In the realm of clinical trials, innovation is a necessity, not just a luxury, especially as we strive to accelerate drug development. Yet the slow pace of innovation in the U.S. clinical trials system is impeding progress and frustrating many stakeholders. Resource limitations at the U.S. Food and Drug Administration (FDA) have inadvertently prioritized pharmaceutical company-sponsored programs, creating bottlenecks that prevent the adoption of more efficient, patient-friendly methods. As a result, expected breakthroughs from pharmaceutical companies have not materialized, leaving patients and researchers in limbo.
Pharmaceutical companies have traditionally been at the forefront of clinical trial programs, and the FDA's focus on these organizations signals an expectation that they will lead innovation. However, due to factors such as the high costs and risks associated with drug development, regulatory complexity, and the conservatism inherent in large organizations, reliance on pharmaceutical companies has not produced the advances expected.
The current regulatory framework provides 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 adequate resources and timely review for drug development programs. Since its introduction in the 1990s, PDUFA has significantly accelerated sponsors' drug review timelines. In contrast, programs without a user fee component do not receive adequate resources for evaluation.
What is needed is a paradigm shift: establishing pathways for FDA review and approval of innovative methodologies. These pathways should drive the adoption of new technologies and approaches, including those enabled by AI, that can streamline clinical trials, reduce costs, and most importantly, get effective treatments to patients faster.
National discussion about AI could be used to strengthen FDA
The integration of AI into various sectors poses a dual challenge for the United States: fostering innovation to maintain global technological leadership while ensuring responsible and safe use of the technology. While the government's approach to regulating AI is understandably cautious given the technology's complexity and its potential impacts, we need policies that foster innovation without compromising safety.
The rapid growth of AI and its attendant complexity have led to calls for a more centralized approach to AI oversight, such as establishing a single government agency to oversee AI. Proponents of the idea believe this would bring regulatory clarity and consistency. While the proposal may seem logical, it raises concerns about overregulation, which could stifle innovation and impede technological progress without necessarily increasing safety. As such, many lawmakers agree 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 recognizes the broad potential of AI in healthcare, from research to drug development. However, its ability to evaluate these technologies is hampered by a lack of resources for timely evaluation. Very few applications are accepted into the current methodological review program, and applicants may wait years for a response. As a result, PDUFA's prioritization of drug manufacturer-driven innovation has limited the adoption of discoveries from industry and academic institutions that could significantly accelerate drug development. As such, progress in clinical trial innovation over the past 30 years has been minimal.
Given the current focus on AI regulation across the country, now is a golden opportunity to give the FDA the resources and authority it needs to effectively regulate AI in healthcare, and implementing a user-fee pathway for methodology review is essential. A careful regulatory approach can only be successful if federal agencies have the resources they need to fully grasp the complexities of AI.
New Paths to Innovation
Expanding the user fee program to include companies focused on innovative technologies will ensure FDA has the resources it needs to conduct timely and impartial technology evaluations. Introducing user fees to review drug development technologies will strengthen FDA's ability to support innovation, thoroughly evaluate AI applications in healthcare, and protect public health.
One way to implement this expansion would be to establish a tiered fee structure based on the risk level and potential impact of the technology being reviewed. Companies developing methods that pose a higher probability of risk could be assessed higher fees to reflect the increased resources required for a thorough evaluation. This tiered approach would ensure that FDA has the resources it needs to evaluate higher-impact technologies while minimizing burden on companies developing safer applications. It also reflects the diverse risk profiles of both AI applications and other types of methodologies.
In addition, FDA may offer incentives to companies that participate in user fee programs. For example, participating companies may receive expedited review of other products, additional support from FDA experts, and additional education through the Small Business Industry Assistance (SBIA) program. These incentives encourage companies to invest in the development of innovative technologies and contribute to medical advances.
Valued at $20.9 billion in 2024 and projected to grow to a staggering $148.4 billion by 2029, AI is transforming the future of healthcare at an unprecedented pace. As the FDA positions itself as the lead 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 PDUFA, or adding new ones that take into account transformative technologies like AI, can equip the FDA with the resources it needs to effectively evaluate these advancements.
Photo: Sarah Silbiger, Getty Images
Dr. Jess Ross serves as Senior Director of Government Relations at Unlearn.AI, a pioneering startup revolutionizing healthcare through artificial intelligence. In this role, Dr. Ross advances regulatory approval of AI-driven clinical trials through academic publications and policy advocacy in the biostatistics and medical communities. Dr. Ross received her PhD in Neuroscience from the University of Cincinnati/Cincinnati Children's Hospital, where she received the 2017 Presidential Graduate Student Excellence Award for her advocacy, community contributions, and prolific scientific achievements. After a postdoctoral fellowship in the Department of Anesthesiology at Stanford University School of Medicine, Dr. Ross transitioned into medical writing for innovative startups. Her broad expertise spans AI policy, biomedical sciences, biostatistics, and biotherapeutics, positioning her as an emerging talent in the responsible integration of AI in healthcare.
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