Chapter 7 134 Disease areas Oncology Hematology Lung cancer Basal cell carcinoma Thyroid cancer Mesothelioma Glioma Colorectal cancer Renal cell carcinoma Prostate cancer Breast cancer Melanoma Other Infectious disease Gastro- enterology Neurology Internal medicine Other Pulmonology Benign hematology Ophtalmology Dermatology Acute myeloid leukemia Multiple myeloma Hodgkin lymphoma Stem cell transplant Acute lymphoblastic leukemia MDS GvHD VOD Chronic myeloid leukemia Chronic lymphoblastic leukemia Other Figure 10: Distribution of disease areas covered by expanded access literature from 2000 – 2021. MDS, myelodysplastic syndromes. GvHD, Graft-versus-Host Disease. Pharmacological therapeutics A total of 523 unique pharmacological therapeutics were described in our data set. Eighty-seven publications covered more than one therapeutic. The top ten most frequently appearing drugs are depicted in Table 5. Nivolumab was the most frequently appearing drug, likely due to its use in a variety of solid tumors (non-small cell lung cancer, glioblastoma, renal cell carcinoma) and nonsolid tumors (Hodgkin lymphoma), as well as its use in combination therapy for melanoma with ipilimumab. There may also be one drug-indication pair featuring a multiplicity of publications. In the case of cabazitaxel, a chemotherapy for castrate-resistant prostate cancer, there are individual expanded access publications from single centers,73 various single-country publications in Europe,74–76 as well as a Europe-wide publication.77 Furthermore, these 15 publications focus on different aspects of the treatment, such as safety,78 or quality-of-life.79 The effects of the COVID-19 pandemic The steep increase in publications in recent years can partially be attributed to COVID-19 pandemic. In 2020 and 2021, a large portion of publications was dedicated to medicine that could potentially treat COVID-19 infections: 29% (39/136) in 2020 and 29% (50/175) in 2021. Similarly, 49% (87/176) of expanded access publications on infectious diseases are related to COVID-19.
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