What Is an Observational Study?

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Topics include: Treatment and Understanding

In addition to standard clinical trials, observational studies also play a key role in cancer research. But what’s the difference between a clinical trial and an observational study? Dr. Brady Stein explains, sharing what kind of data is collected from an observational study and how it is used to further cancer research and treatment. Dr. Stein specifically discusses the REVEAL study (www.revealpvstudy.com) for those living with polycythemia vera (PV) and notes the critical importance of these types of studies in moving research forward. 

This town meeting was sponsored by Incyte Corporation. It was produced by Patient Power in partnership with the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

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Transcript

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Jeff Folloder:

So, Dr. Stein, there’s more than one kind of trial going on. We were just talking about clinical trials. There are other trials, observational studies. What are those?

Dr. Stein:         

So, an observational study, what Dr. Pemmaraju is talking about are interventional trials where a medicine is given to a patient.

It’s something active. It’s an intervention. So observational trials are observational. And investigator is acting like a fly on the wall and watching a process. Nothing has actively been given to a patient. We’re observing a course, a process, a symptom, a sign or something of that nature. So we learn a lot about demographics, epidemiology, practice patterns, impacts on quality of life. So we can learn a tremendous amount from these observational studies. So an observational study is a look going forward. So we call that perspective. We’re starting at one point. We have questions we’re asking.

And we’re going to get these questions later on. It’s going to take us a couple of years. We’re going to follow prospectively. We’re going to follow forward. That’s a difference between many of the studies. A great majority of the studies that are done in our field are retrospective.

We’re taking a look back. We’re asking a question, but we’re looking at a series of patients that have been followed from years back. And those are important studies. It’s very, very hard to do prospective studies in this field, because it’s a relatively rare disease. Thankfully, some of the things that we’re focusing on, the event rate is low, blood clotting and transitions in the disease, and it may take many, many years to observe it. So most of the studies we know are retrospective. We’re looking back. And some of those studies have bias. There are things about those studies that can affect the quality.

So there’s one study right now that has enrolled over 2,000 patients. It’s following patients with polycythemia vera. It’s called the REVEAL Study. And it’s a study that what’s very, very unique is that the patients are coming from what we call a real-world setting. So about 80 percent of the patients are probably coming from a community practice.

And 20 percent are coming from an academic practice. That’s probably more like real life. Not all patients are seen solely at a university. Many of the publications or the papers that we know about that have been landmark or have made historical breakthroughs, a lot of those are coming from single centers where patients only get their care at a place that may only see patients with polycythemia vera. So this is a little bit different. It’s a real-world snapshot. Most of the patients are coming from the community. It’s all over the United States. And only a minority are going to be seen in an academic center.

And we’re going to look at those patients going forward, and we’re going to know about what they contemporary demographic is in PV in 2017. The epidemiology, the burden of symptoms, how that affects quality of life, finances. What changes over time in terms of symptoms? What may change in time in terms of complications, sequences of therapy. So that information is coming. And the studies enroll over 2,000 patients.

The same types of studies are being conducted in myelofibrosis, a subset of patients with myelofibrosis and a subset of patients with essential thrombocythemia. So it helps us make contemporary observations about your diseases. And that will help us inform future treatments. It helps us identify limitations in what we’re doing. So these studies are really critically important.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

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Page last updated on November 3, 2017