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. 2021 May 18;4(5):e2110456. doi: 10.1001/jamanetworkopen.2021.10456

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.

a

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

b

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.