How the FDA Is Transforming Clinical Trials

The U.S. Food and Drug Administration’s push to modernize the clinical trial industry should be well received by clinical trial and pharmaceutical stakeholders. Encouraging innovative, modern study design facilitates both a patient-centric focus and cost savings for all.

FDA Commissioner Scott Gottlieb, M.D., recently called for rethinking study designs, using continuous trials and master protocols, creating a ripple effect of approval in this staid industry and all who are affected by it.

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His embrace of the latest technologies is well met. Digitizing clinical studies enables the “site-less trial.” It empowers modern design — transforming research for more impactful data and effective medical breakthroughs while democratizing clinical studies and reining in the cost of medications to benefit all.

Time and Cost of Clinical Trials

Reduced costs and accelerated timelines made possible by digital clinical studies can help our industry to identify new treatments and get them to market faster. Recruitment and induction are often faster in a digital clinical study. Since there is no need for a physical site, recruitment isn’t geo-fenced, making global recruitment possible. Moreover, the use of electronic informed consent eases the induction burden on subjects. Electronic informed consent saves time, which saves money.

Digital clinical studies also address the cost burden to governments, payers, employers and patients. The site-less model can cost half of the price of a traditional clinical study. Hybrid trials — trials with traditional brick-and-mortar site visits and digital components — can cut a quarter of the normal study cost by reducing time to completion. The savings to pharmaceutical companies can be in the hundreds of millions of dollars and years of collective saved time — with a cost-savings effect that benefits governments, payers, employers and patients alike.

Real-World Data for Clinical Trials

Efficacy versus effectiveness is a prominent issue in our industry. A drug might prove to be successful among a compliant group of subjects, but will it be effective in the real world, where adherence matters most? One of the advantages to a digital clinical study is that the data are collected in the real-world, as subjects go about their daily lives. This means the real-world evidence obtained in digital clinical studies are more likely to reveal how the drug will work once it’s on the market, thereby affecting market access via real-world data.

Streaming Media Meets Streaming Trials

Digitizing clinical studies enables Gottlieb’s vision of streamlining clinical studies via surrogate endpoints and compression of the clinical study phases.

Surrogate endpoints are readily obtained within the domain of a digital clinical study via real-time, real-world data capture. This facilitates the FDA’s vision for adaptive design by being able to course correct mid-stream within a clinical study for such indices as dosage or population size, rather than waiting for a study to fail. The effects: both time and money saved. Add in the advent of “bring your own device,” which digital clinical studies enable, and the real-world evidence data pool becomes deeper and more insightful in a shorter period via rules-based engines, machine learning and artificial intelligence (AI). That fosters the visionary excellence Gottlieb is espousing.

Compression of the standard three-phase trial into one long and continuous study (Gottlieb’s vision) is empowered by digital and, more specifically, machine learning and AI. Machine learning is ideally suited to longitudinal data insights, speeding the arrival of endpoint goals with mathematical precision while creating new insights into a drug’s use. It also identifies dangerous anomalies earlier than was ever possible before. The tech ultimately results in billions of dollars potentially saved from speed to market, increased patient safety and averted legal issues.

When you add post-marketing surveillance and the ability to monitor a patient’s lifetime use of the drug, then you’ve created the utopia being sought by big pharma, governments, insurers, employer groups and patients — all made possible by the modernized FDA and Gottlieb.

Bryan Silverman is the CEO of ObvioHealth.

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