On any given day, laboratories and innovation hubs across the United States are bustling with activity as researchers push the boundaries of science and medicine. For decades, U.S. health-care innovators, such as Regeneron Pharmaceuticals, benefited from an ecosystem that allowed for unparalleled collaboration leading to global breakthroughs and worked securely [PDF] in the knowledge that it was one of the world's leading translational ecosystems, as demonstrated by the rapid development of a coronavirus vaccine during the COVID-19 pandemic.
Despite these achievements, regulatory strictures threaten the country's competitive edge in life sciences innovation. Although the U.S. Food and Drug Administration (FDA) has long been seen as the gold standard for drug and medical device regulation, its stringent protocols and lengthy approval processes—considered huge advantages in the service of patient safety—are becoming blunt tools in a world that requires speed and adaptability. Meanwhile, China's life sciences sector benefits from the tailwinds of streamlined regulations, proactive government policies and strategic focus, and an ecosystem designed for rapid innovation.
Without reforms, the United States is at risk of losing its position as the global leader in life sciences
As geopolitical competition spreads into the arena of health-care innovation, the implications of specific regulatory choices take on greater importance. Against that backdrop, the U.S. life sciences sector is at a critical juncture. Although the FDA's stringent standards are crucial for safeguarding public health, they should be harmonized with policies that foster responsible risk-taking and efficacy. Marty Makary, the Donald Trump administration's nominee for FDA commissioner, has made some comments that support this view, but any such changes are still on the horizon. China's coordinated and adaptive regulatory framework that has helped its life sciences sector grow rapidly could provide valuable lessons for U.S. regulatory agencies.
The U.S. Regulatory Framework: A Double-Edged Sword
The FDA's regulatory processes are a mixed blessing. On one hand, they help ensure public safety by following high standards for approving new drugs and medical devices. On the other, those safeguards come with opportunity costs in an era when medical innovation is accelerating.
The FDA's approval pathways can take years to navigate, slowing the commercialization of treatments, and recent cuts threaten to make the process even longer. Those delays not only affect patients but also reduce U.S. competitiveness in a fast-moving global market. By contrast, China has introduced measures such as conditional approvals and accelerated pathways for innovative drugs, enabling its companies to launch products more quickly and the capture market share in areas of high demand such as oncology.
Although the FDA has introduced fast-track processes, including the Breakthrough Therapy Designation as well as the Priority Review Pathways, studies show that China's policies, such as looser restrictions on foreign trial data, could be faster and have no reported effects on safety. These policies reflect an approach that balances safety-driven oversight with the need for greater flexibility in a rapidly evolving market, creating an environment in which new therapies reach patients more quickly.
The cost of compliance with FDA standards is another critical hurdle, particularly for smaller firms that have traditionally driven the bulk of biotech innovation. Clinical trials, documentation, and regulatory fees create financial barriers that require onerous fundraising, which then prevents startups from pursuing innovative ideas or delays their ability to execute plans. In China, compliance reforms have reduced entry barriers for many firms, encouraging engagement from both domestic and foreign companies.
China's Rapid Ascent in Life Sciences
China's life sciences sector is no longer in its infancy. Over the past decade, consistent with national priorities, it has been repositioned as a strategic emerging industry, receiving the highest level of support across the government.
Without reforms, the United States is at risk of losing its position as the global leader in life sciences. Such a loss would be particularly challenging in an era of rising geopolitical tensions that have already started to threaten pharmaceutical supply chains with ripple effects on drug access and prices.
One of the consequences of the FDA's slow and cautious approach to regulating emerging technologies is the opportunity cost of the United States losing out on the development of new technologies. Although the United States remains a leader in basic research—aimed at expanding understanding a subject without an immediate goal—its slow transition to applied innovation could allow competitors such as China to dominate in new areas including synthetic biology and artificial intelligence–driven drug discovery.
Reimagining U.S. Competitiveness Through Regulatory Reform
For the United States to remain a global leader in life sciences, it needs to streamline its regulatory framework to keep pace with an evolving world. A few specific steps would enable it to do so.
1. Embrace Adaptive and Seamless Trial Designs
Using adaptive, seamless trial designs such as integrated phase 2 and phase 3 designs and innovative dose-finding approaches, as proposed in recent oncology studies, could speed up the evaluation of dose, efficacy, and safety. This would reduce the need for separate, sequential studies and accelerate decision-making.
2. Expand the Use of Real-World Evidence and Validated Surrogate Endpoints
By expanding acceptance of robust real-world evidence and validated surrogate endpoints—particularly for life‐threatening or rare diseases—the FDA could provide earlier drug approvals. Real-world evidence has traditionally been used on a limited basis as a support for effectiveness. Expansion for the types of data that can be accepted for approval decisions is a key element in driving speedier access to these therapies.
This strategy would mimic some of China's conditional approval policies, which use surrogate markers to predict clinical benefit and allowing for the dispensing of the therapy while requiring confirmatory post-approval studies.
3. Modernize Administrative and Data Submission Processes
Transitioning from paper-based, strict submission protocols to simpler electronic submissions with standardized data formats and real-time tracking could reduce lengthy administrative delays.
A rolling review process—in which data is evaluated on a real-time or near real-time basis rather than waiting for a complete set—could further accelerate the process, an approach used successfully in the European Union during COVID-19
4. Leverage User Fees to Boost Resources Without Compromising Rigor
Funding increases for regulatory agencies have historically helped drive increased speed in approvals. The Prescription Drug User Fee Act has helped the FDA increase staffing and materially reduce review times. By focusing additional energy on these sorts of fee programs to ensure that the agency has adequate and modern resources, reviews could be further streamlined while balancing the need for independence and scientific rigor.
5. Enhance Postmarket Surveillance and Risk Management
Streamlined premarket processes could be complemented by strong postmarket surveillance. This approach, used more aggressively in China, has borne fruit in speeding access to therapies. Conditional approvals paired with stringent postmarket risk management plans (similar to the Accelerated Approval Program) allow earlier patient access while balancing the need for long-term safety and efficacy.
6. Incorporate Patient-Centric and Real-Time Data Analytics
Real-time monitoring of clinical trials promises to speed up the evaluation of therapies. By expanding the use of real-time data and patient-centric endpoints, regulators could quickly assess drug performance, allowing for rapid modifications in ongoing trial design as well as accelerated decision-making.
Reforms to accelerate approval processes could reduce the time-to-market for new therapies. Although the FDA has created fast-track approaches toward rare diseases, more is needed, and quickly, with just-released guidance, China is increasing support for R&D and industry expansion, as well as an adaptable regulatory system, all changes that could potentially extend its lead in evolving frameworks that are even more fit-for-purpose to guide the development of the next-generation of biotechnologies.
