Chapter 6 104 access’.2 The primary intent of expanded access programs is to provide patients and physicians in dire need with potential treatment options outside of clinical trials. Secondary, such programs may potentially collect real world data in a regulatory pre-approval setting, but the generation and useability of evidence derived from these programs remains a topic of debate.11,12,50–54 Data from expanded access programs may be used for various purposes in the appraisal process, for example to inform formal safety or efficacy analyses, to inform resource use and associated costs in real-world settings, to estimate the size of the patient population, or to gain insights into the treatment experience from patients or physicians that participated in an expanded access program. These data are increasingly accepted to support evidence of clinical efficacy by regulators, especially when collecting data in controlled settings is infeasible, such as in (ultra-) rare diseases, or is deemed unethical, in the case of extremely large treatment effects.50 However, the use of expanded access data by payers or HTA bodies remains unquantified. Understanding the role of expanded access data in TAs may clarify the value of these data for payers, pharmaceutical industry, physicians and patients, and is relevant for cost-effectiveness decision making and evaluation of HTA policy. Therefore, we here investigate the usage of expanded access data in NICE decision making by reviewing all appraisals presented to NICE between 2010 and 2020. METHODS Documents relating to all TAs conducted are provided on the NICE website. We investigated TAs published between January 1st 2010 and January 1st 2021. Terminated, withdrawn, or replaced appraisals were removed as documentation was unavailable. A schematic overview of our workflow is provided in Figure 5.
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