 3834 | Multiple myeloma, received prior therapies | Low value (24.0) |
Idelalisib | Targeted | 2513 | Refractory indolent non-Hodgkin lymphoma and relapsed chronic lymphocytic leukemia | Substantial value (72.0)b |
Liposomal irinotecan | Cytotoxic | 944 | Metastatic adenocarcinoma of pancreas | Low value (33.0) |
Moxetumomab pasudotox | Immunotherapy | 157 | Relapsed or refractory hairy cell leukemia | No completed comparative RCT |
Necitumumab | Targeted | 2315 | First-line treatment for locally advanced or metastatic squamous NSCLC | Low value (16.0) |
Obinutuzumab | Immunotherapy | 6058 | Previously untreated chronic lymphocytic leukemia | Substantial value (65.6) |
Pexidartinib | Targeted | 908 | Symptomatic tenosynovial giant cell tumor | No completed comparative RCT |
Ramucirumab | Targeted | 7498 | Advanced gastric or gastroesophageal junction adenocarcinoma after prior chemotherapy | Low value (22.4) |
Trametinib | Targeted | 3776 | Unresectable or metastatic melanoma with BRAF variation | Intermediate value (44.0) |
Vemurafenib | Targeted | 4655 | Unresectable or metastatic melanoma with BRAF variation | Substantial value (59.2) |
Ziv-aflibercept | Targeted | 5779 | Metastatic colorectal cancer | Low value (18.3) |
Abbreviations: ALL, acute lymphoblastic lymphoma; ASCO-VF, American Society of Clinical Oncology Value Framework; NSCLC, non–small cell lung cancer; RCT, randomized clinical trial.
Based on scoring of ASCO scores of the earliest trial assessed in the study by Saluja et al20; low (≤40), intermediate (>40 to <45), substantial (≥45) value score interpretation by Cherny et al.21
Approved for 2 different indications (non-Hodgkin lymphoma and chronic lymphocytic leukemia) on the same date; for the purposes of our analysis, we counted this as a single first approval and used the date of submission of the New Drug Application for non-Hodgkin lymphoma, as this was submitted prior to that of chronic lymphocytic leukemia. The ASCO-VF score is for a phase 3 trial in chronic lymphocytic leukemia, which was the only phase 3 trial performed at the time of approval.