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Event Title
Remarks to the National Academy of Sciences on the Impact of Real World Evidence on Medical Product Development
September 26, 2017

Remarks by Scott Gottlieb, M.D.

Commissioner of Food and Drugs

Examining the Impact of Real-World Evidence on Medical Product Development―Workshop One: Incentives
National Academy of Sciences
Washington, DC
September 19, 2017


"Advancing Public Health Opportunities with Real World Evidence"


Thank you for having me here today. I’m delighted to have the opportunity to share with you some of the efforts that FDA is pursuing to expand the opportunities we have to use real world evidence (RWE) in support of our decision-making. I want to thank the National Academies for organizing today’s workshop. A special thanks goes to Greg Simon and the members of the Planning Committee, and especially my colleagues from FDA who supported this effort.


Today’s workshop is part of a collaborative effort between FDA and the National Academies to help develop methods for collecting and evaluating real world evidence in ways that can support FDA’s work.


We’ve been talking about RWE for a number of years. But let me tell you why RWE matters to me. The more widespread use of RWE can make our medical product development process more efficient, and help lower the cost of development. More importantly, it can help make sure doctors and patients are better informed about the clinical use of new products, enabling them to make more effective, efficient medical choices. This will ultimately help us achieve better outcomes, and safer and more efficient use of expensive technology.


But there’s uncertainty among sponsors about the role RWE plays in regulatory decisions. The FDA’s interest in advancing the adoption of RWE in support of its programs remains a top priority. And it’s a high priority of mine. We need to close the evidence gap between the information we use to make FDA’s decisions, and the evidence increasingly used by the medical community, by payers, and by others charged with making healthcare decisions.


Real world evidence is becoming especially prominent as a way to make decisions on coverage and reimbursement. As the payor community makes more widespread use of these constructs, the rigor by which RWE is being collected is also gaining more precision. We’re seeing the advent of more rigorous clinical registries. We’re seeing electronic medical records (EMRs) being used in more effective ways to collect information at the point of care.


As the breadth and reliability of RWE increases, so do the opportunities for FDA to also make use of this information.


The hierarchy of evidence is evolving as a consequence. This is the hierarchy that has long defined the reliability of clinical evidence, and puts the randomized, prospective, placebo controlled trial at the top of that pyramid.


This pyramid has remained unchanged, even as our tools for collecting practical data have advanced; even as the constructs that fall underneath the fully randomized, prospective, placebo-controlled trial are expanding, and their usefulness for informing our decisions becoming more apparent; even as the reliability of other forms of evidence is increasing as the methodologies for evaluating different forms of data continue to improve. That includes RWE.


At FDA, we need to plan carefully for how we can leverage these new constructs as a way to better inform our work.


This starts by adapting how we see our role in this information matrix, including how people can talk about results based on these evidentiary standards. FDA needs to think of itself as a curator of information. Not just an arbiter, where a single truth standard is secured to a fixed orthodoxy. The latter concept is increasingly at odds with the way decisions are being made by others who depend on reliable evidence to guide their paths, including doctors and health plans. FDA is often an important, independent evaluator of information.


Given our function, and the significant role it plays in public health promotion, we want to support the development of, and access to, appropriate forms of reliable evidence that meet our standards of approval.


The fact is there’s often no single truth standard when it comes to the evidence used to support medical decisions. Clinical choices are made all the time, based on a mosaic of information of various precision and certainty. That continuum includes real world evidence, as well as the facts gleaned from rigorous and carefully fashioned trials...and a lot of evidence constructs in between.


The question for FDA is this: How do we make room for the wealth of evidence that can better inform our decisions, evidence that’s becoming more available, and more reliable? How do we fit RWE into our regulatory hierarchy?


To address these questions, and advance the use of RWE across our pre- and post-market medical review process, we’ll be publishing consensus definitions that relate to how different parts of FDA use this information as one component of our regulatory considerations. Among other things, we’ll be including a detailed description of RWE and its potential applications for satisfying aspects of FDA’s pre- and post-market requirements as part of a guidance document we’re developing. As we consider efforts to make wider use of RWE, our abiding faith will remain one thing above all else: Strengthening and advancing FDA’s gold standard for regulatory decision-making.


But make no mistake; there’s nothing in our statute or regulations that prevent FDA from using a broad range of informative sources of evidence.  On the contrary, many of our statutory responsibilities boil down to one principal calculus: What do we know, and how do we balance benefits and risk based on the fullest possible information.


This construct already exists in FDA’s statute. There’s nothing novel about this approach. FDA routinely draws inferences on how medical products behave on the basis of practical data. On the evaluation of safety, FDA regularly relies on real world evidence to make decisions. This is also spelled out in our guidance on evaluation of emerging risks. Our public health mandate drives us to look at all possible data sources to better inform our decisions. FDA has wide latitude to use evidence based on substantial clinical experience in appropriate circumstances.


For those who’d challenge the suitability of our effort to incorporate real world evidence into our regulatory model, I’d challenge you with the opposite intention: Should a product be marketed based on a data set that speaks to a limited and rigidly constructed circumstance, when the clinical use, and in turn the evidence we might have to evaluate the product, could have been far richer, far more diverse, and more informative?


The pre- and post-market evaluation of medical products is not a binary line. It’s part of a continuum. No product is all risky and uncertain one day, and completely safe and effective the next. It’s true that we often need a clear standard and line of demarcation as a way to make decisions as a regulatory agency. This is especially true when one views the medical product approval process. But we have a broader mandate as a public health agency to engage in the life cycle of how products are used. That means embracing the full continuum of evidence that informs their clinical use. We see this approach gaining more rapid and widespread acceptance when it comes to some medical products. But we need to continue to more widely advance this life cycle concept of regulation.


We can’t allow our need for a point of regulatory accountability to prevent us from looking across the line we have to draw, at practical information that’s collected both before and after our point of demarcation, when a product gains a license for initial market entry. That’s why the discussion of RWE is illuminating.  It forces us to confront certain realities. This kind of evidence can make our regulatory obligations better informed about the true benefit-risk profile of a medical product as well as provide earlier identification and a richer understanding of new safety concerns. At FDA, many groups already use RWE to make decisions, including regulatory decisions on benefits and risks.


To enable greater adoption of RWE in clinical and regulatory decisions, we’ll need to work with the healthcare system to change the way clinical information is collected. Ideally, we’d like to have a system where providers have the right incentives to enter clinically relevant information into EMRs at the point of care. But a lot of the incentives force data to be structured in ways that are geared to billing. Clinically relevant information that can tell us what’s happening to patients often remains in an unstructured notes. We’re unable to learn as much as we could about a product’s profile when that information isn’t accessible. We also need to find a better way to collect information directly from patients, because an EMR and claims data is really the patient’s perspective filtered via the provider.


FDA also needs to do its part to advance the use of RWE. I recognize that FDA isn’t always clear about our approach to RWE in regulatory decisions. So how innovators incorporate these concepts into medical product development is also likely to falter. Knowing this, FDA is taking steps to provide more clarity.


Last month, we issued final guidance on the use of RWE in the development of devices. Since early 2015 alone, we’ve approved or cleared more than eight new medical devices and expanded the use of more than six technologies based on evidence derived from RWD. This includes drug-eluting stents, transcatheter heart valves, and technologies for spinal cord stimulation and esophageal atresia, as well as in IVDs. In these cases, we’re using robust evidence that was generated in less time and at a lower cost than in the past, in some cases saving one to two years of development time. Increasingly, medical device makers are also meeting their post-market study requirements by leveraging real world data sources.


These approaches allow for more rapid data collection. It increases the likelihood that the evidence will be generated, because it’s already being gathered as a part of routine clinical practice. We’re now working on policies to support the use of RWE in the approval of new indications for already marketed drugs. This may be especially relevant in settings like rare diseases or other unmet medical needs, where it can be hard to enroll patients in clinical trials. We’re also expanding policies to enable the use of RWE to support post-approval drug study requirements.


But we must also be realistic about current limitations. Even if we made progress on the challenges, RWE won’t replace data from traditional clinical trial data in many cases. But we can achieve more opportunity in the pre- and post-market context to use RWE in settings where doing so will improve medical product development.


During today’s workshop, and in the years ahead, I want you to know that FDA will support your work on these efforts. At FDA, we intend to expand our regulatory policy development work on achieving the more appropriate adoption of RWE as part of the entire life cycle of medical products. We can’t do it alone. Your collaboration on these efforts is going to be critical to our success. 










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