– Written by Matt Baldwin, Biomedical Data Stewardship Senior Manager at Amgen and PHUSE Working Group member

Commonly accepted clinical data standards for regulatory submission purposes, namely CDISC, have evolved over the last three decades, with input from a multistakeholder community. This moment in time is no different, as evaluations are in progress on many fronts to continue innovating and finding solutions. Two areas being tackled by PHUSE project teams include estimands implementation and submitting real-world data (RWD).

The PHUSE Optimizing the Use of Data Standards Working Group launched a project team in 2022 Q1 to address Implementation of Estimands (ICH E9 (R1)) using Data Standards. The goal from the start was to develop a white paper with guidance for industry. After almost two years of discussions, the draft white paper, including an appendix example, has completed a public review period and is being finalised for publication. This paper provides recommendations to facilitate the implementation of the estimands framework, introduced by the ICH E9(R1) addendum, within the data flow, promoting alignment across the industry. The progress and summarised content of this effort was shared at numerous events in 2023, including the PHUSE US Connect, multiple PHUSE Single Day Events (SDEs), the DIA Global Annual Meeting, the PHUSE CSS, CDISC US Interchange and the PHUSE EU Connect. The Implementation of Estimands (ICH E9 (R1)) using Data Standards project team also conducted a 2023 Q1 survey on estimands implementation, and a summary of the results is publicly available. The survey results can assist future PHUSE efforts on this topic. For access to detailed survey results, please send a request to workinggroups@phuse.global.

The PHUSE Real World Evidence Working Group launched a project team in 2023Q2 to address Submitting Real World Data. The project deliverables are still coming into focus as meaningful discussions occur, so now is the time to get involved! A PHUSE Community Forum on the topic of Experiences with Adapting RWD to CDISC Submission Standards (webinar details and recording) took place on 5 October 2023. The conversation continues to build on the foundational work of the PHUSE Real World Evidence Working Group, summarised in a May 2023 white paper on Real-world Evidence: Requirements and Recommendations for Regulatory Submissions. An important contribution that must be accounted for is the recently finalised FDA guidance on Data Standards for Drug and Biological Product Submissions Containing Real-World Data in December 2023. Instead of summarising or providing selective content here, click through the various links for more details. For those involved in this space, it’s obvious that this is just the beginning of considerations around submitting RWD, and there is so much more to come.

As you can see, a common theme among the topics highlighted above is the underlying use of clinical data standards, and in these cases specifically CDISC. Data standards are constantly adapting and improving as needs arise, but the collaborative, multistakeholder journeys are often not quick or straightforward. However, the benefits of solutions to both old (estimands) and new (RWD) problems are certainly worth the effort. Be on the lookout across the industry, including PHUSE Working Group project teams, for more on estimands implementation and submission of RWD as it relates to data standard enhancements. Who knows, maybe one day these topics will overlap, and we’ll be discussing estimands implementation for submission of RWD! But let’s not get ahead of ourselves. One step at a time…