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FDA’s Real-World Evidence Framework Is Out. Now What?

Dec. 10, 2018, 11:30 AM

Drug companies could rewrite their playbooks for demonstrating how well their medicines work under a new FDA blueprint on how it will accept analyses of routine health data.

The Food and Drug Administration released its framework Dec. 6 on how it plans to incorporate real-world evidence—data pulled from electronic health records, claims data, and even data collected from mobile apps like Apple Watches and Fitbits—into its regulation of drug effectiveness.

The framework signals a major step forward for the FDA as it moves away from relying exclusively on data collected from expensive, time-consuming randomized controlled trials. Using real-world data—any health data that don’t come from a randomized controlled trial—could save drug developers millions of dollars and speed the FDA’s ability to make drug approval decisions.

“This is an opportunity for drug companies to be much more nimble, but that requires them changing their infrastructure and evaluating how they can use this, and not just considering it a fun innovation that they might try someday,” said Nancy Dreyer, senior vice president of real world evidence for IQVIA, a technology, analytics and clinical research organization provider based in Durham, N.C.

The highly-anticipated document aims to help repair what the agency’s drug chief Janet Woodcock has called a “broken” clinical trials system. Randomized controlled trials historically have been considered the gold standard in demonstrating a drug meets the agency’s standards for patient use.

But the agency wants to turn to other data sources to fill in gaps that a controlled environment can’t answer, such as how a drug impacts a patient’s daily life.

Top FDA Priority

FDA Commissioner Scott Gottlieb called the incorporation of real-world evidence a top priority for the agency. “We’re committed to realizing the full potential of these tools in advancing the development of novel therapeutic products and strengthening our regulatory oversight of medical products across the life-cycle continuum,” he said in a Dec. 6 statement.

Gregory W. Daniel, who leads the Duke-Margolis Center for Health Policy’s pharmaceutical and medical device policy portfolio, told Bloomberg Law the framework means drug companies are likely to pull other teams into their regulatory submissions. Historically, those submissions have mostly relied on randomized controlled trials. Drug companies now likely will pull in more of their real-world evidence and outcome’s research groups. Those other units seek to understand the end results of particular health-care practices and interventions.

“The organizations have the expertise,” he said in a Dec. 7 interview. “It’s just bringing the people who are experts in real-world evidence into the process for generating regulatory-grade evidence.” There might be a lot of cross-functional working groups within companies to bridge the two camps.

The Pharmaceutical Research and Manufacturers of America, the brand-name drug industry trade group, applauded the release of the framework. PhRMA spokesman Andrew Powaleny told Bloomberg Law the framework provides insight and understanding into how FDA views the use of real-world data to support regulatory decision-making.

The FDA defines real-world data as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources” including electronic health records, claims and billing data. Real-world evidence is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data.

Real-world evidence “has great promise to speed up the availability of information about the safety and effectiveness of a medicine,” Powaleny said in a Dec. 7 email.

Both Daniel and Dreyer said the framework is just the beginning of a major effort to use real-world evidence to demonstrate effectiveness.

New Guidances Coming

The FDA said its real-world evidence program will focus on how these data can be used to support changes to labeling about drug product effectiveness. Those labels discuss things like changes in a drug dose or how to administer the medicine. The agency also said it plans to issue at least half a dozen new guidances to answer lingering questions about the real-world evidence program.

“This is the opening salvo,” Dreyer told Bloomberg Law.

Those forthcoming guidances will identify potential gaps in real-world data sources and strategies to address them. They’ll also examine how to determine whether real-world data collected from electronic health records, medical claims, and health registries are reliable enough to generate evidence. One of the major appeals of a randomizing patients into different study arms of a clinical trial is that it eliminates bias of the data being generated.

The FDA also plans to finalize draft guidance issued last year on the Use of Electronic Records and Electronic Signatures in Clinical Investigations.

Daniel said he’s pleased to see the FDA focus on observational study designs in the framework. Observational studies measure associations between a substance and a disease but don’t have the controlled setting of intervention studies

The framework culminates two years of work from groups like the National Academies and the Duke-Margolis Center for Health Policy, which the FDA charged to study real-world evidence. It also fulfills a mandate under 21st Century Cures (Pub. L. 114-255)—the 2016 law to spur new drugs and devices—to produce the framework by Dec. 13, 2018.

To contact the reporter on this story: Jeannie Baumann in Washington at jbaumann@bloomberglaw.com