WTAS: FDA Announces Major Steps to Implement Real-Time Clinical Trials

Agency unveils real-time trial proofs-of-concept and upcoming pilot program

The U.S. Food and Drug Administration today announced two major steps as part of an initiative to advance the implementation of real-time clinical trials (RTCT). First, the agency unveiled the successful initiation of two proof-of-concept clinical trials that will report endpoints and data signals to the agency in real time. Second, the agency released a Request for Information (RFI) regarding a proposed pilot program for RTCT that will launch this summer. 

Early-phase clinical trials are a bottleneck in drug development, often characterized by high uncertainty, limited patient populations, and inefficient decision-making processes. Data is typically reported from sites to sponsors, who analyze and subsequently submit data to the FDA. With improvements in AI and data science, sponsors and trial sites have the opportunity to conduct real-time trials in a way that enhances safety monitoring and radically increases efficiency. 

Headlines
Medical Professionals
Advocates & Organizations
Influencers & Members of the Media

Headlines:

• The Wall Street Journal: FDA Turns to AI to Speed Up Clinical Trials
• Bloomberg: FDA Plans to Speed Up Drug Trials With Real-Time Data, AI
• Reuters: US FDA to monitor clinical trial data in real time in pilot program aimed at speeding approvals
• ABC News: FDA set to pilot test real-time tracking of clinical trials
• Fierce Biotech: FDA unveils plan for real-time review of clinical trial data, with AstraZeneca and Amgen already on board
• Axios: FDA to use AI to track clinical trials in real time
• STAT: FDA launches effort to speed up clinical trials, using AI
• US News & World Report: US FDA to Monitor Clinical Trial Data in Real Time in Pilot Program Aimed at Speeding Approvals
• MedPage Today: FDA Says First ‘Real-Time’ Clinical Trial is Underway
• BioSpace: Amgen, AstraZeneca join FDA effort harnessing cloud for real-time clinical trials
• Government Executive: FDA to pilot real-time clinical drug trials through cloud and AI
• Clinical Trials Arena: FDA launches pilot for real-time clinical trials
• Becker’s Hospital Review: FDA launches ‘real-time’ clinical trials
• BioPharma Dive: FDA tests out ‘real-time’ clinical trials; AbbVie closes in on a KRAS biotech
• American Hospital Association: FDA announces real-time clinical trials, request for information

Medical Professionals:

• Penn Medicine Senior Vice Dean for Clinical and Translational Research, Dr. Emma Meagher, via The Wall Street Journal: “In general, we have a sense that the way we do clinical trials is dysfunctional in many ways… The whole purpose here is to accelerate cures…”
• Johns Hopkins University Bioethicist, Stephanie Morain, Ph.D., via Fierce Biotech: “I will readily accede that there are plenty of ways where, administratively, we could be moving faster…I remain enthusiastic for seeing efforts that are being made in good faith to speed the trial timelines.”
• Bentley University in Massachusetts Director of the Center for Integration of Science and Industry, Fred Ledley, M.D., via Fierce Biotech: “…a step forward…”
• Calderon Medicine Concierge Mental Health CEO, Alston B. Calderon, PA-C, via X: “This is how we move the needle forward in medicine.”
• University of Michigan Professor of Surgery, Andrew M. Ibrahim M.D., MSc, via X: “In many domains, real time data feedback loops would be so helpful. Love this innovation!”
• Williams Cancer Institute Longevity Director and oncologist, Jason R. Williams, M.D., DABR, via X: “The lag between innovation and regulation has been one of the biggest barriers to getting effective treatments to cancer patients, and real-time data feeds close part of that gap. The next step is figuring out how to enable approvals for combinations of agents, not just single drugs. Most cancer cures are going to come from stacking 4, 8, sometimes more agents in the right sequence, and the current one-drug-at-a-time framework can't accommodate that. Glad to see the FDA moving in this direction.”
• Wilfrid Laurier University Assistant Professor, Department of Health Sciences, Nirosha J. Murugan, via X: “This is exactly what a lean, responsive scientific community needs... the ability to pivot when confronted with new information. For decades, it would take several years to move from asking good questions, to securing funding, performing the experiments, and publishing the results. Think of the inertia. Think of the wasted time and resources. Now, novel insights (with rigorous structure) can shift directions.”
• Derya Unutmaz, M.D., via X: “This is a very big deal! Incredible to see this acceleration in clinical trials happening thanks to the amazing leadership at @US_FDA! Accelerating clinical trials via AI is a key milestone toward my bold claim that we can treat/cure most, or even all, diseases within a decade!”
• Venk Murthy, M.D., Ph.D., via X: “Great to see this type of investment in modernization which will lead to faster, more efficient trials without compromising scientific merit or confidence!”

Advocates & Organizations:

• Paradigm Health CEO, Kent Thoelke, via STAT: “What the FDA has done here, which is really revolutionary, is instead of asking for data, they’re asking for us to identify when signals happen to a patient in the electronic health record…Then we send that signal to the pharma sponsor and the agency.”
• Paradigm Health CEO, Kent Thoelke, via press release: “This collaboration represents an important step toward expediting how clinical trial data is submitted to and reviewed by the FDA…With Paradigm Health's platform, clinical trial data can be analyzed for key signals in near real time and shared with trial sponsors and the FDA in days, rather than months. This means that regulators, sponsors, and providers can have a continuous view of safety and efficacy. By modernizing the flow of information from sites to sponsors to regulators, we have an opportunity to accelerate the delivery of innovative therapies to patients across the country.”
• Paradigm Health Co-Founder and Board Chair, Robert Nelsen, via press release: “The initiative leverages the best of U.S. technology and innovation to bolster clinical development in the United States… This model can align the interests of policymakers, pharmaceutical and biotech companies, and American patients, and make U.S. clinical research faster and more cost-effective.”
• Amgen Senior Vice President and Chief Medical Officer, Dr. Paul Burton, via The Wall Street Journal: “This program has the potential to be transformational on so many levels in how we do clinical research…”
• MD Anderson Chief Clinical Research Officer, Jennifer Litton, via STAT: “Right now, we collect vast amounts of complex data, often manually, and then we put them in multiple different systems that were never made to talk to each …When we do that, we take the time away from our patients and our researchers…If you need the hope for a clinical trial, you want it right then…”
• Bloomberg Intelligence Director of Research, Global Industries, Sam Fazeli, via X: “This is very promising, allowing drugs to get to patients faster and costing pharma companies a little less.”
• Evercore ISI analysts quoted via Fierce Biotech: “This is a logical next step from the FDA rather than a major step-change, and is an example of regulators doing what they should do by responding to a fast-moving technology and beginning to define a workable pathway for its use…”
• Jefferies analysts quoted via BioSpace: “[A]nother step towards reducing structural inefficiencies and accelerating timelines…Receiving data flow in real-time enables the FDA to make decisions faster—all while preserving the foundational requirements of safety, monitoring, governance, and data integrity.”
• Alnylam Founding CEO, John Maraganore, via X: “This is a terrific initiative @DrMakaryFDA! It’s a positive step forward to potentially bring innovation to patients faster.”
• Noetik CEO and Co-Founder, Ron Alfa, via X: “Big move toward AI-enabled clinical trials. A new era starting where technology impacts the most important step for developing new medicines.”
• Rain Consulting Group via X: “This is real progress. Thank you @DrMakaryFDA.”
• theMultiplicity CEO, Andrew Critch Ph.D., via X: “Holy sh*t this is important if you care about health and longevity. The body is homeostatic, so any intervention can backfire. So as we complexify our intervention suite for extending healthspan, real-time RCT data streaming will be CRUCIAL to supporting each other as fellow humans. My family's data stream can help yours, and yours can help mine, all anonymously. I thought this was at least another year of bureaucratic wrangling away, but it's here. Major respect to Dr. Makary and all involved!”
• Gradient Venture Capital Partner, Andrew Brackin, via X: “[M]assive opportunity in real-time trials…”
• Biocon Limited Executive Chair, Kiran Mazumdar-Shaw, via X: “The @US_FDA pilot initiative marks a significant step forward in accelerating drug development and expediting approvals. By receiving only aggregated signals, such as adverse event rates and tumor response percentages, the regulator can make informed, data-driven decisions efficiently, without the need to directly handle underlying clinical datasets.”
• Triatomic Capital Co-Founder, Jeff Huber, via X: “Huge progress from the @US_FDA, to enable innovation with speed and quality with 'real-time clinical trials.' Many thanks to @JonathanHirsch & the Paradigm Health Team for helping show the way!”
• InVitria Founder and CEO, Scott E. Deeter, via X: “This is smart!”
• Tevogen Bio Founder and CEO, Ryan Saadi, via X: “Brilliant! Faster development of life-saving therapies, Improved patient affordability & U.S. biotech competitiveness. Congratulations @DrMakaryFDA @US_FDA”
• Khosla Ventures Partner, Alexander A. Morgan, M.D., Ph.D., via X: “Awesome shift in clinical study operations by @DrMakaryFDA and @US_FDA. Kudos on pushing this forward.”
• Moffitt Cancer Center Director of Business Operational Transformation, Bill Arneson, via X: “Great to see continued innovation for clinical trials!”
• Fenn Wolo, independent AI and biotech researcher via X: “FDA is really transforming. I’m excited to see what other milestones they have.”
• Matt Ripkey, healthcare technologist and investor via X: “This is fantastic progress! Clinical trials is a market that, implemented effectively, will dramatically improve public perception of AI. It shouldn't take 8-12 years to bring treatments to market. We can do better identification, better enrollment, better monitoring, and even better simulation of outcomes now than we could just two years ago.”
• Errik B. Anderson, biotech entrepreneur via X: “Another example of the great work under @DrMakaryFDA and the current @US_FDA. We all really appreciate the dedication to good science, good medicine and better velocity - while keeping patients at the heart of the story.”
• Allison Krug, MPH, epidemiologist via X: “@DrMakaryFDA crushing it! This is great news…”

Influencers & Members of the Media:

• Bullish (@bullishbruk) via X: “Dr. Marty Makary’s real-time clinical trial initiative is a historic leap for medicine…This move proves that regulatory processes can finally be as dynamic and innovative as the science they oversee and we have to give credit where credit is due!”
• Jeff Tang (@jefftangx) via X: “Clinical trials are the biggest bottleneck to drug development. FDA is cooking insanely hard.”
• Dr. Steven Quay (@quay_dr), scientist and author via X: “A remarkable step forward by @US_FDAand @DrMakaryFDA today. I know how drugs are developed, having invented and developed seven drugs that have received FDA approval. Dr. Makary is leading the FDA with innovation and creativity into a future that will benefit all Americans.”
• The Limiting Factor (@LimitingThe) via X: “Finally! Moving into 21st century medicine.”
• Annette Bosworth, M.D. (@AnnetteBosworth), author and physician via X: “Well played.  Great step forward.”
• Omar M. Khateeb (@OmarMKhateeb), Host of State of Medtech Podcast, via X: “Really exciting news for healthcare innovators.”
• Mansi (@mansizzzzle) via X: “America is taking a big step in the right direction…”