Enabling Clinical Trial Innovation Through AI Regulation at FDA

Innovation is not just a luxury but a necessity in the realm of clinical trials, particularly as we strive to accelerate drug development. Yet, the slow pace of innovation within the U.S. clinical trials system has impeded progress, leaving many stakeholders frustrated. U.S. Food and Drug Administration (FDA) resource limitations cause the inadvertent prioritization of programs sponsored by pharmaceutical companies, resulting in a bottleneck that stifles the adoption of more efficient and patient-friendly methods. Thus, expected breakthroughs from pharma have not materialized, leaving patients and researchers at a standstill. 

Pharmaceutical companies have traditionally been at the forefront of clinical trial programs, and the FDA’s focus on these entities suggests an expectation that they will lead the way in innovation. However, this reliance on pharma has not yielded the anticipated advancements due to factors such as high costs and risks associated with drug development, regulatory complexities, and the inherent conservatism of large organizations.

Current regulatory frameworks provide resources for interactions with drug developers through the Prescription Drug User Fee Act (PDUFA). The PDUFA enables drug manufacturers to partially fund the drug review process, ensuring well-resourced and timely reviews of their drug development programs. Since its introduction in the 1990s, the PDUFA has significantly accelerated drug review timelines for sponsors. In contrast, programs without a user fee component have not received adequate resources for evaluation. 

What is needed is a paradigm shift — establishing pathways for the review and approval of innovative methodologies by the FDA. These pathways should facilitate the adoption of new techniques and approaches, including those enabled by AI, that can streamline clinical trials, reduce costs, and, most importantly, bring effective treatments to patients sooner.

National conversation on AI could be leveraged to empower FDA

Integrating AI into various sectors poses a dual challenge for the United States: fostering innovation to maintain its global technology leadership while ensuring responsible and safe technology use. While the government’s approach to AI regulation is understandably cautious given the complexity of the technology and its potential impact, there is a need for policies that facilitate innovation without compromising safety. 

The rapid growth of AI and the complexities involved have prompted a call for a more centralized approach to AI oversight; for example, establishing a single governmental entity to oversee AI. Proponents of this idea believe it would provide clarity and consistency in regulation. While this proposal may seem logical, it raises concerns about overregulation, which could potentially stifle innovation and hinder technological progress without necessarily enhancing safety. Thus, many legislators have agreed that the U.S. should adopt a risk-based approach to AI regulation within existing sector-specific regulatory agencies, such as the FDA.

The FDA has acknowledged the broad potential of AI in healthcare, from research to drug development and beyond. However, its capacity to assess these technologies has been hampered by a lack of resources for timely evaluation. Very few applications are accepted into current methodological review programs, and applicants may wait years for a response. Consequently, the prioritization of drug manufacturer-driven innovation through PDUFA has limited the adoption of discoveries from industrial and academic organizations that could greatly accelerate drug development. As such, there has been minimal progress in clinical trial innovation over the past 30 years. 

Given the current national focus on AI regulation, it is an opportune time to empower the FDA with the resources and authority needed to regulate AI in healthcare effectively, and introducing user fee-based pathways for methods review is essential. For a deliberate regulatory approach to succeed, federal agencies must be equipped with the necessary resources to fully grasp AI’s complexities.

A new path forward for innovation

Broadening user fee programs to encompass companies specializing in innovative technologies could provide the FDA with the necessary resources for timely and equitable technology assessment. By implementing user fees for reviewing new drug development technologies, the FDA could enhance its capacity to support innovation, thoroughly evaluate AI applications in healthcare, and safeguard public health.

One approach to implementing this expansion is by establishing a tiered fee structure based on the risk level and potential impact of the technology under review. Companies developing methods with greater risk potential could be subject to a higher fee, reflecting the increased resources required for thorough evaluation. This tiered approach ensures that the FDA has the necessary resources to evaluate high-impact technologies while minimizing the burden on companies developing safer applications, and is reflective of the diverse risk profile within both AI applications and other types of methodologies.

Additionally, the FDA could offer incentives for companies participating in user fee programs. For instance, participating companies could receive expedited reviews for other products, additional support from FDA experts, or additional education through the Small Business and Industry Assistance (SBIA) program. These incentives would encourage companies to invest in developing innovative technologies and contribute to healthcare advancement.

Currently valued at $20.9 billion in 2024 and projected to grow to a staggering $148.4 billion by 2029, AI is reshaping the future of healthcare at an unprecedented pace. As the FDA stakes its claim as the primary regulator of AI in healthcare, it must navigate the delicate balance between fostering innovation and ensuring safety and responsibility. Modifying existing regulatory frameworks, such as the PDUFA, or adding new ones that account for innovative technologies like AI, could equip the FDA with the necessary resources to effectively evaluate these advancements.

Photo: Getty Images, Sarah Silbiger

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