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Scott Gottlieb, M.D. @SGottliebFDA
, 23 tweets, 10 min read Read on Twitter
As a science-based agency we rely on evidence generated from data to inform regulatory decisions. #FDA's #SundayTweetorial discusses how we use data from real-world settings to enhance what we know about medical products. This week we'll announce policies to advance these efforts
What I’m talking about is the use of “real-world data” (RWD) to generate “real-world evidence” (RWE), concepts promoted by Congress in 21st Century Cures Act of 2016 and of great interest to #FDA To better understand our efforts, let’s define some terms.
RWD are data relating to patient health status and/or delivery of health care routinely collected from a variety of sources, including health insurance claims, electronic health records (EHRs), product and disease registries, lab results, patient-generated data and mobile devices
RWE refers to the clinical evidence about the usage and potential benefits or risks of a medical product derived from an analysis of RWD, analysis that can include randomized trials performed outside of a traditional randomized clinical trial (RCT) and observational studies.
Here’s why RWD is useful. Traditional RCTs provide the high-quality evidence #FDA needs to determine whether a product is safe and effective for its intended use but not info about long-term outcomes in broader patient groups as the product moves out of the study setting.
Also, CTs may be conducted in select hospitals and clinics, provide more intensive medical monitoring and exclude patients at later disease stage on multiple other treatments or with other diseases; even when these are the types of patients who may receive a product if approved.
Careful use of RWD can provide #FDA with a more comprehensive picture of how a product works in broader patient populations outside of a controlled research setting. Our challenge has been how to tap into high-quality RWD in a systematic way to generate RWE.
The Sentinel System launched in 2008 is our first major routine, large-scale use of RWE. Our partners are insurance companies and health plans who own their claims and pharmacy data, kept in a standard format. They can quickly answer FDA queries while protecting patient privacy
Sentinel conducts active postmarket risk ID and analysis. When safety questions arise about a medical product, we ask our partners to scan their data (> 292 million unique patient identifiers from 2000-2017) and report to us with a summary of the results.
Sentinel data have eliminated the need for postmarketing studies on 9 potential safety issues involving 5 products. Such studies typically require yrs. to design and complete and cost millions. We’ve also used Sentinel to understand patterns of opioid use
The National Evaluation System for Health Technology coordinating center is our public-private enterprise for collecting medical device RWE; with agreements with 12 organizations from 195 hospitals, 4,000 outpatient clinics and 495 million patient records
It will soon launch pilot programs testing the use of patient registries and claims data to evaluate total joint and knee replacement surgery, different wound closure techniques and the safety of intervertebral body fusion devices for spinal conditions
Most RWE efforts to date have been focused on postmarket safety questions. But RWD data from patient registries, natural history studies of the course of a disease and chart studies have been used for comparison arms in some single arm cancer and rare disease trials.
We’ve used RWE for premarket decisions on medical devices, particularly new indications for use, as we did in 2013 by extending a transcatheter heart valve to patients with inoperable aortic valve stenosis based in part on data from patient registries.
Additional RWE use requires assessing RWD quality and our analytical methods. Claims data is structured with medical care info from many health systems. EHRs provide a wider range of info - lab results, imaging and clinical assessments but are often unstructured and inconsistent.
FDA is funding a study by Brigham & Women's Hospital & Harvard Medical School, seeking to use claims databases to replicate 30 randomized clinical trials (RCTs) to determine whether RWE can reliably reach the same effectiveness conclusion about a product as an RCT.
INFORMED, a data group in our Oncology Center of Excellence, is collaborating with Flatiron Health and CancerLinQ in use of RWD to understand real-world use of new immunotherapies and assess completeness, accuracy and consistency of electronic health data
And #FDA is supporting the first RCT in Sentinel to test an educational intervention to address the underuse of effective medications to reduce stroke in patients with atrial fibrillation. It could serve as proof of concept for future RWE trials.
RWE will still need to meet the same high standards as all evidence submitted to FDA to support a regulatory decision. In most cases it won’t be the only source of evidence. Instead, it will help provide a broader picture of a product’s safety and effectiveness over its lifecycle
Moreover, extensive use of RWD in the conduct and design of traditional clinical trials has the potential to make our medical product development processes more efficient and help lower the cost of development.
Consider our MyStudiesapp which makes it easy for patients to input RWD and transfer it to their EHR for use in CTs and registries. We’ve released its source code and technical docs for others to use and there’s a Sentinel pilot of it underway.
More RWD/RWE work is coming. This week we’ll be releasing a new 2019 Strategic Framework for evaluating the potential use of RWD and RWE in regulatory decisions. We discussed this framework at a recent workshop we participated in.
Our goal is to close the evidence gap between the information we use to make #FDA’s decisions and the evidence used and collected by the medical community to provide patients with better care and more informed treatment decisions.
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