Getting Real

January 29, 2019


In the world of drug development, when things are working as they should, data drives decisions.

The double-blind, placebo-controlled trial, often referred to as the “gold standard” in the world of the U.S. Food and Drug Administration, reflects the interest in making decision about the safety and efficacy of drugs based on data that is collected in a clear and consistent way without bias.

But the proliferation of information technology, as well as new sources of data—such as electronic health records, low-cost sensors, and smart devices—are creating the opportunity to capture a wealth of data that can provide new insight into patients and the safety and efficacy of drugs and devices.

It is reassuring to hear that the U.S. Food and Drug Administration is moving toward expanding and formalizing the use of these sources of real-world data in its decision-making. Speaking at the Bipartisan Policy Center, FDA Commissioner Scott Gottlieb called advancing real-world data to regulatory quality real world evidence “a key strategic priority for the FDA.”

“This isn’t an indictment of the randomized controlled trial. Far from it,” he said. “It’s a recognition that new approaches and new technologies can help expand the sources of evidence that we can use to make more reliable treatment decisions. And it’s a recognition that this evidence base can continue to build and improve throughout the therapeutic life of an FDA approved drug or medical device.”

Real-world data and real-world evidence are being used today for postmarket safety monitoring. As an example, Gottlieb pointed to data derived from the agency’s Sentinel system that eliminated the need for postmarketing studies on nine potential safety issues involving five products. That, he said, made the agency post-market evaluation of safety “timelier and more effective.”

Perhaps a more compelling potential is how real-world evidence can be used to improve the clinical trials process and reduce cost, increase speed, and allow patients more easily to participate. This can be particularly beneficial in trials for rare diseases where patients enrolled in a study may be geographically dispersed and far from a clinical trial site. It can also help on the challenge of enrollment by using patient data from natural history studies as controls, as has been done.

In December, the FDA released its 2019 Strategic Framework that outlines how the agency plans to use new digital tools and opportunities to advance its review of product effectiveness and post-market safety.

Among other things, the framework calls on the agency to develop a path for ensuring that real-world solutions can play a more integral role in drug development and regulatory life cycle at the FDA.

During his speech Gottlieb announced four additional efforts intended to help the FDA and stakeholders advance opportunities for the use of real-world data. He announced plans to convene a stakeholder meeting in 2019 to help develop a framework on the integration of digital technologies in clinical trials. The agency also plans to use digital technologies to bring clinical trials to the patient rather than always requiring patients to travel to the investigator.

He said the agency is exploring how reviewers can have more insight into how labeling changes inform provider prescribing decisions and patient outcomes by using real-world data to examine the impact of a recent FDA labeling change for two approved immune checkpoint inhibitors from weight-based dosing to flat dosing. Last, the agency will use new software-based machine learning algorithms – like natural language processing or deep learning – to help develop regulatory science tools like surrogate endpoints or digital biomarkers that can be used to guide more efficient development programs.

The richness of real-world data is also its challenge. Validating data sources, ensuring interoperability, and making sure data measurements are consistent are among the issues that will need to be addressed.

But the FDA’s interest in expanding and formalizing the use of real-world data is good news for rare disease patients, who are likely to be among the earliest beneficiaries of this effort.


January 29, 2019
Photo: FDA Commissioner Scott Gottlieb



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