AI-Enabled Optimization of Early- Phase Clinical Trials Pilot Program; Request for Information

The commenter, representing an industry entity, supports the proposed pilot program as a way to drive efficiency and industry-wide adoption of real-time clinical trials. They argue that the pilot builds on successful previous standards-based regulatory advancements and will significantly reduce sponsor burden while improving data quality and integrity through AI-enabled automation.

OmniScience, Inc. expresses strong support for the FDA's AI-Enabled Optimization of Early-Phase Clinical Trials Pilot Program, highlighting its potential to shorten drug development timelines and reduce costs. The company offers to serve as a technology partner and provides specific recommendations on pilot design, including prioritizing high-value use cases, ensuring diverse participant representation, and establishing interoperability standards.

Sthenic Labs is submitting a formal response to the RFI, proposing a hardware-rooted architecture called the "Sthenic Protocol" to ensure data integrity in decentralized clinical trials. They argue that their technology provides a "physical root of trust" to prevent synthetic fraud and AI-generated data injection, enabling safer and more reliable AI-driven dose optimization.

PhRMA, representing the pharmaceutical research and manufacturing industry, supports the FDA's proposed pilot program to explore AI-enabled optimization in early-phase clinical trials. They advocate for a risk-based approach to AI validation, the protection of confidential commercial information, and the use of established practices to share learnings from the pilot.

The commenter supports the FDA's pilot program for AI in early-phase clinical trials and emphasizes the need for continuous monitoring of AI models throughout their lifecycle. They also recommend prioritizing reproducibility, measuring both operational and clinical outcomes, and ensuring the pilot includes organizations of varying sizes to promote scalability.

Novartis Pharmaceuticals Corp. supports the FDA's initiative to explore AI-enabled real-time clinical trial data submissions to improve drug development efficiency. They recommend focusing the pilot on identifying use cases that reduce pauses between development phases, such as improving patient recruitment and speeding up transitions between dosing cohorts.

DEEP Measures, an organization that helps pharmaceutical and biotech companies develop digital health measures, supports the FDA's pilot program for AI-enabled optimization of early-phase clinical trials. They argue that the pilot must prioritize the definition and validation of "fit-for-purpose" measures before applying AI to ensure that speed does not amplify incorrect scientific questions or flawed endpoints.

The U.S. Chamber of Commerce supports the FDA's proposed pilot program to use AI in early-phase clinical trials, praising its voluntary and structured approach. They recommend aligning the pilot with principles of efficiency, neutrality, proportionality, collegiality, and flexibility while ensuring robust protection of intellectual property and human accountability.

Unlearn.AI, a technology company specializing in AI-based digital twins for clinical trials, supports the FDA's pilot program to optimize early-phase clinical trials. They propose specific use cases, including digital twin efficacy benchmarks and real-time trial simulations, to improve decision-making, shorten development timelines, and enhance data quality.

Melinda B. Chu, CEO and Founder of Cythera Bio, supports the FDA's pilot program to integrate AI into early-phase clinical trials. She argues that the program should initially focus on therapeutically simpler platforms, such as topical or intranasal products, to establish transferable methodologies before moving to more complex modalities like cell and gene therapies.