Real world evidence, or RWE for short, is the term made famous by the 21st Century Cures Act, a law enacted by the United States Congress in December 2016.
Section 3022 of the bill calls for the FDA to establish a program and framework for evaluating the use of RWE, defined as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials.”
In other words, RWE is health care information gathered outside of a typical clinical trial environment. Instead, the data is collected from “atypical sources, including electronic health records, billing databases, and product and disease registries — to assess the safety and effectiveness of drugs and devices.” . To clarify the term, and to provide guidance on the relevance and proper collection of RWE, the FDA has also provided a Use of Real-World Evidence Document.
The push for RWE encourages pharmaceutical companies to collect data about drug usage following FDA approval as well as during actual patient interaction. The benefits of RWE for patients, pharmaceutical companies and the healthcare industry as a whole are significant.
RWE’s Superiority Over In-Clinic Trial Data
The ability to gather large data sets from drug users in differentiated populations and environments allows researchers and drug developers to identify side effects and adverse reactions that may not have been evident in a clinical trial setting. Data from electronic health records and personal health devices can tell the story of a drug’s or a digital health product’s success (or lack thereof) in real patients’ lives.
As the NEJM Catalyst points out, “Many digital health products that demonstrate impressive results in clinical trials often fail to do so in real-world settings.” One reason for this discrepancy can be the unusual level of engagement and compliance in a trial setting. “Clinical trials are among the most engaging environments in health care,” says the article’s author. “Clinical trials involve the use of a variety of tools (e.g., training, close monitoring, payments) to ensure that patients use the technologies appropriately, but few of these tools are used in the real world.”
Why Pharma Companies Prefer RWE
In February 2017, Abbott Diabetes Care shared RWE demonstrating the effectiveness of the company’s FreeStyle Libre System for glucose monitoring. MobiHealthNews quoted Dr. Mahmood Kazemi, Abbott Diabetes Care Medical Director, on the Real World Data release: “...the device doesn’t only have tangible benefits in clinical trials, which can be rather artificial settings of rather selected populations, but in fact apply to the broad range of users that are out there using the device on a daily basis."
Additionally, SAS noted in its November 2015 article, “Real-world evidence: Why pharma firms are investing,” the large data sets provided by RWE allow subsets of patients to be identified, paving the way for more personalized care as well as leading to improved recommendations for medication use and best practice.
One of the most meaningful implications of the 21st Century Cures Act, according to MobiHealth, is the component that “allows Pharma companies to do post-market launch research around ‘real world use’ (read: off-label) of drug products. If data suggests positive outcomes of off label use, this evidence can be used to expand the indications of use for that drug. This game changer will allow biopharma companies to save tens to hundreds of millions of dollars as the previous standard practice of expanding indications required re-running costly randomized control trials.”
Real World Evidence is benefitting patients, manufacturers, and pharma companies as the abilities of drugs and devices are identified more quickly and efficiently, leading to better outcomes and expanded indications for use. Additionally, payers and providers looking to verify the cost-efficiency and ROI of a medication can look to RWE to guide their decision-making.