We’ve been hearing a lot of buzz lately on use of real-world data (RWD) and real-world evidence (RWE) in healthcare decisions. The Food and Drug Administration (FDA) defines RWD as data derived from various sources associated with real-world outcomes in a heterogeneous patient population, outside the context of randomized controlled trials (RCTs). RWE, on the other hand, is defined as clinical evidence regarding usage and potential product benefits or risks that is derived from analysis of RWD captured within the healthcare system itself.

Pictured: aggregating healthcare data icons.

In the past, RWD represented a very fragmented approach to aggregating patient data, and as such, was subject to criticism and limited use. However, with the growing abundance of measurable and standardized patient data, the design of data repositories prior to/at launch, and the emergence of numerous digital channels for gathering and storing data (e.g., computers, wearables, and other biosensors), RWD has gained heightened attention and appreciation by both the FDA and healthcare industry.  According to a 2018 benchmarking survey by Deloitte (Pharmaceutical Commerce, July 9, 2018), approximately 90 percent of global pharmaceutical companies either have or are building RWD analytical capabilities, with applications ranging across the entire product life cycle. Indeed, a growing trend among pharmaceutical companies is to build internal teams staffed with data scientists using advanced analytics and data-visualization tools.

With this context we can identify several applications for RWD, some current and some forward looking:

1. Role in value-based contracting:

There is increasing interest in using RWE to design and support new value-based contracting (VBC) programs. These contracts tie reimbursement for a drug to successful patient outcomes in the real world. While there still are some barriers that prevent wide-spread adoption of VBCs, RWE will play a critical role in their design and implementation.

2. Supporting regulatory submissions/decision making:

The 21st Century Cures Act, signed into law in 2016, places additional focus on use of RWD/RWE to support regulatory decision making, including efficient detection of adverse events to inform drug or device label changes, supporting post-approval study requirements, and supporting new indications for approved drugs—as in the example above. Last December, the FDA published a Framework for FDA’s RWE Program, which spelled out considerations that will guide the overall RWE program and inform the agency’s assessment of individual drug applications. Leveraging RWD and RWE to enhance regulatory decisions has been identified as a top strategic priority for the FDA.

3. Informing commercialization decisions:

Companies have begun to leverage RWD to expand and refine their understanding across a product’s lifecycle, which in turn can inform effective strategy and commercialization decisions. A key milestone was reached when Pfizer secured a new indication for Ibrance, a drug for men suffering from breast cancer, by using RWD. Pfizer was able to build the case for the indication through RWD compiled from electronic health records and reports outlining the use of the product in this population, which until now did not have many first-line treatment options.

4. Adapting/optimizing clinical trial design:

Drug developers are increasingly incorporating RWE-driven insights to improve clinical trial design and execution. For instance, RWE can be used to assess and validate specific hypotheses (e.g., biomarkers) to help refine trial design. Additionally, given that control arms add time/expense and it is unethical to put patients into a control arm in certain treatment areas, the ability to create a “synthetic” control arm based on RWE derived from patients treated in clinical practice can both reduce costs and streamline the regulatory process, particularly for investigational therapies intended to address unmet treatment needs in rare diseases.


While there is still much work to be done, the pharmaceutical industry and the FDA are moving in the right direction by considering and currently leveraging RWD in several arenas, with the ultimate hope of moving towards the use of RWE in clinical trials. As the FDA continues to build out its framework and develop its policy and research paradigms along these lines, when coupled with pharmaceutical companies progressing their own RWD analytic capabilities, we can expect to see RWD and RWE playing an increasingly important role in healthcare decisions.