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Is Your Real World Evidence Strategy Good Enough?
May 26, 2021
Real World Evidence (RWE) is finally arriving as a valued complement to clinical trials.
RWE is generated outside of the clinical trial environment, through observational data gleaned from electronic medical records (EMR), pharmacy claims, and patient reported information. It provides valuable information about the real world impact of diseases and treatment journeys.
Many stakeholders have been hesitant to use RWE due to the limited control they have over the data collection process and the inability to conduct audits of third-party data. But, as the industry hears more success stories about the benefits of RWE as external comparators for new treatments, and the use of RWE in regulatory approvals increases, stakeholders are becoming more open to using Real World Data (RWD) in their research plans.
The challenge they face now is how to capture and use RWE in a way that is transparent, reliable, and acceptable to regulators.
RWE: What regulators want to know
Earlier this year, I spoke with Dr. Amy Abernethy, then principal deputy commissioner and chief information officer at the FDA, about the changing regulatory landscape for using RWE, and how sponsors can create a robust RWE methodology.
She noted that the best source of insights into what regulators are thinking is FDA’s 2018 Framework For Real-World Evidence Program. The document offers an overarching rubric for evaluating the potential use of RWE in the submission process. It provides the context for RWE use, and can help sponsors begin to define their RWE approach and methodology.
Organizations need to determine what data they are going to collect, where it will come from, and how they will analyze it. “In a landscape where data sources are constantly changing, we want analytic methods that are as near-real time as possible and able to accommodate many use cases,” Dr. Abernethy said.
The biggest challenge for sponsors is figuring out whether the data and analytics methods are good enough for the given use case, and whether they will be acceptable to regulators.
Without clear guidance, creating an acceptable RWE strategy is complicated. But Dr. Abernethy suggested that sponsors address the following five questions to pressure test their study designs and methods, and avoid making choices that weaken the validity of their RWE approach.
- How is the data generated and where does it come from? RWD like electronic medical records (EMRs), pharmacy claims records, and patient registries are used widely, with recognized value. Good practice requires full transparency with a clear description of data provenance and procedures used for extraction and curation. An audit of the data collection process and check for traceability may be requested.
- Does the data set contain the variables you need, and are the characterizations of those variables consistent? All RWD need to be evaluated in the context of how well the data is fit for purpose, and how close it comes to satisfying the research objectives. Both the consistency as well as the accuracy of those variables are important, especially for key endpoints.
- How are data sources linked? If you plan to use data from multiple sources, you need to be able to describe how that connectivity works, and how linkages have been cross-checked to ensure consistency and accuracy in the merged data sets.
- Are the analytic methods appropriate for the task? You must be able to defend your analytic methods in the context of the data use case. For example, if you are developing real-world control arms (comparators that are external to a trial), it can be helpful to demonstrate that the RWE can replicate what is seen in other studies.
- Are your study design and methods good enough? While RWE will never provide the level of control achieved through randomization of treatment or full source data verification, RWD offers information generally not found in clinical trials. Regulators and sponsors are realizing the substantial value of well-designed and appropriately analyzed RWD, including using a hypothetical target trial to guide design, and sensitivity analyses to understand how much the effect estimates vary according to the methods and assumptions used. It’s neither RCT or RWE, but rather understanding the complementarity of both approaches and utilizing each appropriately.
Regulators’ increasing attention and acceptance of RWE offers an opportunity to create more robust submission packages that demonstrate the benefits and value of their treatments in real world settings. Those organizations that invest the time now to create a thoughtful RWE strategy that can be vetted for quality and reliability, and who are willing to talk with regulators in advance of executing any such plans, are likely to produce more compelling cases for market approval, while also quantifying clinical benefits that translate readily to real-world settings. Now is the time to embrace the use of real world evidence to enhance your regulatory submissions.
eBook: Explore Transformative RWD Sources
Discover how you can leverage RWE to accelerate study timelines, shorten time to market, cut research costs, and more. In this interactive eBook, read case examples on how key client questions were answered in various therapeutic areas and glean insights on how you can use RWD to answer challenging research questions.