Summary 249 The main objective of this thesis is to ascertain the value of expanded access programs. In this regard, the notion of value is approached from different perspectives, including the clinical benefit derived by current patients and the merits of evidence generation for the benefit of future patients, while carefully considering the ethical and policy implications of viewing such programs as a partial means to generate information. We have investigated the following research questions. ♣ What are the medical benefits for patients receiving expanded access to experimental treatments? ♦ What are the ways in which data obtained from expanded access programs are utilized, and by whom? ♥ Can existing statistical techniques be adapted to incorporate data from expanded access programs in the context of analyzing clinical trials? ♠ What ethical concerns emerge when using expanded access as a means to generate evidence, and how can improvements be made to expanded access policies? The prelude contains a brief overview of the history of expanded access as a pathway to provide patients with access to experimental medical treatments, along with a detailed description – in Dutch - of the history and legislations of expanded access in the Netherlands. Additionally, we discussed the outcomes of two distinct real-world expanded access programs. In the first program, patients with treatment-resistant herpes viruses had the opportunity to access an investigational antiviral therapy, pritelivir. In the second program, patients with various diseases related to uncontrolled Epstein-Barr virus infections could access an unapproved cell therapy, tabelecleucel. ♣ In Part I, we studied how often ‘promising’ drugs advance through subsequent stages of research, as expanded access is often initiated based on presentations of preliminary findings. To limit the scope, we focused on the field of hematology. We assessed the clinical benefits of investigational drugs in acute myeloid leukemia using disease scales specifically developed to evaluate clinical merit of oncology and hematology drugs. Both oncology and hematology are associated with a high level of unmet medical need, and as such, they attract a considerable number of expanded access requests.Additionally, we used a universal metric of added benefit which allows to compare
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