Results from expanded access programs: a review of academic literature 133 7♦ As the results described in this section aim to abstract information across drugs and conditions, the reader that is interested in a more detailed description of expanded access publications can find an analysis of ten randomly selected articles in the Supplemental Material. This sample demonstrates the heterogeneity of our dataset across a variety of quantitative variables such as sample size, study design, geographic region, patient populations, and EAP duration. But the articles also differ on qualitative aspects. Some authors discuss at length the differences between the patients in the EAP and the clinical trials and how these differences may result in different outcomes. Other authors are unable to report on such trial versus EAP differences, as disparities in sample sizes may prevent a useful comparison (e.g., case report). Additionally, trial results may simply be unavailable for the product while expanded access was provided as trials might have been ongoing or not even initiated. Table 4: Characteristics of top ten most productive countries of national expanded access publications, ranked by the number of publications. US, United States. *, average population between 2006 – 2016. Country Publications Patients Population* Publications per capita US 240 230,566 311.3 0.8 Italy 183 39,100 59.6 3.1 France 90 24,250 65.3 1.4 Germany 88 6,473 81.5 1.1 Spain 83 6,048 46.2 1.8 UK 49 2,945 63.2 0.8 Canada 32 11,736 34.4 0.9 Australia 29 2,599 22.4 1.3 Netherlands 29 3,622 16.7 1.7 Belgium 28 5,869 11.0 2.5 Disease areas The 1,231 publications covered 354 unique diseases across 18 disease areas. The top ten most frequently appearing disease areas are depicted in Figure 10. The two largest areas, oncology 39.6% (488/1,231) and hematology 13.8% (170/1,231), are further broken down per top ten most frequent diseases. Note that a single publication can cover multiple diseases (this is the case in 38 publications), for example where a single drug is tested in adjacent diseases, such as expanded access of azacitidine to treat patients with both acute myeloid leukemia and myelodysplastic syndromes.69–71 Other instances include diseases sharing a common actionable target such as ErbB2/HER2 in breast and colorectal cancer and malignant melanoma.72
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