RESUMO
BACKGROUND: During the past decade, patient engagement (PE) has attracted significant attention in the field of drug development. Readiness to accept the central importance of patients' knowledge and contributions has become evident. This study aimed to synthesize evidence on the current state of PE in drug development: what is actually being done and how. METHODS: A systematic scoping review was conducted based on a PRISMA-informed protocol. Search was performed in PubMed, EMBASE and Web of Science, covering the period between 2011 and 2021. For analysis of extracted data, we developed a framework for analyzing PE in Drug Development. The Framework distinguishes a number of different PE types that take place at different stages of drug development and are characterized by the different degrees of power patients have in the process. It allowed us to assess depth and intensity of PE initiatives included in this review. RESULTS: Most included PE initiatives took place at the stage of designing studies (40 in total). At this stage drug development goals are already set, but the mode of reaching them has not yet been fully determined. PE initiatives on the finetuning details stage followed (16 in total). The finetuning details stage covers the last parts of the drug development trajectory, when only relatively minor issues are still open for patients' contributions. The least numerous were PE initiatives on the stage of setting up R&D program (13 in total). This stage refers to the early steps in drug development where PE has the potential to make the most impact on shaping the subsequent process. In terms of intensity of engagement, most PE initiatives included in this review align with consultation and involvement types, 26 and 30 initiatives, respectively. Partnership was less frequent in the published accounts of PE (13 initiatives). CONCLUSIONS: This review delineated a contemporary landscape of PE in drug development. Although attention to PE in drug development is relatively recent, a wide range of PE practices has already been initiated. The results indicate the necessity of distinguishing between different types of PE in order to understand consequences of choices regarding depth and intensity of PE.
This article summarizes what is publicly found in scientific papers about patient engagement in drug development initiatives between 2011 and 2021. It also introduces a new Framework to use to look at these patient engagement efforts. The Framework breaks these efforts down based on the depth of patient engagement in the drug development process and different degrees of influence or power patients have or intensity of engagement. In terms of depth of patient engagement in the process of drug development, most patient engagement initiatives described efforts involved in designing studies where goals were already set. Next were patient engagement efforts related to finetuning details where patients could make minor contributions. The fewest efforts were found related to setting up a research and development program where patients potentially could make significant impact. In terms of intensity of patient engagement, most initiatives aligned with consultation and involvement intensities, and few examples aligned with the highest intensity of patient engagement that was considered partnership. While patient engagement in drug development is becoming more common, the approaches to doing so vary widely. We have developed a new Framework to help characterize these efforts related to patient influence in the process as well as depth of their engagement.