- 22 Mar 2022
Executive Summary
England’s health technology appraisal body, NICE, will publish a framework that will help it deliver on its ambition to use real-world evidence to close knowledge gaps and improve technology evaluations.
Source: Alamy
NICE, the health technology appraisal institute for England and Wales, is preparing to publish its proposed framework on real-world evidence (RWE) for consultation in April. Stakeholders have so far provided positive feedback on the framework but would like more transparency on how RWE is used in formulating recommendations, the institute said.
NICE first announced it would be making greater use of real-world data (RWD) in a statement of intent published in January 2020. RWD, which is generated outside of controlled trials from real world settings, such as electronic health records, and which is used to generate RWE, also featured in NICE’s five-year strategy for 2021 to 2026. (Also see “UK: RWD At Center Of NICE Five-Year Strategy” – Pink Sheet, 21 Apr, 2021.) This strategy emphasized that RWD is integral to NICE’s “strategic ambitions,” which include rapid, robust and responsive technology evaluations, dynamic living guideline recommendations, effective guidance uptake, and leadership in data, research and science.
To help deliver these ambitions, NICE has developed a Real-World Evidence Framework. At a board meeting on 22 March, Felix Graves, director of science, evidence and analytics at NICE, offered an update on the framework, which will be published for consultation in the coming weeks.
According to NICE, the framework will help identify where RWD can be used to reduce uncertainties and improve guidance. It will also clearly set out best practices for planning, conducting and reporting RWE studies to improve the quality of evidence.
The framework is aimed at stakeholders that develop evidence for use in NICE guidance, including manufacturers, third parties and NICE staff and collaborators. It will help ensure that real-world studies are “undertaken with integrity and reported transparently, following best practice methods,” NICE said.
However, it will not impose minimum standards for RWE studies. “We recognize that the acceptability of a specific study will depend on the use case, the NICE program, and a range of other contextual factors such as decision uncertainty,” it declared.
Core Principles And Content
The framework sets out certain principles that should be followed in order to generate high-quality and trusted RWE:
- Ensuring data is of demonstrable provenance, relevant and of sufficient quality to answer the research questions.
- Ensuring evidence is generated transparently and with integrity from study planning to conduct and reporting of the study.
- Using analytical methods to minimize the risk of bias and characterize uncertainty.
It will include a section dedicated to study conduct, which sets out best practices for planning, conducting and reporting primary RWE studies.
Another section will address data suitability and describe the information needed to assess data provenance and fitness for purpose for specific research questions.
Methods for studying comparative effects will also be covered. This section details recommendations and considerations for conducting non-randomized studies using RWD. These include traditional observational studies as well as clinical trials that use RWD to form an external control.
Feedback
The framework has already received comments from UK health system partners, including the medicines regulator, the MHRA, and international HTA bodies such as the Scottish Medicines Consortium, the All Wales Medicines Strategy Group and Sweden’s Pharmaceutical Benefits Board.
The Association of the British Pharmaceutical Industry and the Association of British Health Tech Industries are among the industry groups to provide feedback.
The framework has been positively received, according to NICE, although there have been calls for further work in some areas. For example, stakeholders want to see the framework made more accessible to those who are not experts in data or epidemiological research.
NICE also notes demand for additional expertise in review groups and committees to evaluate RWE. There have also been calls for greater transparency in how RWE informs recommendations and consistency in evaluation.
NICE says it plans to publish a first version of the framework as a “beta-version” in April for a four-week consultation. The framework will be revised according to the feedback and published in the summer.
Source: https://pink.pharmaintelligence.informa.com/PS145894/Englands-Real-World-Evidence-Framework-Coming-Soon