NICE released a real-world evidence framework

On June 23^rd the UK’s National Institute for Health and Care Excellence (NICE) released a real-world evidence (RWE)  framework as part of its five-year (2021-2026) strategic plan. the framework is intended to be a “living document” which will develop over time as RWE recommendations and methodologies evolve. It will serve as guidance on how researchers could be looking at RWE sources to prove the value of technologies. This framework focuses on the use of real-world data to answer gaps in knowledge and drive further access to innovations for patients. The framework aims to improve the quality of real-world evidence to inform NICE guidance.

The predominant principles for the framework are

• Generating evidence in a transparent way
• Ensuring that the data is trustworthy and fit for the purpose
• Use of appropriate analytical methods that minimize the risk of bias and characterize uncertainty

The framework uses a broad definition of real-world data:

• Real-world data refers to data relating to patient health or experience, or care delivery collected outside of highly controlled clinical trials
• It can come from diverse sources including patient health records, administrative records, patient registries, surveys, observational cohort studies, and digital health technologies

RWE framework provides recommendations on three central premises:

1. Planning, conducting, and reporting RWE studies

1. Mainly for quantitative studies but can be applied to qualitative studies
2. Implies the importance of transparency and has asked researchers to demonstrate the journey from data sources to analytical datasets
3. Minimize the risk of bias and describe uncertainty
   • There is a methods reporting template developed for reporting on methods used to minimize the risk of bias

Also highlights:

• Patients should be consulted throughout all aspects of study planning and conduct (patient experiences)
• In certain scenarios, it may be more difficult to generate High-quality real-world evidence such as rare diseases, and some medical devices (including digital health technologies)
• Listed Common challenges in the evaluation of medical devices and rare diseases

2. Assessing data suitability

1. Assessing data provenance
2. Quality of the data- completeness, and accuracy of key study variables
3. Relevance of quality to address specific research questions
4. Acceptability of evidence will depend on the type of evidence and relative factors

As a part of guidance to evidence developers, NICE has developed Data Suitability Assessment Tool (DataSAT) to help consistent and structured information on data suitability.

3. Methods for real-world studies of comparative effects

Non-randomized studies can be used to provide evidence on comparative effects in the absence of randomized controlled trials such as cohort studies, self-controlled studies, cross-sectional studies, case-control studies, and quasi-experimental studies.

What does this mean for researchers and pharma/biotech companies?

• RWE Framework is a great resource for researchers developing RWE studies
• Companies can reach out to NICE to seek help on using the framework ‘effectively’ as well as the institute’s scientific advice program
• NICE International will assist other countries in improving healthcare decision-making. Hence, regulatory bodies across the globe can use the NICE RWE framework as part of their methods or as a reference to develop their own framework.

Reference:

NICE real-world evidence framework  Corporate document Published: 23 June 2022 www.nice.org.uk/corporate/ecd9