Results from expanded access programs: a review of academic literature 135 7♦ Table 5: Overview of top ten most frequently described pharmacological therapeutics. Drugs Publications Patients Disease entities Nivolumab 48 15,957 Lung Cancer (26x, N=12,327), Renal Cell Carcinoma (7x, N=2,062), Melanoma (5x, N=1,073), Hodgkin Lymphoma (4x, N=259), Basal Cell Carcinoma (1x, N=1), Gastric Cancer (1x, N=113), Germ Cell Tumors (1x, N=7), Glioblastoma (1x, N=1), Hemophagocytic Lymphohistiocytosis (1x, N=7), Mesothelioma (1x, N=107) Ipilimumab 37 8,244 Melanoma (37x, N=8,244) Gefitinib 36 3,900 Lung Cancer (34x, N=3,834), Head and Neck Cancer (2x, N=66) Sofosbuvir 21 2,751 Hepatitis C (20x, N=2,749), Yellow Fever Virus (1x, N=2) Sunitinib 18 23,996 Renal Cell Carcinoma (14x, N=23,964), Colorectal Cancer (2x, N=30), Liposarcoma (1x, N=1), Sarcoma (1x, N=1) Everolimus 17 8,074 Renal Cell Carcinoma (4x, N=4,193), Neuroendocrine Tumors (3x, N=416), Breast Cancer (2x, N=3,282), Epilepsy (2x, N=16), Basal Cell Carcinoma (1x, N=4), Hodgkin Lymphoma (1x, N=33), Paraganglioma; Pheochromocytoma (1x, N=4), Renal Angiomyolipomas Tuberous Sclerosis Complex (1x, N=19), Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis (1x, N=100), Tuberous Sclerosis (1x, N=7) Plerixafor 17 1,378 Stem Cell Transplant (17x, N=1,378) Cabazitaxel 15 4,925 Prostate Cancer (15x, N=4,925) Cannabidiol 15 2,148 Epilepsy (12x, N=1,446), Dravet Syndrome & Lennox-Gastaut Syndrome (2x, N=700), Dravet Syndrome (1x, N=2) Cefiderocol 15 163 COVID-19 (2x, N=124), Osteomyeleitis (2x, N=2), Pseudomonas Aeruginosa (2x, N=2), Acinetobacter Baumanii Infections (1x, N=3), Aortic Valve Endocarditis (1x, N=1), Bacteriemia (1x, N=13), Carbapenem-Resistant (Cr) Gram-Negative Pathogens (1x, N=13), Enterobacter Hormaechei Infection (1x, N=1), Klebsiella Pneumoniae (1x, N=1), Multidrug Resistant Bacterial Infections (1x, N=1), Pancreatic Abscess (1x, N=1), Prosthetic Joint Infection (1x, N=1) The pandemic boosted publications on potential treatments such as convalescent plasma (N=15), remdesivir (N=14), and tocilizumab (N=11). The publications on the use of convalescent plasma to potentially treat COVID-19 infections comprised 194,256 patients, shifting the distribution of patient numbers on its own. We systematically extracted and grouped all end points reported in COVID-19 related publications. The 42 unique endpoints are not limited to mandatory safety monitoring: The five most frequently described endpoints in COVID-19 publications were Adverse Events (57%), mortality (45%), inflammatory markers (34%), oxygen support (32%) and clinical improvement (26%) (Table 6, see Supplementary Material for a complete overview of all used endpoints). In total, 41 unique drugs have been provided under compassionate use to treat COVID-19. Of these, four drugs received EMA approval, and five drugs are authorized for use by the FDA (in part under Emergency Use), as of January 2023.80 The submission to the EMA for the FDA-approved agent baricitinib was withdrawn by the applicant.81
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