Discussion 255 The distinction between treatment and research should be perceived as a continuum rather than a binary choice,7,18 acknowledging the potential of goals and functions in the context of data collection and patient care. In fact, I view clinical evidence as existing along a continuum, ranging from randomized clinical trials to single-arm trials, to expanded access programs, to electronic health records, and even to mining social media for data. This spectrum is depicted in Figure 1. The perceived benefits of expanded access data can vary depending on one’s perspective. While acknowledging that the value of such data may be constrained in comparison to traditional clinical trials, it is noteworthy that they can still meaningfully augment other sources of realworld evidence. Randomized Trial Claims Social Media Large Simple Trial Expanded Access Pragmatic Trial Patient Charts PAES EHR Observational Studies Registries Health Surveys Interventional trials Figure 1: The continuum of data sources from strictly controlled and research-intended, to more treatmentfocused. The rigid categorical approach to continuous concepts can even hinder efficient analysis and data collection, particularly in the context of expanded access programs.19,20 Some regulators only permit the collection of safety data in these programs, while prohibiting the collection of efficacy data.20 Despite theoretical refutations to this separation, practical challenges complicate differentiating safety from efficacy, benefit from risk. In fact, ‘lack of efficacy’ is recognized as an official safety warning in pharmacovigilance terminology, as per the Medical Dictionary for Regulatory Activities.21 Hence, monitoring safety becomes difficult when such efficacy data cannot be collected. Similarly, deaths are considered safety events – but in most (expanded access) settings, survival is also the primary outcome of efficacy. Shifting focus towards the overlap among concepts rather than disparities, can facilitate data collection in expanded access settings.
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