Prelude 18 inform trial data through expanded access programs. We will illustrate our method by analyzing individual patient-level data from the expanded access program and clinical trial of vemurafenib, a treatment for metastatic melanoma. We will evaluate how our method fares compared with traditional methods. ♠ In Part IV, we provide means to improve expanded access policies to expedite patient access together with evidence generation, discussing several inconsistencies in expanded access policies across jurisdictions. While ethical considerations, including the balance between patient autonomy, informed consent, and the potential for false hope, have been thoroughly discussed by previous scholars,25,31,42–47we expand upon their work by addressing the ethical implications of generating evidence through expanded access programs. Accordingly, we pose an outline of the ethical considerations surrounding expanded access research by evaluating if and when the benefits of additional evidence generation outweigh the research strains imposed on patients and physicians, and what policy improvements could be made to harmonize expanded access research in terms of data quality, oversight, and transparency, among others. Finally, our postlude concludes with a summary and discussion. While the limitations and interpretations of each individual research are described within their respective papers and parts, we dedicate the discussion to address the remainder of overarching issues related to expanded access and this thesis.
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