Chapter 6 114 This begs the questions of who decides what formal and informal data collection is and whether all examples put forth in this paper where impermissible for regulators. Regardless of regulatory requirements, it can be a source of frustration when expanded access data are not available, as one Advisory Group (AG) noted: ‘The lack of any efficacy data from the compassionate use program is particularly disappointing,’ AG response to company comments, AG conclusions, TA535 Although the primary intent of expanded access programs is treatment provision and not to conduct research, it seems awkward to treat patients with investigational medicine and not to collect data to inform safety and efficacy. Furthermore, it is difficult to precisely determine where treatment-intent ends and research-intent starts. The changing nature of expanded access programs from sole treatment-intent to treatment-intent with data collection is a current topic of debate among bioethicists.11,33,52 We stress that data collection during expanded access should be light-weight and must not disproportionally burden patient and physicians – hence, a smart design should facilitate data to be collected.52 If so, expanded access programs can be the first source of RWD to inform HTA evaluations gathered in a pre-approval setting – this makes expanded access data different from general RWD sources (e.g., electronic health records or claims and billing data), as the latter will typically only start generating evidence once the drug has been approved. Results from expanded access programs can be obtained via peer-reviewed publications, if published. Alternatively, data can be requested via the medical company using data sharing platforms, such as Vivli.62 Finally, data may be available through local investigators (see HST7, Supplementary Material). Limitations and future research Our work has several limitations. First, we only reviewed TAs from one HTA body: NICE. Formally, NICE’s decisions are only valid within their UK jurisdiction, but informally they lead the way for other European HTA bodies-either via setting an example or via reference pricing.We have chosen NICE for our review as they have the longest history of HTA assessment and ample documentation publicly available. For other HTA bodies, results may be different. Future research should confirm whether our results uphold for other HTA bodies. Preliminary findings presented at a conference concluded that using expanded access data gathered within French compassionate use programs had a positive impact on reimbursement discussions.63 Second, we may have missed use-cases of expanded access data in payer submission as companies or reviewers may have used other terms to indicate expanded access programs (or failed to have done so). Our automated algorithm facilitates high throughput of document screening in health policy analysis, but it may have missed cases that would have been identified in manual evaluation. Therefore, our estimates should be
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