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Clinical trials are valuable sources of information about how well drugs work, how patients react to treatments and what combined factors yield the best health outcomes.

However, evidence from clinical trials is only directly applicable to a subset of research questions. Additionally, clinical trial results may not translate into real-world results due to tightly controlled patient selection, protocol-directed patient treatment and monitoring, and other aspects of the clinical trial. 

To complement the limited resources of information derived from clinical trials, patient and health care data is collected from sources other than clinical trials.

This data is known as real-world data (RWD), and it is increasingly being recognized as a legitimate tool for addressing research questions and informing evidence-based health care measures.

On Dec. 6, 2018, the Food and Drug Administration (FDA) announced a forward-looking decision to accept and proactively advance the use of real-world data drawn from the day-to-day experience of patients and doctors to help inform and improve regulatory decisions.1

The question now is how do we work together to identify the most fruitful approach to collecting and using such data?


Real-world data vs. real-world evidence

Scientists distinguish between real-world data and real-world evidence. According to John Seeger, chief scientific officer at Optum, “real-world data is the byproduct of people passing through routine care; it is comprised of all the data collected and stored in different formats.” Real-world evidence, on the other hand, “is the inferences drawn from the data, so it requires the application of hypotheses, analyses and subsequent conclusions.”

Real-world data typically originates from tools like wearables, electronic health records, lab tests, patient-reported outcomes, product and disease registries, medical claims and clinical assessments, said Kristen Buck, MD, senior vice president and chief of clinical development at Optum. Recently, a supplemental FDA-backed MyStudies app was launched to curate real-time information from our mobile devices.2

“When we link these examples of data together, we discover a patient’s health story,” Buck said. “Taking this one step further, we use it to create new insights at the patient level, the group level, or the population level with regards to the benefits and risks of a drug or a device. From there, we’re able to create an actionable insight, such as a label change, a treatment plan, a best practice or even an FDA guidance or guideline.”

Real-world evidence is the context in which the evidence is gathered, according to a New England Journal of Medicine article. The article cites clinical care and home or community settings as opposed to research-intensive or academic environments.3


Cautionary steps to expand data use and outcomes

The FDA guidance will serve as a catalyst to expand the use of real-world data and evidence, improving what we know about medical care. Yet, we need to proceed carefully.

“There has been rhetoric on utilizing real-world data for clinical development, but the industry was lacking guidance from the FDA on requirements and best practices for the collection, analysis and submission of these data,” explained Lauren Neighbours, senior director of regulatory affairs at Optum. “The framework released by the FDA provides some insight on the Agency’s current thinking and approach to real-world data, but there are several unanswered questions.” Neighbours said these include understanding the logistics for integrating and analyzing real-world data to:

  • Maximize quality
  • Minimize bias
  • Ensure it meets the scientific rigor expected from the FDA

The patient’s voice matters

The FDA has consistently advised sponsors to represent the patient’s experience throughout their clinical development programs, offering direction through programs such as the Patient-Focused Drug Development initiative.

“This focus is equally important in real-world studies,” says Martha Bayliss, vice president of patient insights at Optum. “Patients are hungry for evidence that will inform their clinical care decisions, with real-world outcomes measured in terms that mean the most to patients: how they feel and what they are able to do. Sponsors should approach their real-world evidence programs with a sound measurement strategy that includes patient-reported measures.”4

We're trying to create the finite, tangible output needed to shift mindsets. It's a work-in-progress on both ends, but it is progress.

– Kristen Buck, MD

Navigating our way forward

Organizations that use data to sharpen insights have stepped up to support this new medical care matrix.

“Optum is working with trial sponsors on using real-world data to create real-world evidence,” Buck said. “And we will be approaching the FDA for their guidance. We're trying to create the finite, tangible output needed to shift mindsets. It's a work-in-progress on both ends, but it is progress.”

It’s too early to fully understand and appreciate the breadth of the FDA milestone. To proceed, an innovative yet cautious approach will be required.

“The FDA decision is really important, but it’s only one step,” explained Bayliss. “It’s not prescriptive, but it helps spark the conversation. Optum is well-equipped to help sponsors understand how to operate in a new world, but really we are learning how to navigate it together.”

Patient care remains a priority. “The experience of the patient that comes through patient-reported outcomes and other real-world data illustrates a democratization of health care data,” Seeger said. “That data may more directly represent the patient's voice than clinical trial evidence.”



  1. U.S. Food & Drug Administration. Statement from FDA Commissioner Scott Gottlieb, M.D. Published Dec. 6, 2018. Accessed March 22, 2019.
  2. U.S. Food & Drug Administration. FDA launches new digital tool to help capture real world data from patients to help inform regulatory decision-making. Published Nov. 6, 2018. Accessed March 22, 2019.
  3. New England Journal of Medicine. Real-World Evidence – What Is It and What Can It Tell Us? Published Dec. 8, 2016. Accessed March 22, 2019.
  4. U.S. Food & Drug Administration. Enhancing Benefit-Risk Assessment in Regulatory Decision-Making. Updated March 20, 2018. Accessed April 22, 2019.
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