The Department of Health and Human Services (HHS) recently admitted its processes are “bogged down by bureaucracy and busy work,” and pledged to speed up artificial intelligence (AI) innovation, including drug and biologic approvals at the Food and Drug Administration (FDA).
Regulatory Hurdles Slow Medical Progress
Table of Contents
A shift to a zero-based regulatory approach could unlock AI’s potential to revolutionize drug development.
- History suggests regulatory intervention, rather than scientific complexity, is the biggest obstacle to rapid technological advancement.
- The Human Genome Project demonstrated that scientific progress isn’t inherently slowed by complexity, but can be by subsequent regulation.
- Cases involving stem cells and direct-to-consumer genetics illustrate how regulatory overreach can stifle innovation and alter research directions.
- A “zero-based” regulatory approach—starting fresh with each new technology—could streamline AI adoption in healthcare.
For too long, medical innovation has lagged behind other sectors. As Dr. Scott Podolsky of Harvard Medical School observed, “Medicine in 2020 is much closer to medicine in 1970 than medicine in 1970 was to medicine in 1920.” Breakthroughs like antibiotics, antihypertensives, antidepressants, antipsychotics, and steroids haven’t had the same transformative impact as innovations in the latter half of the 20th century.
Experts point to two primary reasons for this slowdown: the inherent complexity of biological processes and the regulatory approval process. But is it truly the science holding us back, or the red tape?
The Human Genome Project: A Tale of Two Speeds
The Human Genome Project (HGP), completed in 2003, was a monumental scientific achievement, mapping the human genetic code and identifying disease-related genes. Remarkably, the project finished two years ahead of schedule, proving that complex scientific endeavors aren’t automatically delayed by their own intricacy.
However, the pace dramatically slowed once the HGP’s “roadmap” was handed off for drug discovery and development. It took nine years to demonstrate the potential of CRISPR-Cas9 gene editing technology, and another eleven years for the first gene-editing treatment to gain FDA approval. In 2025, the FDA acknowledged its processes weren’t always clear to stakeholders and announced greater regulatory flexibility for cell and gene therapies, but the bottleneck remains.
Currently, CRISPR technology has countless applications, from basic research to clinical trials for diseases like cancer, and over 1,000 CRISPR screens for drug discovery. Yet, the FDA lacks the resources to efficiently process all this activity, inevitably leading to further delays. The delays appear to stem from a lack of FDA resources to administer existing regulations, rather than the scientific complexity itself.
Stem Cells and Genetic Testing: Cautionary Tales
The story of Geron Corporation illustrates the financial constraints imposed by regulation. Beginning stem cell research in the late 1990s, Geron raised $100 million, but spent a significant portion on extensive pre-clinical research—over 2,000 experiments on mice and rats—at the FDA’s direction. Their Investigational New Drug application totaled 22,000 pages and cost $45 million. While a Phase 1 clinical trial showed no safety issues, Geron terminated the program in 2011 due to financial limitations. The company eventually received FDA approval in 2024 for a telomerase inhibitor after 34 years of research.
Similarly, 23andme, a pioneer in direct-to-consumer genetic testing founded in 2006, faced regulatory hurdles. In 2013, the FDA deemed the provision of personal genetic information a “device” requiring approval, potentially leading to unnecessary medical interventions. The FDA estimated over one million tests would need approval, an impossible task for both the agency and the company. While 23andme received some approvals starting in 2015, the regulatory burden ultimately contributed to the company declaring bankruptcy in March 2025.
These cases demonstrate how years of scientific research can stretch into decades of regulatory review. The financial markets are closely watching how the FDA regulates AI in healthcare, anticipating potential delays.
The FDA and AI: Will the Pattern Repeat?
The FDA currently requires all drugs to meet a “reasonable assurance of safety and effectiveness,” as outlined in a 1962 statute. AI-discovered drugs must still undergo the traditional Phase I, II, and III clinical trials. In January 2025, the FDA introduced a 7-step framework for AI-enabled submissions, requiring detailed documentation on algorithms, training data, and potential biases—requirements not typically applied to traditional drug development.
Furthermore, a December 2025 HHS Request for Information revealed concerns among practitioners about liability when using AI technology that the FDA might later deem requiring prior approval. It remains unclear how HHS will navigate this evolving landscape, but a cautious approach could stifle innovation.
A Path Forward: Precision Regulation
A zero-based regulatory approach would end “regulatory creep” and focus on essential safety and effectiveness requirements. Regulators wouldn’t participate in pre-clinical research, and documentation would be limited to specific validation needs. The government’s role would shift to auditing and validating trial outputs, including experimental validation, mechanistic understanding, and ethical oversight.
This streamlined approach would free up FDA resources, allowing more trials to be conducted with adequate oversight. With experience and the application of AI, requirements could be refined through annual reviews. This isn’t deregulation; it’s precision regulation. AI could automate auditing, providing real-time data feeds and exception reporting.
In an efficient market, leaders would leverage AI to accelerate innovation and deliver value. The biggest threat to this outcome in healthcare isn’t the complexity of diseases, but the weight of regulation. HHS leaders should initiate a zero-based regulatory scheme for AI, imposing requirements only where necessary to ensure safety and effectiveness. This wouldn’t require new legislation or rules.
The FDA’s role should evolve from gatekeeper to auditor, observing and assessing scientific progress. This approach would foster innovation and get the healthcare industry back on track. AI is still in its early stages in healthcare, and regulations should reflect that reality. The current FDA framework risks delaying AI innovation and needs adjustment before critical timelines are missed.
Steven Zecola is a former technology executive and government official. He retired 24 years ago with a diagnosis of Parkinson’s disease and is currently an ardent patient advocate.
