Chapter 7 142 Introduction Note – below introduction and text is copied and adjusted from the template of JBI Evidence Synthesis from Literature Reviews1, as well as based on previous articles from TBP. A preliminary search of MEDLINE, the Cochrane Database of Systematic Reviews, PROSPERO, and JBI Evidence Synthesis was conducted and no current or underway systematic reviews or scoping reviews on the topic were identified. Expanded access (EA) is a pathway for patients who suffer from life-threatening conditions, who cannot enter clinical trials, and have exhausted all approved treatment options, to access investigational medicine (i.e., medicines that are not regulatory approved and are still experimental).4 It is also known as ‘compassionate use’, ‘early access’ or ‘non-trial pre-approval access’.2 The primary intent of EA programs is to provide patients and physicians in dire need with potential treatment options outside of clinical trials. Secondary, such programs may be used to collect RWD. It offers a potential opportunity to collect real world data in a pre-approval setting.51,52 Data from EA programs (EAPs) are used increasingly by regulators such as the US Food and Drug Administration and the European Medicines Agency in regulatory decision-making (i.e., to determine whether a drug should be recommended for routine use for a distinct disease and patient population).50 Furthermore, a recent overview of health technology appraisals by the English national institute for cost-effectiveness studies (NICE) concluded that 20% of these appraisals include data from EAPs. It is unknown if, how often and how data from EAPs are disseminated through scientific literature by medical companies or researchers. In this scoping review, we sought to quantify and classify the landscape of scientific literature on compassionate use. Main review question How does compassionate use (data) contribute to research literature? • How many original studies reporting data originating from EAPs have been published? ° Does this vary between disease area, disease, drug type, country, time, etc. • What studies are published on the collection, regulations on and use of expanded access data? Keywords Compassionate Use; Real-World Data; Health Policy; Expanded Access
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