Introduction: Background and outline of this thesis 15 reimbursement decision-making.38 Despite this growing interest, a systematic overview of the use and integration of expanded access data is absent. As a result, the extent to which these data are used by different stakeholders, such as scholars, patients, or regulators, remains uncertain. The reliability of data generated through expanded access remains unknown, and evidentiary requirements can vary depending on the intended use.37,39 For regulatory approval decisions, the highest-grade evidence, such as randomized controlled trials, can be more challenging to be supplemented by expanded access data. For other types of decisions, such as reimbursement decisions, different types of evidence than randomized controlled trials are appropriate. From a quantitative perspective, there is a scarcity of statistical techniques to interpret expanded access data,40 and these techniques may need evaluation or development to numerically incorporate expanded access. To what degree the nature of expanded access has changed, who stands to benefit from expanded access research, how, how often, and why data are collected, remains uncertain. Despite the potential benefits, the novel use of expanded access as data generation mechanism raises several concerns, including issues of patient protection, data quality, transparency, financial burden,41 and research oversight, among others. Research questions In this thesis, we will primarily investigate: ♣ 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? Methodology First, we illustrate the practical context of expanded access by providing real-world examples of the strategy, design, monitoring, and analyses of expanded access programs that we were involved in ourselves and inspired the writing of this thesis. In the theoretical context of expanded access, several research topics including ethics, health policy, and statistics warrant further exploration. We address this diverse range of issues by deploying an equally varied research armamentarium.
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