Expanded access as a source of real-world data: An overview of FDA and EMA approvals 91 5♦ Expanded Access as pivotal evidence We further investigate the approvals where RWD from expanded access programs played a pivotal role. This was the case in one third (13/ 39) of the group mentioned in the previous paragraph. Table 1 gives an overview of these approvals. Twelve out of 13 received orphan designation. This is significantly higher if we compare it to regular approvals. For example, EMA assigned orphan designation to 134 out of 1,111 all-time approved drugs versus 12 out of 13 drugs in the pivotal group (p<0.0001). If we characterize by indication, just under half of the approvals (6/13) concerned treatments for metabolic disorders. The remainder are divided between hemato-oncology (three indications), infectious diseases (two indications) and overdosing (two indications), all covering areas of high unmet medical need. The median ratio of patients from expanded access programs to the total patient population (nexpanded access / N) that pivotally reinforced the efficacy profile was 71% (IQR: 34 – 100). In absolute terms, this varies from only two (vestronidase alfa) up to 558 patients (lutetium oxodotreotride). Albeit small, the former two patients formed 12% (2/17) of the total patient population in pivotal studies. On the other hand, 558 patients comprised 71% (558/787) of the total patient population, meaning that almost three quarters of the patient population was treated under expanded access programs. Although 558 is the largest number of patients we observed in the pivotal group, we have encountered expanded access programs containing more than 13,000 patients (stavudine) that provided information on efficacy with a supportive level of evidence.
RkJQdWJsaXNoZXIy MTk4NDMw