IN 2015, WHEN THE ​U.S. Food and Drug Administration (FDA) approved Ibrance (palbociclib) as a treatment for hormone-sensitive, HER2-negative metastatic breast cancer, it relied on data from clinical trials that had enrolled only postmenopausal women. Even so, oncologists began using the drug to treat men with this type of breast cancer.

No clinical trial systematically tracked how effective Ibrance was in men. But clues to how well the drug was working could be found in the men’s electronic medical records, where their doctors reported results of exams and scans, and recorded tumor progression. This information is what researchers refer to as “real-world data.” When Ibrance received expanded approval from the FDA for use in men in April, it was this type of data, rather than a new clinical trial, that provided the evidence on safety and effectiveness for the FDA to review.

Gregory Daniel, a health policy specialist, is the deputy director of Duke University’s Washington, D.C.-based Duke-Margolis Center for Health Policy. He spoke with Cancer Today about how real-world data and evidence can be used to expand drug approvals and what those approvals could mean for cancer patients.

Q: What is meant by real-world data and evidence?
A: Basically, real-world data is any data that is generated as patients encounter the health care system. When you get a prescription filled, when you go to the doctor’s office, when you are in the hospital—these generate insurance claims. Beyond that, there are the clinical documents that are in electronic medical records or in patient registries. All of these become real-world data sources. Taking that data and doing research with that data generates real-world evidence.

Q: Is this a new idea, or is it that there is now better access to this type of data?
A: A little bit of both. The FDA has used real-world evidence in the past. They use it when treating very rare diseases where it would be unethical or unfeasible to randomize patients [into different treatment groups]. It’s also used to compare trial participants to external controls, where the data comes from real-world data sources. But it was the 21st Century Cures Act [a bill Congress passed in 2016 that provides funding to develop ways to get treatments to patients faster] that focused attention on using the data to add new drug indications. There is a huge opportunity for real-world evidence to bring valuable information to inform regulatory decisions. 

Q: Why is this helpful?
A: Often when a product is already on the market, it’s hard to randomize patients because patients have access to the new product. So studies just don’t get done, but we see off-label use. The ability to use real-world data as evidence should enable companies to perform more studies more cost effectively.

Q: Can patients trust that these​ data are​ as good as the data collected in clinical trials?
A: That’s the big question. The FDA and our center are both helping to develop new and better ways to measure data quality. FDA reviewers are good at measuring clinical trial data purposefully collected. We don’t have a set of standards or common consensus on how to measure data that are coming from claims, electronic medical records, wearable devices or cellphone apps, and we don’t yet know how to assess the quality of that data. There is a whole field of data science that is looking at how to turn data like this into valuable evidence that accounts for biases that might affect causality.

Q: Why is the Ibrance approval important?
A: If you happen to be a man with breast cancer and are talking to your doctor about a drug, you are likely to learn there is no information on how this product works in men. With this new labeling and new regulatory approval, men know evidence has been brought to regulators who have found that Ibrance is effective. That is huge. This is really the biggest opportunity for real-world data and evidence to have an impact because it provides relevant information to patients and physicians when making decisions. 

Q: What is the most important thing for cancer patients to know about real-world evidence?
A: That it can enable us to access more information on patients not in cancer clinical trials—like patients with chronic conditions—and create better opportunities for that evidence to provide more relevant information for patients. We are unlikely to see real-world data lead to brand-new approvals. This is more about collecting data on drugs that are already on the market and have established risk-benefit profiles and using that data to generate real-world evidence to identify other uses.