Mart<\/b>in<\/b> Makary<\/b><\/strong><\/p>\n<\/div>\n \u201cFor the last 50 years, the medical profession has sadly grown to accept a 10- to 12-year timeframe for new drugs to come to market,\u201d Makary said. \u201c[This] is a milestone day for us to challenge that deeply held assumption.\u201d<\/p>\n
Approximately 45% of the drug development process \u2014 the period between initiation of a phase 1 trial and submission of a regulatory application \u2014 is \u201cdead time,\u201d Makary said. During this period, no active research takes place. Instead, investigators and staff complete paperwork and perform other \u201ctedious\u201d tasks that are not always necessary, he added.<\/p>\n
The FDA collaborated with Paradigm Health to implement a model designed to accelerate trial execution and regulatory review. The technology company\u2019s AI-powered platform will automate data collection and analysis, allowing FDA reviewers to access EHR data in the cloud to monitor data related to predetermined efficacy or safety endpoints in real time.<\/p>\n
\u201cWhen a patient in a clinical trial develops a fever or is hospitalized, or when their tumor shrinks and a radiologist reads the new tumor size, FDA regulators can see in real time exactly what is happening,\u201d Makary said. <\/p>\n
Makary called the initiative \u201ca critical step\u201d in the agency\u2019s broader goal to facilitate continuous trials and move away from the traditional clinical development model that consists of multiple distinct phases \u2014 each with its own protocol, a separate study and lag time in between.<\/p>\n
Two proof-of-concept real-time trials \u2014 both of which are evaluating oncology therapies \u2014 got underway this month.<\/p>\n
The randomized phase 2 TRAVERSE study is evaluating acalabrutinib (Calquence, AstraZeneca) in combination with venetoclax (Venclexta; AbbVie, Genentech) and rituximab for treatment-naive mantle cell lymphoma. MD Anderson and Penn are among the trial sites.<\/p>\n
The phase 1B STREAM-SCLC trial will evaluate tarlatamab (Imdelltra, Amgen) for treatment of limited-stage small cell lung cancer.<\/p>\n
Paul Burton, MD, PhD, FACC, MRCS, FRCP,<\/b> Amgen\u2019s senior vice president and chief medical officer, thanked FDA officials for their \u201copenness to new approaches that can advance patient care while upholding the high standards of safety and scientific integrity.\u201d<\/p>\n
\u201cInnovation should not only deliver breakthrough medicines but also rethink how we deliver them,\u201d Burton said during the press briefing. \u201cThat means supporting approaches that bring research closer to patients where they are and help reduce the barriers to participation.<\/p>\n
\u201cThis is where the promise of more integrated data-driven approaches comes into focus,\u201d Burton added. \u201cBy leveraging data from electronic health records, we have an opportunity to help democratize clinical research. We can reach broader patient populations and generate insights in ways that are more reflective of everyday clinical practice, and this is a meaningful step toward greater heath equity and innovation.\u201d<\/p>\n
The agency intends to launch a broader pilot program this summer. As part of that effort, FDA released a request for information seeking public input on that program\u2019s design and implementation, including metrics and criteria to measure success. Comments will be accepted until May 29.<\/p>\n
\u2018Thoughtful\u2019 approach<\/h2>\n FDA officials have discussed the potential value of real-time clinical trials \u201cfor years\u201d and committed last spring to developing an implementation strategy, according to Jeremy Walsh, <\/b>the agency\u2019s chief AI officer.<\/p>\n
Initially, the FDA will continue to collect trial data through the traditional regulatory process in addition to via real-time monitoring. At the conclusion of the pilot process, agency officials will assess what worked well and how the concept can be improved.<\/p>\n
\u201cThe primary objectives of this agency are safety and efficacy, and neither of those things will change,\u201d Walsh said during the press briefing. \u201cThe goal is to get to a regulatory decision in a faster timeline without compromising safety. We have been getting tons of data for decades. We get information overload sometimes and we don\u2019t need everything that we receive. This is kind of a transformative thing where the FDA is trying to reimagine what information we need and when we need it in order to make a decision.\u201d<\/p>\n
Litton praised the dual approach for data collection.<\/p>\n
\u201cIt is really thoughtful to do it in this way and not just flip the switch,\u201d she said. \u201cIt won\u2019t be perfect. If we expect perfection out of the gate, I can promise you we will be disappointed, but perfection should not stop progress. We\u2019ll compare this to what we\u2019re doing currently, we\u2019ll learn, we\u2019ll iterate and we\u2019ll grow.\u201d<\/p>\n
Real-time clinical trials should streamline the collection of vast amounts of trial data that currently are entered \u2014 often manually \u2014 into multiple systems \u201cthat were never designed to talk to each other,\u201d Litton said.<\/p>\n
That consumes valuable time and increases costs, she said.<\/p>\n
\u201cThe way we collect data makes it very hard for researchers and for patients,\u201d Litton said. \u201cWe are collecting so many points of data that never get looked at. If we are faxing a bunch of different forms to eight different places, it might take much longer to see a signal.<\/p>\n
\u201cIf we know what data we really need to evaluate and prospectively collect it \u2014 knowing we can go back to the electronic health record to get anything else we need \u2014 we can see a signal of efficacy as early as possible,\u201d she added. \u201cWe also may get an earlier signal that something isn\u2019t working and we need to move on, or we may pick up early signals of toxicity that can help us understand how to better manage our patients.\u201d<\/p>\n
The real-time clinical trials initiative comes at an \u201cabsolutely pivotal stage\u201d for the drug development industry, Meagher said, noting more clinical trials than ever are being conducted outside the United States.<\/p>\n
Data transfer automation could have an invaluable downstream effect, Meagher said.<\/p>\n
\u201cWe need to keep our workforce trained and engaged, and we need to consider the barriers they face,\u201d Meagher said. \u201cThis particular innovation … will help our research coordinators, our nurses and our project managers, who are so hard to find … and virtually impossible to keep. Easing this process at the academic interface of patient care will have an absolutely revolutionary [effect] on our ability to sustain this workforce and to keep this type of work occurring within the U.S.\u201d<\/p>\n
Meagher also suggested the real-time clinical trials initiative \u2014 coupled with a Trump administration that she noted is \u201csquarely focused on speed\u201d and a desire to streamline processes and eliminate barriers \u2014 could pave the way for other innovative strategies.<\/p>\n
\u201cThe fact that we could start with something so practical could open the door to more versatile utilization of AI in the process,\u201d Meagher said. \u201cIf the FDA shows an appetite for this type of innovation, maybe they will be more willing to work with sponsors using real-world data and simulated data sets for drug approval. This could open the door for application of data science to multiple different aspects of how we evaluate novel therapies, which ideally \u2014 in aggregate \u2014 could result in speeding up the process.\u201d<\/p>\n
For more information:<\/h2>\n Jennifer Keating Litton, MD, MHCM, <\/b>can be reached at jlitton@mdanderson.org.<\/p>\n
Emma A. Meagher, MD,<\/b> can be reached at emma@upenn.edu.<\/p>\n\n
\n
Published by:<\/span>\n
Sources\/Disclosures<\/h3>\n Source: <\/h2>\n Healio Interviews<\/p>\n
Reference:<\/h2>\n\n
Disclosures: <\/strong>
<\/p>\n
\n
\n
<\/p>\n
Ask a clinical question<\/strong> and tap into Healio AI’s knowledge<\/strong> base.<\/p>\n
<\/p>\nPubMed, enrolling\/recruiting trials, guidelines<\/li>\n
<\/p>\n
Clinical Guidance, Healio CME, FDA news<\/li>\n
<\/p>\n
Healio’s exclusive daily news coverage of clinical data<\/li>\n
\n <\/ul>\n
Learn more<\/button>\n <\/div>\n<\/div>\n
![\"hemonc](\"https:\/\/www.healio.com\/~\/media\/h5\/feature\/news\/publogos\/hot.svg?la=en&h=24&w=141&hash=2F86D471C8514C0E334E329AA799E8B4\")
\n <\/div>\n<\/p><\/div>\n<\/p><\/div>\n