The U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) hosted a virtual public listening meeting on September 18th. During this meeting, stakeholders from regulatory agencies, patient advocacy organizations, industry, and academia convened to discuss how prior knowledge and platform approaches could be better leveraged to accelerate the development and approval of cell and gene therapies (CGTs). The group explored whether a more flexible, risk-based regulatory framework could support smarter use of existing data, reduce duplicative data generation, and streamline product development across multiple indications. 

Participants also discussed ways to reduce costs, enhance collaboration, and expand the use of real-world data (RWD) and registries. The concept of a platform technology designation (PTD) was repeatedly referenced, with emphasis on the need for early, structured engagement with FDA to align on what prior knowledge can be leveraged across products using similar manufacturing, delivery, or therapeutic mechanisms. 

Why does this matter for patients and drug development? 

CGTs have the potential to transform outcomes for patients with serious, often life-threatening rare diseases, but their development is incredibly expensive and time intensive. By leveraging platform technologies and shared data, sponsors could avoid repeating preclinical studies, reduce animal testing, and repurpose validated manufacturing processes. This not only would reduce costs but could also bring promising therapies to patients faster. Advocates stressed that using external control arms, shared registries, and natural history data would provide context for clinical trials, especially in small, heterogenous populations where randomized controlled trials may not be feasible or ethical. 

Patient representatives called for increased transparency in trial design, improved data sharing among stakeholders, and the elimination of excessive placebo or control arm use when baseline natural history data already exists. Speakers also emphasized the need for novel endpoints and patient-reported outcomes that reflect what matters most to patients. 

Discussions were organized into three main sessions, each tackling a different dimension of leveraging prior knowledge and platform approaches in gene therapy development. 

Session 1: Speakers emphasized the need for a flexible, risk-based approach to regulatory review, where the amount and type of prior knowledge that can be leveraged depends on the degree of biological change to the product, expected impact on safety and efficacy, and remaining uncertainty. 

Key proposals included: 

• Linking prior Biologics License Applications (BLAs) or Investigational New Drug (IND) applications for sponsors under a formal PTD framework, rather than requiring sponsors to resubmit information in a new BLA.   

• Reducing redundant analytical and toxicology testing if data from similar or identical constructs already exist. 

• Creating an open line of communication with FDA to discuss what prior work can be leveraged across submissions. 

Stakeholders also stressed that shared registries and natural history studies could reduce duplication, serve as external controls, and redefine what constitutes meaningful clinical endpoints in rare and degenerative diseases. 

Session 2: The second session focused on the infrastructure and regulatory innovations needed to support platform development and knowledge sharing. Participants proposed: 

• Early platform meetings with FDA to define what data can be leveraged across programs.  

• Creation of platform master files (PMFs), cloud-based systems that sponsors and reviewers can reference across multiple INDs and BLAs. 

• Development of new guidance documents to clarify when and how prior knowledge from CGT platforms can be used. 

Speakers called for formal criteria to define platform technologies, especially for gene editing approaches that can be applied across diseases. They also proposed master protocols and shared clinical infrastructure to reduce costs and expedite trials for commercially challenging indications. 

Other speakers suggested launching a public genomic medicine knowledge hub to house standard assays, off-target data, and clinical outcomes, potentially partnering with FDA’s recently proposed Rare Disease Evidence Program (RDEP) to foster collaboration between regulators and developers. 

Session 3: The final session focused on how better data sharing and modernized safety monitoring can further streamline development. 

• One speaker noted that while sponsors are generally willing to share safety and efficacy data post-approval, they are cautious of sharing interim data or proprietary primary efficacy results prior to publication or regulatory decision-making. 

• Speakers encouraged FDA to create confidential, anonymized data hubs that could pool comparative data across sponsors.   

• Speakers proposed using unified registries and simplified post-marketing surveillance, run by neutral entities, to ensure safety monitoring is less burdensome and more patient-centric. 

Meeting outcomes and implications. 

The discussions showed broad agreement that leveraging existing knowledge and platform approaches is key to accelerating CGT development, especially in rare diseases where time and resources are limited. Stakeholders urged FDA to formalize new tools and frameworks— such as PTDs, PMFs, and early platform meetings— to provide clarity and efficiency in clinical development. 

Key takeaways included: 

• The need for a flexible, risk-based regulatory framework for developing therapies, with tailored requirements depending on the degree of biological change, level of potential impact on safety and efficacy, and the prior evidence available. 

• Strong support for public-private collaboration, including registries, consortia, and knowledge-sharing platforms. 

• The importance of transparent communication between regulators, sponsors, and patient communities to align on meaningful endpoints and reduce duplication. 

Participants repeatedly emphasized that these reforms are not just about saving time or money, rather, about enabling effective treatments to reach patients faster, especially in cases of high unmet need. As one speaker emphasized, we shouldn’t be starting from scratch every time we try to help the same patient community. 

These conversations reflect and reinforce key priorities in Friends’ ongoing Real-World Data Portfolio and ECA project, both of which aim to create clear, patient-centered pathways for the use of real-world and historical data in drug development and regulatory decision making. Additionally, concepts outlined in discussions and white papers from Friends’ Cell and Gene Therapies work, including leveraging prior knowledge and sharing learnings, align with sessions throughout the day. Friends provided comments to the public docket for this meeting here.