Working Groups Report – Q2 2025

We had two short projects this quarter from two of our Working Groups:

The Optimizing the Use of Data Standards Working Group formed a team to provide a PHUSE response to the FDA Federal Register notice requesting feedback on the potential adoption of Dataset-JSON as a new exchange standard.

The FDA was seeking comments on whether to accept Dataset-JSON for regulatory applications, specifically regarding:

·      The risks and benefits of industry adoption of Dataset-JSON as a new exchange standard.

·      Any integration challenges with existing tools and systems.

The Real World Evidence Working Group formed a team to provide a PHUSE response to the FDA Federal Register requesting comments on HL7 FHIR for Submission of Data Collected from Real-world Data Sources.

The U.S. Food and Drug Administration (FDA) has recently published a Federal Register Notice exploring the Health Level Seven (HL7) Fast Healthcare Interoperability Resources (FHIR) for submission of data collected from real-world data (RWD) sources. Read the full notice here.

We have two more published deliverables this quarter:

Optimizing the Use of Data Standards

Dataset-JSON as Alternative Transport Format for Regulatory Submissions

• The Dataset-JSON as Alternative Transport Format for Regulatory Submissions project have published their Business Case. 

Dataset-JSON is a widely accepted data format that will streamline data exchange between organisations. Moving to Dataset-JSON will prepare us for the current digital world and enable companies to more accurately, completely and efficiently represent their clinical research data, to better serve their patients/customers. Dataset-JSON supports current data standards but also allows for standards to evolve and removes current limitations imposed by xpt format.

Implementation of Estimands (ICH E9 (R1)) using Data Standards

• The Implementation of Estimands (ICH E9 (R1)) using Data Standards have published their white paper and Example Document!

This white paper provides recommendations and examples to illustrate the implementation of the estimands framework introduced by the ICH E9 (R1) Addendum on Estimands and Sensitivity Analysis in Clinical Trials to the Guideline on Statistical Principles for Clinical Trials [ICH E9 (R1), 2019] using data standards (as part of the clinical data flow).

• The RWD Guideline for Programming and Analysis Processes project within Real World Evidence Working Group, has a White Paper which will soon going out for public feedback.

• Dataset-JSON as Alternative Transport Format for Regulatory Submissions 

• FDA Requests Public Comment on CDISC Dataset-JSON Standard 

• Implementation of Estimands (ICH E9 (R1)) using Data Standards