Pharmaceutical industry groups are urging the US Food and Drug Administration to refine its draft guidance on postapproval data collection for cell and gene therapies (CGTs), citing the need for greater flexibility, clarity, and alignment with evolving real-world data practices. The comments highlight growing industry concern that existing frameworks may not fully support the long-term safety and effectiveness monitoring required for these complex therapies.
The draft guidance, issued in September 2025, outlines approaches FDA expects sponsors to use when collecting postapproval data, including real-world data and evidence, electronic health records, registries, and decentralized data collection models. FDA emphasized that robust postapproval monitoring is critical for CGTs, given the limited patient populations typically enrolled in pre-approval clinical trials.
Calls for Broader Registry Models
While industry groups broadly supported FDA’s recognition of multiple postapproval data sources, several stakeholders argued that the guidance places too much emphasis on registries operated by clinical professional societies. Pharmaceutical companies and trade groups recommended expanding the framework to explicitly support broader, more flexible data infrastructures.
Both PhRMA and BIO suggested replacing the term “Coordinated Registry Networks” with “Federated Data Networks” throughout the guidance. They argued that federated models better reflect how postapproval data are increasingly collected, allowing distributed data systems maintained by pharmaceutical companies, regulators, healthcare systems, and other public and private entities to contribute to real-world evidence generation.
According to commenters, this broader terminology would encourage innovation in data collection while better reflecting the diversity of existing and emerging data infrastructures.
Balancing Registries With Other Real-World Data Sources
Some sponsors also challenged FDA’s characterization of registries as having inherent advantages over other real-world data sources, such as medical claims and electronic health record datasets. Industry feedback emphasized that registries face challenges of their own, including incomplete data capture, inconsistent reporting, and patient attrition over long follow-up periods.
Advances in EHR interoperability and data standardization, commenters noted, have already enabled successful use of EHR-derived real-world evidence to support postmarket obligations. Several stakeholders urged FDA to present a more balanced view of the strengths and limitations of each data source rather than favoring one model.
Alignment With ICH M14 and Decentralized Data Collection
Multiple commenters recommended that FDA align its guidance more closely with the recently adopted ICH M14 guideline, which focuses on the planning and conduct of pharmacoepidemiologic studies using real-world data. Stakeholders noted that ICH M14 provides a more comprehensive discussion of feasibility assessment across data sources than the FDA draft guidance.
Industry groups also requested additional clarity on decentralized and hybrid data collection models. Sponsors asked FDA to outline best practices for combining site-based and remote data collection, including the use of digitally self-reported patient data through mobile applications and other tools.
Clarifying the Scope of Postapproval Expectations
Finally, several organizations requested clearer articulation of the guidance’s scope. Commenters noted ambiguity around whether the guidance primarily applies to long-term follow-up following traditional approvals or also extends to postmarketing requirements under accelerated approval pathways. Clear differentiation, they argued, would help sponsors design appropriate data collection strategies across the product lifecycle.
What This Means for CGT Developers
The feedback reflects a broader industry push for postapproval frameworks that are flexible, risk-based, and aligned with global real-world evidence standards. As CGT pipelines continue to grow, sponsors will need regulatory strategies that balance innovation in data collection with FDA’s expectations for safety, durability, and effectiveness monitoring.
How EMMA International Supports Postapproval Strategy
At EMMA International, we support cell and gene therapy developers with postapproval strategy, real-world evidence planning, pharmacovigilance alignment, and lifecycle compliance across FDA and global regulatory frameworks. Our teams help sponsors design data collection approaches that are scientifically sound, operationally feasible, and regulator-ready.
For more information on how EMMA International can assist, visit www.emmainternational.com or contact us at (248) 987-4497 or info@emmainternational.com.
Reference:
U.S. Food and Drug Administration. Draft guidance on postapproval safety and efficacy data collection for cell and gene therapies. September 2025.
Pharmaceutical Research and Manufacturers of America. Public comments on FDA draft CGT postapproval guidance. 2025.
International Council for Harmonisation. ICH M14: Guideline on observational studies using real-world data. September 2025.
