The Reality Behind Real-World Data and Real-World Evidence

Pharmaceutical companies must stay on top of advances in real-world evidence.



As regulators, payers, and pharmaceutical companies require more robust evidence around the true effectiveness of new therapeutics and medical devices, they have increasingly relied on real-world data and real-world evidence. The terms “real-world data” and “real-world evidence” are often used interchangeably. Yet in reality, they are quite different concepts. 

A core aspect of healthcare involves the fundamental ability to access data that describe the current standard of care, as well as gaps and deficiencies in the care model, and social or patient-reported data, such as patient reported outcomes.  This is where real-world data comes from – a myriad of sources that when linked together provide a view of a patient’s health history that can be acted upon using insights from advanced analytics. 

Finding Patterns in Data

From real-world data, advanced analytics can extract meaningful patterns of information that will help clinicians make appropriate treatment decisions. Analytics can be directed to a specific patient population, stage of a disease, or even a group of health providers.  Some analytics methods focus on finding patterns that were not previously seen or known – using machine learning and other advanced approaches to let the data itself reveal patterns and relationships that provide new insights. 

Real-world data and advanced analytics are changing how clinical trials are designed, how medical affairs experts may identify the ‘long responders’ to specific treatment approaches, and how commercial organizations are evaluating the effectiveness of patient services programs. Increasingly, sources of real-world data provide a focus on the total population within a region or country, encompassing the various questions and insights that distribution data or chart reviews may have provided in the past. 

Evidence as Key Conclusions

Now, the term real-world evidence has generally referred to the key conclusions that could be derived from published studies in peer-reviewed journals.  For instance, the organ-system focused cancer groups at the American Society of Clinical Oncologists and at the National Comprehensive Cancer Network use published studies to develop their recommendations for new treatment guidelines targeting very narrowly defined patient populations. 

The information in these published studies comes from independent and company-sponsored research performed within targeted populations that often range from a few hundred to a few thousand patients.  The process usually takes place over several years, and encompasses study concept, study funding, study execution, the summarization of results, publication of the information, and the definition of guidelines.

Traditional evidence has multiple uses today. Leading academic centers harness evidence and their own data to develop specific treatment approaches, pathways and order sets -standardized lists of treatment steps, diagnostics and therapeutics for a specific diagnosis - to make consistent and high quality treatment decisions. Health insurers and payers use guidelines and evidence as the basis for determining whether to support patient access and reimbursement coverage. Pharmaceutical, biopharmaceutical, and medical device companies need to constantly consult these studies to assess and determine their own strategies for undertaking follow-on studies, creating value dossiers and for medical communications. 

Evidence Generating Insights

By contrast, real-world evidence involves using the growing wealth of real-world data, increasingly at the population level, to generate meaningful insights.  Real-world evidence essentially is a product of analyzed real-world data. With real-world evidence, the rigor of analysis should be comparable to randomized and controlled clinical studies, be they regulatory or observational. The strength of real-world evidence should be that it’s a new form of evidence in itself that complements traditional sources, and which holds the promise to more significantly bring patient benefit because of its greater representativeness and timeliness. 

Such groups as the Patient-Centered Outcomes Research Institute are advancing novel designs, such as pragmatic studies, which test the effectiveness of a therapeutic in a broad routine clinical practice and which bring the rigor of regulatory trials to observational, real-world studies.  Companies too are undertaking direct sponsorship of real-work studies and real-world registry-like approaches.  Real-world evidence provides the validation between the results seen in regulatory clinical studies that initially supported approval and the post-approval validation process, which showed that consistent or improving benefit was being realized. 

A New Foundation

Real-world evidence is now becoming the foundation for new pricing strategies which are more explicitly linked to therapeutic value for patients and health outcomes benefits to health systems and risk-bearers. This evidence provides the support and confidence needed to undertake value-based contracting, deploy patient services, and to run the emerging new patient care management businesses that best manage care processes and resource utilization to optimize value. 

Real-world evidence provides a new foundation, or language, for establishing business-to-business relationships between or for having leadership conversations between large risk-bearing health systems and increasingly analytic-centric health insurers and payers. 

Pharmaceutical companies must stay on top of advances in real-world evidence through their own investment and partnerships in real-world data sources and advanced real-world analytics services. They should be reshaping their necessary skillsets to best address the value, market, and financial opportunities that real-world evidence will offer them.


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