Why does it take a new drug 10 years, on average, to come to market? Part of the reason lies in the dead time in the process.
Historically, trials have required tedious tabulations and repeated application submissions between phases, which is why 45% of the time from a Phase 1 trial until final submission is spent without any ongoing clinical trial in progress — idle time in the system.
On Tuesday the Food and Drug Administration announced a bold new approach that could fundamentally transform the development of new treatments: real-time clinical trials.
For the first time, FDA regulators will be able to see what’s happening in a clinical trial, looking at endpoints in the cloud as they occur. This is an important step toward our broader goal of moving toward continuous trials, reducing dead time in the system.
This approach uses modern technology to challenge the deeply held assumption that new cures require a long wait. We believe powerful treatments can reach patients faster without cutting any corners on safety.
During my career as a surgical oncologist at Johns Hopkins, I monitored my patients in the operating room and the intensive care unit using continuous, real-time data. If a patient’s vitals dropped, we intervened immediately. We didn’t wait for a retrospective report to be mailed to us weeks later. Why should the regulatory oversight of powerful experimental drugs be any different?
In my career, I’ve had to inform patients of a new cancer diagnosis more than a thousand times. It never got easier. Every time, I wondered: “Why can’t we, as a modern society, figure out a way to make powerful treatments available sooner to those who are willing to try them?” Those experiences had a profound impact on my thinking and have shaped our reforms.
By viewing safety signals and clinical endpoints in real time, FDA scientists can detect adverse reactions sooner, potentially enhancing patient safety. Conversely, when a new therapy is showing strong efficacy, we can identify that success rapidly. Real-time trials are not just technically possible; they are transformative for the entire clinical ecosystem.
On Tuesday we unveiled the successful initiation of a proof-of-concept clinical trial, conducted by AstraZeneca with participation from the University of Texas MD Anderson Cancer Center and the University of Pennsylvania, that will report endpoints and data signals to the FDA in real time. Working closely with the sponsor, the FDA has established the technical framework — powered by improvements in artificial intelligence and modern data science — to securely stream and validate key safety and efficacy signals as these trials progress. Another is being set up with Amgen.
We are not stopping with these two proof-of-concept trials. To build on this success, we will be launching a broader real-time clinical trials pilot program this summer that will evaluate expanded cloud-based systems, define standardized metrics for success, and bring more sponsors into this new, dynamic regulatory paradigm.
By coupling these design principles with real-time data streaming, we can bring unbroken drug development to all disease areas. Whether we are fighting rare genetic disorders, autoimmune diseases, or widespread chronic conditions, continuous trials will ensure that data informs decisions instantly. We can learn from every single patient in real time, minimize exposure to ineffective doses, and drastically accelerate the delivery of cures.
This initiative represents one part of our FDA common sense modernization campaign. We have already replaced onerous animal testing requirements with computational modeling and other newer methodologies, reduced burdensome requirements for biosimilars, and launched a new National Priority Voucher pilot program to get review decisions out in one to two months instead of the typical 10 months to a year. Six drugs have already been approved, including zongertinib, a lung cancer drug that can bring patients out of hospice, and Otarmeni, which can restore hearing to some children with genetic hearing loss. Patients who can potentially benefit from these game-changing new therapies deserve an FDA that is modern and efficient.
These reforms also hold promise to lower drug prices for everyday Americans. That’s because these reforms lower R&D costs — savings that can be passed on to patients.
The future of medicine should not be kept waiting. At the FDA, we are ensuring it arrives in real time.
Marty Makary, M.D., M.P.H., is commissioner of the Food and Drug Administration.
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