Pharma groups suggest modification to FDA’s CGT postapproval guidance

Pharmaceutical industry groups are asking the US Food and Drug Administration (FDA) to revise its draft guidance on postapproval methods to capture safety and efficacy data on cell and gene therapies (CGT) to include a broader registry system that can collect postapproval data on these products. This data should be gathered by a variety of public and private organizations, not just clinical professional societies that currently collect postapproval data.

Additionally, some commenters also recommended incorporating references to the International Council on Harmonisation’s (ICH) M14 guidance, requested more information on decentralized data collection, and requested more clarity on the scope of guidance.

The draft guidance, issued in September 2025, addresses the different approaches that can be used to capture postapproval data, such as the use of real-world data (RWD) and real-world evidence (RWE), the use of electronic health records (EHRs), and the use of registries and decentralized data collection. (RELATED: FDA issues guidances on CGT, regenerative medicine development, Regulatory Focus 25 September 2025)

The guidance emphasizes that collecting postapproval study data for gene and cell therapy (CGT) products is crucial. This is because the number of patients receiving CGT products during pre-marketing clinical development is usually limited.

The Pharmaceutical Research and Manufacturers of America (PhRMA) said that it “applauds FDA for outlining various methods and approaches for capturing postapproval safety and efficacy data for CGT products in the Draft Guidance. PhRMA and its members recognize that guidance, templates, and frameworks are critical to collecting real world data (“RWD”) and generating real-world evidence (“RWE”) for CGT products”

CRNs vs. FDNs

Yet pharmaceutical industry groups and drugmakers also suggested that there is room for improvement. PhRMA and the Biotechnology Innovation Organization (BIO) recommended that FDA replace the term “Coordinated Registry Networks” with “Federated Data Networks” to encourage their broader use among stakeholders for data collection.

PhRMA stated that “FDA’s discussion of registries as a data source focuses on Coordinated Registry Networks (“CRNs”). Registries can, however, be single site, multi-site, single country, or international, and can come in the form of a Federated Data Network (“FDN”) based on multiple local databases. For this reason, PhRMA recommends that FDA replace the term “Coordinated Registry Network” with the broader term “Federated Data Network” throughout the Draft Guidance.”

BIO concurred, stating that it recommends “replacing the term Coordinated Registry Network with the broader term Federated Data Network throughout the guidance. While Coordinated Registry Networks typically refer to registries established by clinical professional societies, Federated Data Networks encompass distributed data systems that can be developed and maintained by a wider range of public and private entities, including pharmaceutical companies, health authorities, and groups of clinicians. Using the broader term better reflects the diversity of data infrastructure models that support RWE generation.”

Registries over other RWD sources?

Kite Pharma contests the agency's claim in its guidance that registries have advantages over other sources of real-world data (RWD), such as medical claims or electronic health record (EHR) datasets. The company said the guidance downplays the challenges registries face in collecting long-term safety follow-up data, including incomplete data capture, inconsistent reporting, and patient attrition.

Kite said this characterization “does not reflect current advances in EHR interoperability and data standardization. These advances have already enabled early applications of EHR-derived RWD and the use of real-world evidence to satisfy postmarket requirements or commitments within CBER in fiscal year 2024. FDA’s Biologics Effectiveness and Safety Initiative (BEST) has also piloted direct data exchange for post-vaccination adverse event monitoring, demonstrating growing feasibility for broader future use.”

The company recommended the agency provide a more “balanced view of registries’ strengths and weaknesses.”

ICH M14

Both PhRMA and IQVIA suggested that the guidance incorporate references to the ICH M14 guideline, which was adopted in September. (RELATED: ICH adopts M14 guideline on observational studies, Regulatory Focus 15 September 2025)

The ICH guideline aims to harmonize the planning, design, and reporting of pharmacoepidemiologic studies that use real-world data (RWD) to evaluate the safety of marketed drugs and vaccines. The guideline also provides high-level best practices for conducting these studies.

IQVIA noted that the ICH guideline also “provides a thorough discussion of how to assess study feasibility, regardless of the data source, whereas the draft guidance only mentions assessing feasibility in the subsection on use of electronic health records, medical claims, and vital statistics data.”

Decentralized data collection

PhRMA and the Parenteral Drug Association (PDA) requested clarification on how to use a decentralized model for collecting postapproval data for CGT products.

“PhRMA recommends that FDA provide additional guidance for sponsors collecting data through hybrid trials and/or cross reference existing guidance, where applicable. For example, FDA could outline best practices for sponsors using centralized or clinical site-based and decentralized or remote-based data collection within the same study,” the group wrote.

PDA said that the guidance should add discussion on the use of digitally self-reported patent data. This can include smart phone applications “where patients input specific data on a routine basis without the need to visit a facility.”

Clarity on scope

BIO and IQVIA also requested more clarity on the scope of the guidance such as whether the guidance primarily addresses long-term follow-up (LTFU) obligations following approval under a traditional pathway, or whether it covers mandated data collection such as via a post-marketing requirement for products approved under accelerated approval pathways.

“If the guidance is intended to address both, we recommend that the text explicitly differentiates the implementation of these methods depending on the approval type, should they differ,” BIO wrote.

IQVIA also wrote that “the Introduction section of the draft guidance would benefit from clearer articulation of the document’s overall scope, which is foundational to interpreting later sections on data sources and collection approaches.”

The group added that “this ambiguity makes it difficult for sponsors to determine which patient populations FDA expects to be covered by this guidance, whether and how different cohorts could be integrated across the product lifecycle, and the type of data collected beyond long-term safety (e.g., durability).

Comments

Pharma groups suggest modification to FDA’s CGT postapproval guidance

Related topics

Recommended content

Welcome to the new RAPS Digital Experience