Chapter 6 108 appraisals. On the other hand, disease areas such as cardiology, gastroenterology, endocrinology, dermatology, rheumatology, and ophthalmology jointly make up 24.5% of all appraisals, whereas they merely account for 2.6% of the appraisals that included expanded access data. These results can be found in Table 2. Table 2: Technology appraisals that did (‘Yes’) or did not (‘No’) include expanded access data to support the profile of safety, efficacy and/or resource use, classified on disease area. Included expanded access data Disease area No1 Yes1 Total1 p-value2 0.001 Benign hematology 5 (1.7%) 3 (3.8%) 8 (2.1%) Cardiology 14 (4.7%) 0 (0%) 14 (3.7%) Dermatology 12 (4.0%) 1 (1.3%) 13 (3.4%) Endocrinology 12 (4.0%) 0 (0%) 12 (3.2%) Gastroenterology 13 (4.3%) 0 (0%) 13 (3.4%) Hematology 35 (12%) 20 (25%) 55 (14%) Internal medicine 23 (7.6%) 9 (11%) 32 (8.4%) Neurology 14 (4.7%) 6 (7.6%) 20 (5.3%) Oncology 106 (35%) 32 (41%) 138 (36%) Ophthalmology 18 (6.0%) 0 (0%) 18 (4.7%) Psychiatry 3 (1.0%) 1 (1.3%) 4 (1.1%) Pulmonology 6 (2.0%) 4 (5.1%) 10 (2.6%) Rheumatology 22 (7.3%) 1 (1.3%) 23 (6.1%) Surgery 4 (1.3%) 1 (1.3%) 5 (1.3%) Urology 1 (0.3%) 1 (1.3%) 2 (0.5%) Vascular medicine 13 (4.3%) 0 (0%) 13 (3.4%) Total 301 (79%) 79 (21%) 380 (100%) 1 n (%) 2 Pearson chi-square test Examples To give the reader a better sense of the main labels ‘safety, efficacy, resource use’ as well as the secondary ‘treatment experience’ label, we here provide illustrative examples from the TAs that were supported by expanded access data. Safety Safety data from expanded access programs are often described rather qualitatively, supporting results from clinical trials. For example, in the appraisal of gefitinib for the first-line treatment of locally advanced or metastatic non-small-cell lung cancer, the appraisal committee noted: ‘The favorable safety profile of gefitinib demonstrated in the phase III studies is consistent with that observed in everyday settings. In addition to the data from clinical trials, the Early Access Program for gefitinib in Caucasian patients indicated that gefitinib is well tolerated by
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