Reality Check: Dr. Christoph Koenen on Key Things to Know About Real-World Data

August 25, 2017


r. Christoph Koenen, Cardiovascular Marketed Products Development Lead at Bristol-Myers Squibb, recently shared his thoughts on the importance of real-world data analyses in helping inform clinical decision making on patient care. Real-world data is derived from a variety of different sources, including de-identified electronic health records (EHR), insurance claims databases and patient registries, to shed light on practice decisions made by physicians in the “real world” versus in a controlled, clinical trial setting. Dr. Koenen shared how these data can help inform clinical development and explained how Bristol-Myers Squibb is working to understand trends in clinical practice, uncover insights and apply learnings from real-world data to develop and deliver innovative medicines.

Q. Dr. Koenen, we are hearing so much these days about real-world data. What is it, and why does it matter?

Dr. Christoph Koenen, Cardiovascular Marketed Products Development Lead at Bristol-Myers Squibb

Dr. Christoph Koenen, Cardiovascular Marketed Products Development Lead at Bristol-Myers Squibb

The double-blind, placebo-controlled clinical study has been and continues to be the gold standard in pharmaceutical research. While that remains fundamental to evaluating the safety and efficacy of any drug, real-world data analyses can give us insight to what happens when the medicine is used in the “real world” – where conditions and the variables in each patient’s life may come into play. No matter how thorough our clinical studies are, there is always more to learn once a medicine goes into that real-world use. As Dr. Tom Frieden, the former director of the U.S. Centers for Disease Control and Prevention, recently wrote for the New England Journal of Medicine, randomized controlled trials can establish a drug’s efficacy, while data from real-world observations are useful to refine dosages and identify side effects, especially rare ones.

In the past, individual clinicians would use their own real-world experience to inform their decisions. Now, electronic health records in hospitals and clinical offices make it possible for health care providers and their patients to contribute de-identified data seamlessly and for researchers to access aggregated data easily. That, plus the development of technology that can process enormous amounts of data, lets us study the real-world experience of large numbers of people. We can potentially spot emerging trends in clinical practice or pinpoint the characteristics of patients that may optimize outcomes. 

One of the most exciting benefits of real-world data is that it will help us design better, more efficient clinical trials. This is important because study design and recruitment of patients into clinical trials are among the major variables that affect how long it takes for a new drug to proceed from the laboratory to regulatory review and ultimately, to patients. By analyzing data from millions of people, we can better target the most suitable patients to enroll in clinical trials. The faster we can recruit eligible patients into clinical trials, the faster we bring new medicines to patients in need.

Real world data

Q. What new information can these kinds of studies provide to physicians and patients?

The power of real-world data comes from its scale. Because we can potentially look at information from the experience of large numbers of people, we can zoom-in to subgroups that would be difficult or impossible to derive from randomized clinical trials. Now, we can aggregate patients with similar characteristics across geographies. The emerging wealth of data lets us study associations with our medicines in patients in routine clinical setting conditions, and share that information with physicians.

Real-world data can also provide insight into important questions about the relative costs, benefits and risks of different medicines, surgical procedures, and other healthcare interventions. This knowledge enables health care providers to take into account many factors, such as clinical outcomes and cost, which can help patients have more informed discussions with their healthcare professionals about their choices in care.

Q. How is Bristol-Myers Squibb advancing the use of real-world data?

Bristol-Myers Squibb has invested heavily in our ability to leverage real-world data analyses to supplement data from randomized controlled trials to better understand how different types of health interventions work. In the cardiovascular medicine area alone, we’ve analyzed hundreds of thousands of non-identifiable patient records and published our findings to supplement clinical trial data results to inform clinical, health system and payer decisions.

It is important to note that there are several limitations to real-world data analyses, including that this type of data is subject to potential patient-selection biases, and that the source and type of data used may also limit the ability to generalize the results and endpoints to the overall population. Therefore, real-world data cannot be used as stand-alone evidence for treatment evaluation.

Access to these large data sets collected in the real-world lets us analyze how comorbidities and other variables in the real-world could impact health outcomes, quality, and cost-effectiveness, helping us work towards our goal of providing new medicines that help clinicians improve patient outcomes with better value to healthcare systems.