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To get an update on the emerging and evolving role of real-world data in the global effort towards high value health care, we sat down with Pfizer’s Andy Schmeltz, Senior Vice President, Patient & Health Impact. Here are the highlights from our interview.
Q. We are hearing so much these days about real-world data. What is it, and why does it matter?
The randomized, double-blind, controlled clinical study has been and continues to be the gold standard to evaluate the safety and efficacy of any drug, and is essential to providing robust evidence about the benefits and risks of a medicine. However, given the controlled conditions, those studies cannot provide complete information about what may happen when the drug is used in the "real world" – where certain conditions and variables in each patient’s life may come into play. The patient populations in clinical trials are typically more limited versus those in clinical practice due to the need to minimize “noise” in detecting treatment efficacy and safety signals, and certain populations may be inadequately represented. Also, individuals may behave differently when under clinical trial constraints, and may be, for example, more vigilant with taking medications than in routine clinical practice. Despite how thorough clinical trials are, there is always more to learn once a drug is approved and goes into real-world use in routine clinical settings. Collecting and analyzing large amounts of real-world data is a way to understand individual patient experiences and provide healthcare professionals all over the world with additional evidence so that they can make more informed decisions to improve care for patients.
Q. What new information can real-world data analyses provide to physicians?
Because we can look at information from the experience of large numbers of people, we can zoom in on subgroups that would be difficult or even impossible to derive from randomized clinical trials. Using data from electronic health records (EHR), insurance claims databases and patient registries, we can gain deeper insights into how medicines work in the “real world”. The emerging wealth of data lets us study associations with our medicines in patients in routine clinical setting conditions, and share the data with healthcare professionals so that they can use this knowledge in everyday practice.
Real-world data can provide additional information on the safety, effectiveness, adherence and even costs associated with different treatments in routine medical practice. Moreover, these analyses also allow us to compare the effectiveness associations of different interventions in patients over time, and share that knowledge with healthcare professionals worldwide to help inform treatment decisions for their patients.
But these data need to be interpreted with caution. The quality of these data can vary greatly depending on the database, how the data are compiled, and whether these are collected with research purposes in mind. The source and type of data, as well as lack of controls compared to clinical studies, may limit the generalizability of the results. For this reason, adequate methodologies need to be used to account for, as much as possible, potential biases inherent in observational data analyses.
This is why safety and effectiveness results from real-world data studies should be interpreted as supplementary to clinical trial data when used for clinical decisions.
Q. What are the benefits to patients from real-world data?
Analysis of real-world data has the potential to unlock evidence and insights that can help improve patient care. It can provide insight into important questions about the benefits and risks of different medicines, surgical procedures, or other healthcare interventions as well as their relative costs. This knowledge enables patients to have more informed discussions with their healthcare professionals about their choices in care.
Q. What are some ways that real-world data can speed drug development?
One of the exciting benefits of real-world data is that it can help us design better, more efficient clinical trials. This is important because clinical trial design and recruitment of patients are among the major variables that affect how long it takes for a new drug to proceed from the laboratory through clinical evaluation 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 may be able to bring new medicines to patients in need.
Researchers are also increasingly turning towards hybrid “pragmatic trials” – studies that combine some of the rigorous elements of randomized, controlled trials with more real-world clinical settings. That means picking endpoints that are specifically useful to clinicians and the types of patients that doctors are likely to encounter in their practice.
Ultimately, these trials may impact the future of modern clinical trial design, offering physicians and payors a deeper understanding of the most suitable treatment approaches at both the patient and population levels.
Increasingly, regulatory bodies are also beginning to explore the use and potential benefits of real-world evidence. The 21st Century Cures Act, signed into U.S. law in 2016, acknowledges the value of real-world evidence, as well as the need to establish a program to evaluate its potential use in supporting the review of new indications for previously approved medicines and helping support post-approval study requirements.
Q. How do real world data help inform payors?
We are working with healthcare organizations and payors to analyze real-world data to provide insights that can help inform formulary placement, access and reimbursement of our medicines for patients.
By looking across aggregate data for thousands – or millions – of patients via claims databases, registries or health-record data, we can more easily spot where a drug is providing value, especially economic value. By going beyond the questions typically asked in clinical trials, we can offer payors important information about how appropriate use of our drugs can improve outcomes and reduce cost.
We are also working to develop predictive models that analyze and predict future benefit of different treatment approaches based on real-world data, enabling more informed decisions based on the future value of medicines to patients and society.
Q. How is Pfizer advancing the use of real-world data?
We're living in a new era with wide access to large amounts of data from various sources and stronger capabilities to mine that data for insights and evidence. Recognizing immense potential, Pfizer has invested heavily in real-world data and novel analytics, and is committed to using these to improve patient care and support decision-making by healthcare systems and providers who are focused on health outcomes, quality and cost-effectiveness. This is what we mean by better value healthcare.