FDA Policy/General

GAO Faults FDA Organ-on-a-Chip Regulation

David McFarland

— 1 min read
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A Government Accountability Office (GAO) technology assessment report on human organ-on-a-chip (OOC) technologies says an issue facing the development of such technologies is unclear guidance and messaging from FDA. The report says OOC is an emerging technology that incorporates human adult cells in a small laboratory device to mimic how organs such as the brain, heart, or lungs work. “Researchers are developing and using OOCs to assess aspects of drug safety and efficacy,” GAO says. “Currently, OOCs cannot replace animal testing but may be used alongside animals.”

The report identifies four challenges to developing and using OOCs:

  • obtaining human cells of sufficiently high quality;
  • lack of benchmarks and validation studies;
  • limited data sharing; and
  • regulatory uncertainties.

In its discussion of regulatory issues, GAO says FDA officials reported that they were working to better understand OOC performance from a regulatory perspective, including how to interpret results and understand reproducibility. “However,” the report says, “FDA officials told us that having trained staff to evaluate and interpret OOC data is the largest barrier the agency faces with regards to OOCs, given the newness and rapidly changing nature of the technology. The agency is evaluating various OOCs for the potential regulatory utility based on specific contexts of use, including their benefits and limitations, and engaging in workshops with developers and end users.”

The agency released a roadmap in 4/2025 that described how it might reduce its reliance on animal testing for drug safety studies in favor of validated alternatives such as OOC. “Although FDA has published recent statements communicating its support for replacing and reducing animal testing where appropriate, and using alternatives to animal studies such as OOCs, the agency has not published guidance specific to OOCs,” GAO says…. “Selected developers and end users told us they do not fully understand which data they should submit to FDA for regulatory submissions.

The GAO report lists six policy options that it says could help mitigate challenges to OOC development and use:

  • provide additional regulatory guidance;
  • support efforts to increase access to diverse, high-quality human cells;
  • support standards development;
  • encourage more research and development of benchmarks and validation studies;
  • create or participate in mechanisms for data sharing; and
  • maintain the status quo.

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