With the creation of the Federal Drug Administration’s (FDA’s) real world evidence (RWE) program, and the release of several draft guidances, organizations such as payers and providers are beginning to evaluate how to incorporate RWE into their clinical care and research initiatives. While traditional trials involving placebo control groups will remain a critical tool for drug development, there are a growing number of opportunities, especially within precision medicine and rare disease research, to supplement with real world data (RWD).  
 
Knowing when and where to use RWD, which data sets are most relevant, and how to prove its value to stakeholders is critical to successfully implementing and utilizing the resource. It’s no longer a question of if organizations will adopt RWD but when.  
 
How RWE is Proving its Value 
As the industry continues to evolve and places more emphasis on personalized medicine, RWE is becoming an invaluable tool for advancing treatment innovations. Especially when focused on rare, complex diseases, biopharma and life sciences organizations will need to have a robust foundation of data on the patient population to secure stakeholder funding and conduct successful clinical trials.  
 
Examples of how organizations can benefit from and prove the value of RWE include illustrating market size, unmet needs, morbidity and mortality, and patient burden (e.g., financial, social, psychological, etc.). Additionally, RWE will be a vital tool for clinical trial feasibility, granular patient matching based on phenotypic biomarkers, and regulatory approval for single-arm trial drug development. However, to gain the most value from and investment in RWE, understanding its proper application and collaborating with industry stakeholders will be key.  
 
Understanding RWD and its Proper Application 
There are countless sources of RWD, from patient registries to electronic health records (EHRs), that can be used in various settings. To gain valuable insight from RWD, you first need to know what information is necessary for a specific goal and what the stakeholder needs are. What is relevant for a payer may not be for a physician, and what’s relevant for a regulator may not be as important to a patient, so knowing what is appropriate based on your goals and audience is critical.
 
Using a degenerative eye disease as an example, the priority for regulators may be to determine if, after a certain amount of time, the patient can remain on the same line of an eye chart in their ophthalmologist’s office – an attempt at a standard outcome measure. However, from the patient’s perspective, the priorities are likely more focused on their activities of daily living, like the ability to drive or maintain mobility, rather than eye chart performance.  
 
The Importance of Interoperability and Collective Engagement
Different industry stakeholders can bring valuable insight into a variety of aspects of a patient’s health and disease etiology. Patient registries offer unmatched insight into longitudinal and self-reported patient outcomes; EHRs provide a snapshot of clinical encounters and lab testing; and researchers bring historical, scientific data on drug development, efficacy, and safety.  
 
In order to make an impact in these early discussions with regulators, organizations should come together to share their collective knowledge about the successes of RWE. Arguably, the strongest approach will be for different organizations to share case studies highlighting the power of RWD at each stage of the clinical care and research process and its ability to advance treatment development.
 
As the quality and accessibility of RWD improves, adoption will undoubtedly increase, but data standards alone will not eliminate all the challenges that have hindered progress to date. The most powerful tool organizations likely have is partnering with each other to develop mutually beneficial strategies and ensure patient needs stay front and center.  
 
Next Steps 
Looking forward, we anticipate seeing more reliance on RWD by life science companies attempting to secure product investment and clinical trial recruitment, especially for rare diseases with very small patient populations. While the pandemic has shown how quickly things can shift in the face of necessity, regulator change is a slow-moving process. To take an active role in shaping regulations and stay competitive in the evolving landscape of patient care, organizations need to be proactive.  
 
This may include gathering RWD to secure stakeholder funding in a new disease treatment, such as market size and patient burden, or using RWD as an external comparator for single-arm trials. Whichever avenue your organization chooses, there are a multitude of opportunities for organizations to gain and demonstrate the value of RWD based on their unique goals and needs. Regardless of its application, when patients are front and center, and the use of RWE to improve their quality of life becomes a collective effort, the way we understand and treat diseases will reach new heights.