COMMENTARY

Don't Discount 'Real-World Evidence'

Chadi Nabhan, MD, MBA

Disclosures

December 13, 2019

This transcript has been edited for clarity.

I am Chadi Nabhan, a hematologist and medical oncologist, and the chief medical officer of Aptitude Health. Today I want to talk about real-world evidence, because it gets a bad rap in the lay press and on social media.

You may be aware that fewer than 5% of adult patients with cancer in the United States are enrolled in clinical trials[1]; 90% of them are white,[2] and fewer than one quarter are over the age of 65, even though close to two thirds of patients with cancer in the United States are over the age of 65.[1,2] So it goes without saying that patients seen in clinical trials do not always represent the patients whom oncologists see day in and day out, outside of the clinical trial setting.

The data from patients who are seen outside of clinical trials—how these patients are treated and their outcomes—are essentially what we refer to as "real-world evidence." Without question, this type of information is important because many times, it includes toxicities that were not seen in clinical trials. The data also tell us how patient populations that are underrepresented in clinical trials are being cared for. They may also allow us to look at the total cost of care. Given the fact that many patients in clinical trials may have some of their care covered by the sponsor or the study, we may not know the true costs of care outside the clinical trial setting. In addition, real-world evidence gives us the ability to investigate the effectiveness of treatments on a larger scale because it represents more patients being treated for a given disease. So there are definitely benefits to understanding how these patients are being cared for.

The types of real-world data we usually analyze may be derived from social media data, pharmacy claims or other types of claims, electronic medical records, lab data, biomarker data, registries, and observational studies. All of these data sources, in aggregate, represent real-world data. And the way we analyze them and generate the evidence represents real-world evidence.

I'm not saying that real-world evidence is great all the time, because obviously it has its limitations. Clearly, there is no standardized approach to assessing response with real-world data, whatever disease we may be talking about. We have no standardized way to dictate when the physician should treat, or switch therapy, and so on. There is no standardized way to assess progression, again regardless of the disease.

But hear me out. Despite these limitations and the imperfections of the real world, it remains the world in which we see patients. It represents 95% of adult patients with cancer who are being cared for. So if we are going to take the data generated from the 5% who are included in clinical trials and apply them to the 95% of patients who are being seen outside of clinical trials, I think we also need to pay attention to what we call the real-world experience.

Many of the criticisms of real-world data are valid, but I would also say that there are a lot of factors driving real-world data right now. The 2016 21st Century Cures Act  supports real-world evidence and real-world data and how they can be used for regulatory approvals. Payers are using real-world evidence to determine coverages and premiums. Manufacturers are using these data to make strategic decisions. Physicians are using real-world data in some cases to decide how to treat patients and build algorithms, predictive models, prognostic models, and so forth. So it's fair to say that real-world evidence is here to stay.

How can we reconcile the limitations of real-world evidence with its benefits to make sure we help patients? We need to keep an open mind. It probably doesn't matter where the evidence comes from, as long as that evidence will help one patient. The goal is to help our patients. Wherever the data come from, the onus is on us to analyze and understand those data so we can actually help our patients moving forward.

In my opinion, real-world evidence is complementary to clinical trial evidence; it is not to be disregarded or thrown away. It is not to be criticized day and night as an awful thing to consider. We need to use it as a complement to randomized clinical trial data, because ultimately our goal is to help our patients.

Dr Chadi Nabhan practices hematology and medical oncology in Chicago, Illinois, and is executive vice president and chief medical officer of Aptitude Health.

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