Evidence from randomized clinical trials (RCTs) continues to be the standard reference point for treatment efficacy across the world. However, RCTs enrol patients based on strict inclusion and exclusion criteria, and hence RCT evidence is often not generalizable and inadequate for contributing to the day-to-day clinical practice decisions. Consequently, researchers are more interested in using real-world data (RWD) to guide healthcare decisions.(1) Analysis of RWD that enables risk vs. benefit assessment while also providing data on the utility of medical intervention is called the real-world evidence (RWE).(2)
With the growing interest in adopting RWE for decision-making, regulators are formally introducing recommendations for both collecting RWD and generating RWE. The latest is the National Institute of Health Care and Excellence (NICE, UK). In June 2022, NICE launched a real-world evidence (RWE) framework for optimizing RWD collection to fill the knowledge gaps and make innovative healthcare interventions easily accessible to patients.(3)
The NICE defines RWD as data on patient healthcare delivery gathered from real-world sources not controlled by any eligibility criteria like those in RCTs. The NICE guidance suggests that RWD and RWE can be used for various purposes, such as to distinguish diseases, interventions, and patient outcomes, to design and endorse economic models, to validate digital health applications (cases of RWD used to develop clinical algorithms), to address health inequalities, and to assess the impact of interventions on care delivery, among others.(3)
The NICE RWE Framework is a part of NICE Strategy 2021 to 2026,(4) a five-year strategic plan focusing on the RWE use to fill evidence gaps, enhance NICE’s decision making, and facilitate patient access to innovative health technologies. The Framework will help identify the need to use RWE to limit uncertainties and advance guidance. The Framework thoroughly defines the best practices for designing, conducting, and reporting RWE studies to improve the quality and transparency of the evidence.(3)
The NICE RWE Framework outlines the role of RWE in health technology assessments (HTAs). It has a separate dedicated section on best practices for comparative effectiveness studies as they have more refined considerations requiring higher precision. The Framework also offers the Data Suitability Assessment Tool (DataSAT) for assessing the data’s relevance and validity for the intended purpose. The goal of DataSAT is to compel researchers to justify the choice of the RWD source. Like other frameworks provided by FDA and EMA, the DataSAT focuses on the origin, quality, and reliability of data. The Framework also recommends that researchers follow the ‘target trial’ approach while designing an RWE study to pursue all measures to limit bias, control the confounders, and assess the robustness of the findings.(5)
Although the NICE RWE Framework is at par with the other guidance documents by the FDA and EMA, it still has some gaps. For instance, it does not define the minimal criteria for acceptance of RWE study elements. Also, not all the RWE study elements are mentioned; therefore, more detailed processes and checklists are required for more comprehensive guidance.(5)
It is expected that the NICE RWE Framework will keep updating with evolving processes and methodologies. In addition to the existing frameworks by other regulators such as the USFDA,(2) the NICE RWE framework will be an excellent resource for developing RWE studies, particularly comparative effectiveness studies. NICE encourages the submission of RWE studies with an open dialogue with the pharmaceutical companies. Transparency and accountability are central components of the RWE Framework as it enables researchers to provide all the possible documentation and justification of the RWE study design and execution.(5)
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