Expanded access as a source of real-world data: An overview of FDA and EMA approvals 85 5♦ INTRODUCTION Patients suffering from seriously debilitating or life-threatening conditions who are not eligible for further treatments or any clinical trials, may resort to ‘expanded access’: pre-approval access to investigational treatments. Expanded access, also known as early access, pre-approval access or compassionate use,2 is the formal regulation adopted by the Food and Drug Administration (FDA) in 1987,3 propelled by the HIV/AIDS crisis. In the United States (US) the FDA regulates this process of formalized non-clinical trial access while in the European Union (EU) the responsibility lies with individual member states.4 The exact conditions, types (single patient, group, protocolized, emergency) and definitions of expanded access vary between member states.5 The numbers of requests for expanded access are growing and state and federal legislation, such as Right-to-Try laws in the US,6 stress the need and interest of patients in having earlier access to medicines that are still under clinical investigation. Also of interest, and closely related to expanded access, is the field of real-world data (RWD). RWD are information on health care that is derived from multiple sources outside typical clinical research settings.7 Recent publications and regulatory frameworks have boosted the promise of RWD.8–10 It can come in many forms and shapes, such as electronic health records, social media or claims databases. Expanded access programs are generally considered to be a source of RWD.1 Historically though, expanded access programs were only deemed fit for treatment and not for research. Although the primary purpose of expanded access is treatment, scholars have argued that there is a moral obligation to collect outcome data in all cases where patients are treated with investigational medicine.11–13 The debate on combining data collection and expanded access has substantially increased,14–16 with FDA-officials confirming beginning 2018 their willingness to review data from expanded access programs to support drug applications.11 Considering the increasing interest in both expanded access and RWD, the question arises whether alternative ways of access to novel treatments can provide clinical information and impact regulatory decision making. In this research, we systematically assess the role of RWD from expanded access programs in the regulatory approval process of the FDA and the European Medicines Agency (EMA), comparing and characterizing all approvals that utilize RWD on efficacy from expanded access programs. METHOD In the US, the FDA oversees both expanded access programs and marketing authorizations. In the EU, expanded access is supervised by individual member states, whereas marketing authorizations
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