The real-world impact of National Institute for Health and Care Excellence's real-world evidence framework.

The international health and care landscape is undergoing sweeping changes, involving: the rapid pace of innovation in technologies [1,2]; improved quality and access to routine data sources [3,4]; and strong economic pressures driving the need to optimize resources [5]. These trends were accelerated by the global COVID-19 pandemic.Post pandemic, real-world data continues to deliver evidence with richer outcomes, more timely conclusions and populations more representative of those to which care will be delivered, including patients traditionally underrepresented in randomized controlled trial environments, or with unequal access to care. Recognizing the still substantial untapped potential, the National Institute for Health and Care Excellence (NICE) is looking to real-world data to support: quicker, more proportionate appraisals [6]; dynamic 'living' guidance with responsive updates [7]; to monitor and influence the uptake of its guidance [8]; and to manage uncertainty post evaluation for earlier access to technologies [9,10]. Broadly, the use of real-world evidence is hampered for two main reasons: the trustworthiness of real-world data and the trustworthiness of methods used to analyze that data. Non-systematic identification of data sources, unclear curation processes, and complex and opaque study designs lead to general concerns over data suitability for the research question of interest and risk of bias, particularly for studies of comparative intervention effects. Meanwhile, for those developing evidence, it may also be unclear what NICE considers to be a reasonable threshold of quality to support decision making [11]. NICE's Real-world evidence framework was therefore developed to provide a steer for 'what good looks like' in the design and reporting of real-world evidence and its use, encouraging both a more rigorous standard of evidence and more consistent appraisal of that evidence [11]. A separate document to NICE's methods and guidelines manuals, it provides more detailed advice on the identification of suitable data, and the conduct and reporting of real-world studies, without being overly prescriptive.The framework has a strong emphasis on supporting implementability with links to numerous case studies displaying the range of use cases in supporting NICE guidance. Also included are longer form vignettes exploring relevant academic work. Tools and resources are referenced or have been developed if not available externally. For example, the Data Suitability Assessment Tool (DataSAT) within the framework provides a reporting template to help developers report all the key elements of data suitability to sufficiently inform NICE committees.The RWE framework was welcomed by stakeholders, and now sits with several other guidance publications from international regulators and HTA bodies including the FDA [12], EMA [13], HAS [14], and CADTH [15] guiding the use of real-world data to support policy decisions.