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How OpenAI and the FDA Could Transform Drug Approval With AI

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A New Era for Drug Evaluation Is Emerging

Imagine a future where patients no longer wait years for innovative treatments to become available. That vision is closer to reality as OpenAI and the FDA explore ways to integrate artificial intelligence into the drug evaluation process. By leveraging advanced AI, both organizations hope to modernize regulatory review, potentially slashing approval timelines and improving patient access to groundbreaking therapies.

Inside the OpenAI-FDA Collaboration

Recent high-level discussions have brought together OpenAI, the FDA, and the Department of Government Efficiency to brainstorm AI-driven solutions for drug review. At the heart of these talks is a potential project called cderGPT, named after the FDA’s Center for Drug Evaluation and Research. With Jeremy Walsh, the FDA’s first AI officer, leading the initiative, the agency is signaling its commitment to technological innovation, even though formal agreements are still pending.

FDA commissioner Marty Makary has voiced concerns about the slow pace of current approval processes. He recently announced the completion of the FDA’s first AI-assisted scientific review, suggesting this could be the first of many. The agency is particularly interested in applying AI to areas with urgent need, such as diabetes and cancer treatment evaluations.

Opportunities and Skepticism

Stakeholders across the healthcare and biotech industries are keeping a close eye on these developments. Former FDA commissioner Robert Califf acknowledges that AI is already in use internally, mainly for support tasks. He notes, however, that AI currently plays a minor role in the vast and complex journey from drug discovery to regulatory approval.

  • Industry leaders believe AI can automate routine review tasks, speeding up the initial feedback cycle for drug applicants.
  • Rafael Rosengarten, CEO of Genialis, highlights the need for clear guidance on data quality and AI performance standards.
  • Some former FDA staff caution that today’s AI models may generate unreliable or "hallucinated" information, underscoring the necessity for rigorous validation.

This blend of optimism and caution reflects the need for a balanced approach: embracing efficiency while maintaining scientific integrity and patient safety.

AI and Existing Fast-Track Processes

The FDA already uses mechanisms like fast track and breakthrough therapy to expedite reviews for critical drugs. While these help speed up access, the agency believes that AI could further compress timelines without sacrificing thoroughness. Experts and advocacy groups, including PhRMA, emphasize that AI must be adopted thoughtfully, with a focus on patient outcomes, transparency, and continuous oversight.

Building Trust Through Research and Compliance

The FDA has been actively researching AI’s role in regulatory science, recently seeking partners to develop large language models for precision medicine. Meanwhile, OpenAI is working to ensure its technology meets federal security standards, including launching ChatGPT Gov and pursuing FedRAMP accreditation for ChatGPT Enterprise. These steps are crucial for handling sensitive government data and building public trust in AI-driven healthcare solutions.

Although the OpenAI-FDA partnership is still in its infancy, its potential impact on the pharmaceutical landscape is substantial. If technical, ethical, and legal hurdles are overcome, this collaboration could set a new standard for how life-saving treatments reach those in need.

Final Thoughts: Advancing Innovation, Guarding Public Health

OpenAI and the FDA stand at the forefront of a major transformation in healthcare. Accelerating drug approval with AI holds immense promise, but the process must be guided by prudence, transparency, and rigorous oversight. As technology and regulation converge, the ultimate goal remains clear: ensuring that innovations serve the best interests of patients and society.

Source: WIRED


How OpenAI and the FDA Could Transform Drug Approval With AI
Joshua Berkowitz May 8, 2025
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