Jul 25
2025
news
Regulatory Focus — FDA white paper encourages adoption of selective safety data collection
Joanne S. Eglovitch
The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) this week released a white paper aimed at encouraging the use of selective safety data collection (SSDC).
This approach is designed to simplify the data gathering process for drugs that have well-established safety profiles with the goal of reducing the collection of non-serious adverse events (AEs) that are unlikely to provide significant additional insights into drug safety.
The paper includes examples of trials that have included SSDC and describes the International Council for Harmonization’s E19 guidance on SSDC.
The ICH E19 guideline, which was adopted in September 2022, describes a risk-based approach for collecting safety data for late-stage pre-approval or post-approval studies when the safety of a drug is well-understood. (RELATED: ICH advances guidelines on selective reporting of safety data, viral safety, Regulatory Focus 11 October 2022).
Since the adoption of the guideline, FDA noted in the white paper that “the community does not know the extent to which sponsors have applied SSDC to clinical trials, the therapeutic areas, the settings (i.e., pre-approval or post-approval), the challenges sponsors have encountered (including agreement with regulatory authorities), and the efficiencies that have been observed in trials using SSDC.”
This lack of awareness on SSDC was also observed during an FDA-led scientific session of the 2024 Drug Information Association annual meeting, where representatives from FDA, Health Canada, the European Medicines Agency, and industry (Novartis) discussed their experience with implementing the E19 guideline. “Although the session generated much interest from the audience, it was apparent that unawareness of E19 signals the need for broader dissemination of the guideline and shared lessons learned from sponsors and regulators who have experience with SSDC in clinical trials,” FDA wrote.
The paper refers to a 2022 study conducted by Yuki Yamatani and colleagues, which evaluated the extent to which SSDC has been incorporated into clinical trials. The authors performed a literature search of clinical trials published in the New England Journal of Medicine from February 2016 to December 2019. They identified 459 trials published during this time frame. Among these, 44 trials, accounting for 9.6%, included one or more features identified as SSDCs as described in the ICH E19 guideline. The majority of the trials focused on cardiovascular diseases, comprising 54.5%, followed by trials on infectious diseases at 18.2%.
The paper also includes two studies that demonstrated successful use of SSDC, the Pragmatica-Lung study, and the VICTORION-2-PREVENT study.
The Pragmatica-Lung Study, was a randomized phase 3 trial assessing the combination of Cyramza (ramucirumab) and Keytruda (pembrolizumab) compared to standard of care treatments in patients with advanced non-small cell lung cancer (NSCLC). The two drugs have been well-studied with known side effects, and the study minimized the data reporting requirements by reducing the time point data required, the number of forms needed, and the number of data elements required to be reported within a form.
The VICTORION-2-PREVENT study was a randomized double-blind placebo-controlled multicenter trial that assessed the impact of inclisiran on major cardiovascular events in patients with cardiovascular disease. “The US approval was based on three placebo-controlled trials that included 1,833 patients treated with inclisiran, including 1,682 exposed to the drug for 18 months. Although the trial is being conducted post-approval, it was initiated before approvals, as knowledge from the large phase 3 programs enabled the sponsor and FDA to agree that the risks of inclisiran were sufficiently characterized to apply SSDC,” FDA wrote.