The pharmaceutical industry is calling on the FDA to modernise its vision for tracking the long-term safety and efficacy of cell and gene therapies (CGT).
Following the draft guidance released by the agency in September 2025, major industry groups like PhRMA and the Biotechnology Innovation Organization (BIO) argue that the current focus on traditional, society-led registries – which rely on manual data entry by clinicians into niche, professional databases – is too restrictive for modern medicine. Instead, they suggest a more flexible, automated framework is required to ensure these high-tech gene therapies are monitored effectively.
A shift toward federated networks
The central recommendation is a shift toward federated data networks (FDNs). Unlike traditional registries, which are often siloed within specific clinical professional societies, FDNs allow data to remain in local databases while being accessed and queried collectively. This model encourages collaboration between pharma, private healthcare organisations, and international health authorities.
PhRMA emphasised the importance of this shift toward a more inclusive data ecosystem, recommending that the FDA replace the term “coordinated registry network” with the broader term “federated data network” throughout the draft guidance. In its comments, the organisation noted that registries may be single-site, multi-site, national, or international, and can take the form of a federated data network built on multiple local databases. As such, the group argued that using the broader term would more accurately reflect the range of registry models capable of supporting post-approval data collection.
International alignment and efficiency
Beyond terminology, PhRMA is pushing for better alignment with international standards like the International Council for Harmonisation’s ICH M14 to ensure safety and efficacy evidence is globally accepted. By establishing a harmonised international framework for regulatory-grade, real-world reporting, ICH M14 enables sponsors to use automated electronic health record feeds and smartphone-based patient reports to satisfy multiple regulators at once, drastically reducing the administrative burden on busy clinics.
BIO echoed this view, noting that the evolution toward FDNs is essential for a diverse and global research landscape: “Using the broader term better reflects the diversity of data infrastructure models that support RWE generation. Federated data networks encompass distributed systems that can be maintained by a wider range of public and private entities, including pharmaceutical companies, health authorities and groups of clinicians.”
For the medical community, these updates could transform post-approval tracking from a rigid task into a flexible, technology-driven ecosystem that secures the future of CGT.
