Working Group XIII – Real-World Data and Real-World Evidence in Regulatory Decision Making

Participants at the fifth meeting of the Working Group, held on the 15th and 17th of December 2020

Background

There is increasing interest in the use of Real-World data (RWD) to support regulatory decision making across the product life cycle. Key sources of RWD are electronic health records, claims data, prescription data, and patient registries. Real-world evidence (RWE) is the clinical evidence about the usage and potential benefits or risks of a medical product derived from an analysis of RWD. For decades, such evidence has been well accepted for satisfying post-approval safety monitoring requirements but has not been used commonly to demonstrate drug effectiveness, which is a relatively new concept.

As applications are increasing, it is proposed that CIOMS develop a consensus report and recommendations for the use of RWD and RWE in regulatory decision-making related to biopharmaceutical products. This report will cover three key areas:

• Articulate the different RWD/RWE requirements depending on the intent of use e.g. Regulatory, Payers, and Public Health;
• Propose harmonized practices and guidance for using RWD and RWE for regulatory purposes (given that there are no existing consensus guidelines);
• Articulate point of view (POV) on key ethical issues relevant to RWD and RWE and a provisional set of standards to address those issues (the high-level POV may lead to a separate/satellite group of ethicists to deal with it).

Full Concept Note (31 March 2020)

Working Group objectives

The CIOMS guidance would create a reference worldwide for researchers and regulators involved in RWD/RWE studies, as well as pharmaceutical and biotechnology companies involved in product development and marketing.

The primary remit of the proposed CIOMS WG would be to develop, for global use, a consensus report and recommendations on principles to be applied regarding triggers, objectives, research questions, design features, and timing of RWD and RWE as part of the regulatory process for products in the peri-approval stage of development or for authorized products.

The working group would also be expected to evaluate the increasingly novel methodologies that are being used to design studies, collect and analyse data, and use the output in the regulatory process to assess how RWE can contribute to effectiveness decisions.

Supporting this, the proposed CIOMS WG would recommend an approach and prepare a consensus report promoting harmonization across global drug regulatory authorities and develop a POV on ethical considerations and challenges related to RWD and RWE.

Progress

• 1st meeting held on 30 March 2020 (virtual meeting) (Minutes)
• 2nd meeting held on 29 June 2020 (virtual meeting) (Minutes confidential)
• 3rd meeting held on 19 August 2020 (virtual meeting) (Minutes)
• 4th meeting held on 23-24 September 2020 (virtual meeting) (Minutes)
• 5th meeting held on 15 and 17 December 2020 (virtual meeting) (Minutes)
• 6th meeting held on 26 February 2021 (virtual meeting) (Minutes)
• 7th meeting held on 1 April 2021 (virtual meeting) (Minutes)
• 8th meeting held on 6 May 2021 (virtual meeting) (Minutes)
• 9th meeting held on 22 June 2021 (virtual meeting) (Minutes)
• 10th meeting held on 27 September 2021 (virtual meeting) (Minutes)
• 11th meeting held on 3 December 2021 (virtual meeting) (Minutes)
• 12th meeting held on 4 February 2022 (virtual meeting) (Minutes)
• 13th meeting held on 5 April 2022 (virtual meeting) (Minutes)
• 14th meeting held on 7 June 2022 (virtual meeting) (Minutes)
• 15th meeting held on 9 September 2022 (virtual meeting) (Minutes)
• 16th meeting held on 5 December 2022 (virtual meeting) (Minutes)
• 17th meeting held on 30 May 2023 (virtual meeting) (Minutes)
• 18th meeting held on 4-5 October 2023 (Geneva, Switzerland) (Minutes)
• 19th meeting held on 8 April 2024 (virtual meeting) (Minutes to follow soon)

Feedback

The Working Group (WG) makes minutes of its in person and virtual meetings public. Persons and parties not part of the Working Group (WG) are welcome to give their feedback on the WG work by sending their input to info@cioms.ch

CIOMS Working Groups usually take 2-4 years to finalize their consensus document and recommendations. Most groups hold two in-person meetings per year, with telework in between. The groups make use of collaborative efforts and capitalize on existing initiatives in order to provide output that is as comprehensive as possible, does not duplicate other efforts and has added value.