Innovation is not just a luxury, but a necessity in clinical trials, especially as we strive to accelerate drug development. Yet the slow pace of innovation in the U.S. clinical trials system has hampered progress, frustrating many stakeholders. Resource constraints at the U.S. Food and Drug Administration (FDA) lead to unintended prioritization of drug company-sponsored programs, creating a bottleneck that hinders the adoption of more efficient and patient-friendly approaches. As a result, anticipated advances from the pharmaceutical industry have not materialized, leaving patients and researchers in limbo.
Pharmaceutical companies have always been at the forefront of clinical trial programs, and the FDA’s focus on these entities suggests that they will be at the forefront of innovation. However, this reliance on pharmaceutical companies has not yielded the desired results due to factors such as the 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). PDUFA allows drug manufacturers to partially fund the drug review process, ensuring timely and well-funded reviews of their drug development programs. Since its introduction in the 1990s, PDUFA has significantly accelerated drug review timelines for sponsors. In contrast, no-user-fee programs have not received adequate resources for review.
A paradigm shift is needed: establishing procedures for FDA review and approval of innovative methodologies. These procedures should facilitate the adoption of new techniques and approaches, including those enabled by AI, that can simplify clinical trials, reduce costs, and, most importantly, bring effective treatments to patients more quickly.
National AI Debate Could Be Used to Empower FDA
The integration of AI across various sectors poses a dual challenge for the United States: fostering innovation to maintain its global technology leadership while ensuring responsible and safe use of the technology. While the government’s approach to regulating AI is understandably cautious given the complexity of the technology and its potential impact, there is a need to put in place policies that facilitate innovation without compromising safety.
The rapid growth of AI and the complexity of its applications have led to calls for a more centralized approach to AI oversight, such as creating a single government entity to oversee AI. Proponents of the idea argue that it would bring clarity and consistency to regulation. While this proposal may seem logical, it raises concerns about overregulation, which could potentially stifle innovation and hinder technological progress without necessarily improving safety. As such, many lawmakers have agreed that the United States should adopt a risk-based approach to AI regulation within existing sector-specific regulatory agencies, such as the FDA.
The FDA has recognized the vast potential of AI in healthcare, from research to drug development and beyond. However, its ability to evaluate these technologies has been hampered by a lack of resources for rapid assessment. Very few applications are accepted into current methodological review programs, and applicants can wait years for a response. As a result, the focus on drugmaker-led innovation through PDUFA has limited the adoption of discoveries from industry and academic organizations that could significantly accelerate drug development. As a result, progress on innovation in clinical trials has been minimal over the past 30 years.
Given the current national focus on AI regulation, it is timely to equip the FDA with the resources and authority to effectively regulate AI in healthcare. Introducing user fee mechanisms for method review is essential. For a deliberate regulatory approach to succeed, federal agencies must be equipped with the resources to fully grasp the complexities of AI.
A new path to innovation
Expanding user fee programs to companies that specialize in innovative technologies could provide the FDA with the resources needed to evaluate technologies quickly and fairly. By implementing user fees for the review of new drug development technologies, the FDA could improve its ability to support innovation, thoroughly evaluate AI applications in health care, and protect public health.
One approach to implementing this expansion is to establish a tiered fee structure based on the risk level and potential impact of the technology being reviewed. Companies that develop methods with higher risk potential could be subject to higher fees, reflecting the increased resources required for a thorough evaluation. This tiered approach ensures that FDA has the resources needed to evaluate high-impact technologies while minimizing the burden on companies that develop safer applications, and reflects the diverse risk profile of AI applications and other types of methodologies.
In addition, the FDA may offer incentives to companies that participate in user fee programs. For example, participating companies could receive expedited reviews for additional products, additional support from FDA experts, or additional training through the Small Business and Industry Assistance (SBIA) program. These incentives would encourage companies to invest in the development of innovative technologies and contribute to the advancement of health care.
Currently rated at $20.9 billion in 2024 With an estimated budget of $148.4 billion by 2029, AI is transforming the future of healthcare at an unprecedented pace. As the FDA stakes its claim as the primary regulator of AI in healthcare, it must strike a delicate balance between fostering innovation and ensuring safety and accountability. Modifying existing regulatory frameworks, such as the PDUFA, or adding new regulatory frameworks that account for innovative technologies like AI could equip the FDA with the resources needed to effectively assess these advances.
Photo: Getty Images, Sarah Silbiger
Dr. Jess Ross, Ph.D.holds the position of Senior Government Affairs Officer at Unlearn.AI, a pioneering startup revolutionizing medicine with artificial intelligence. In this role, Dr. Ross champions regulatory acceptance of AI-driven clinical trials through academic publications in the biostatistics and medical communities, as well as policy advocacy. She received her PhD in neuroscience from the University of Cincinnati/Cincinnati Children’s Hospital, where she was awarded the 2017 Presidential Medal for Graduate Student Excellence for her advocacy, community contributions, and prolific scientific achievements. After a postdoctoral fellowship in the Department of Anesthesia at Stanford Medicine, Dr. Ross transitioned to medical writing for innovative startups. Her broad expertise spans AI policy, biomedical science, biostatistics, and biotherapeutics, positioning her as an emerging talent in the responsible integration of AI in medicine.
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