ICH reflection paper signifies steps towards global convergence on real-world data and real-world evidence

The reflection paper aims to harmonise terminologies and enable the convergence of general principles for planning and reporting studies around real-world data and real-world evidence to support regulatory decision-making.

The past few years have seen a surge in the role of real-world data (RWD) and real-world evidence (RWE) in health care assessment and decision-making. Accordingly, several initiatives have been launched by regulators and health technology assessment bodies towards creating standards and frameworks for the appropriate use of RWE. There have also been several speculative papers discussing the move from fragmented RWE recommendations to comprehensive guidance.

However, before such guidance is developed, several challenges remain that limit the broader use of RWE. These include lack of standardisation of RWD/RWE formats and terminology, heterogeneity of data quality across RWD sources, and inconsistency in study designs.

To address this need, the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) has published a reflection paper that builds on the statement from the International Coalition of Medicines Regulatory Authorities (ICMRA) released in 2022. Co-sponsored by the EMA, the US FDA and Health Canada, the goal of the paper is to potentially inform future ICH guidelines on the use of RWD and RWE, which could include best practices for data quality, data standards for RWD and appropriate applications of study designs and data analyses.

“By supporting the delivery of a regulatory system able to integrate RWE in a more harmonised way into submissions for medicines approval and decision-making, this proposal can support timely decisions on the development of innovative treatments, help to address unmet medical needs and support the safe and effective use of medicines.”

The ICH plan to take a strategic approach towards addressing the challenges associated with integration of RWE into regulatory submissions and decision-making. As noted in the reflection paper, the steps toward harmonisation include:

• convergence of the terminology for RWD and RWE
• alignment on the format for protocols and reports of study results submitted to regulatory agencies throughout the lifecycle of medicinal products
• promotion of registration of protocols and reports

European stakeholders, including regulators, academic researchers and the pharmaceutical industry, are encouraged to provide feedback on the paper by 30 September 2023. This will “ensure that all relevant parties are informed and given the opportunity to bring forward their views on technical and operational aspects to be considered when addressing harmonisation of the different focus areas”.