What is Real-World Evidence in Clinical Trials?

What is Real World Evidence?
What is Real World Evidence?

Real-world evidence (RWE) is a method of gathering information from medical records and other sources about how a treatment works in practice. RWE can assist researchers in understanding practical outcomes that go beyond what they see in studies, allowing them to make more informed decisions about which treatments to make available. In fact, the FDA employs RWE to aid in making decisions about drug approval or label changes.

This is significant because there are increasingly more drugs that are being approved based on RWE rather than clinical trial results. One major reason for this is that organizations find it difficult to conduct large, long-term studies on real patients with real-world conditions and then publish their findings because there is no profit motive.

Clinical trials are tightly controlled studies in which participants are given specific interventions according to a protocol to evaluate their effects on health outcomes.

Clinical trials are created to limit variability, confounding factors, and selection bias. The study design includes randomization, blinding (where participants do not know whether they are receiving an intervention or a placebo), adequate sample size, and duration of the study. Participants in clinical trials are increasingly important in advancing science by sharing their data to help others with diseases all over the world.

The use of patient data generated outside of clinical trials and observational studies is known as “Real-World Evidence” (RWE). RWE has been shown to improve the quality and cost-effectiveness of healthcare.

The term “real-world” refers to how patients interact with their healthcare system, which can be difficult for researchers to study. To gain a better understanding of this interaction, researchers must conduct various types of RWE studies. Examples of these include:

  • Observational study: an indirect method used by doctors to gather information about their patients’ experiences with treatments or procedures. This type of study is frequently conducted on people who are unable to participate in clinical trials due to a medical condition. Researchers collect the data and can use it to conclude the efficacy of the treatment.
  • Real world data (RWD): medical record data used as RWE. It’s frequently gleaned from electronic health record systems and insurance claims databases. An example of RWD are RDAs (research data alliances), which are used to calculate the frequency of drug-related adverse events such as infections, blood clots, and strokes. They can be used to predict outcomes such as death or disability from a heart attack or stroke in patients with heart disease in some cases.

RWE is important because it provides information about how a treatment works outside of a clinical trial, which may be more relevant when deciding which treatments to use in a patients’ care. For example, if a randomized controlled trial (RCT) demonstrates that a particular drug lowers blood pressure in hypertensive patients but there are no other related studies on the topic, any conclusions drawn from the findings of the study may be inappropriate for all hypertensive patients, who may individually have different needs and requirements.

Real-World Evidence complements clinical trial data to ensure that new therapies benefit everyone who requires them.

Real-world evidence is a powerful tool for answering questions that are difficult to study in clinical trials. It can help us understand how new therapies work for everyone who requires them, including those who do not take part in clinical trials. RWE also advances our understanding of how therapy works for different people, such as children and the elderly, and ensures that it is safe and effective for all ages.

There are a few advantages of RWE studies that clinical trial data may not offer. For example, it can be used to learn about treatments that are difficult to study in randomized controlled trials or have never been tested in such trials before. Many new cancer drugs, for example, are not approved by the Food and Drug Administration (FDA) because they cannot be proven safe before being administered to patients. Some of these drugs, however, may be useful in treating other diseases such as heart disease, diabetes, and Alzheimer’s disease. In addition to this, another advantage of using RWE is that it allows physicians to see how effective a treatment is over a long period of time and a broad range of individuals, not just individuals who participate in a clinical trial that lasts a certain amount of time.

The FDA has methods for obtaining RWE, which can aid in drug approval decisions.

The FDA has implemented a program that allows it to collect RWE from patients. This data can assist the FDA in making drug approval decisions. They accomplish this in a variety of ways, including:

  • Surveying people who have used a drug about how it worked for them
  • Requesting that doctors and nurses report what happened when their patients took a specific medication

Real-World Evidence is also now used to support marketing authorization applications (MAAs) for investigational products, post-marketing surveillance, health technology assessments, comparative effectiveness research, and other applications. In addition, it can be used to support the FDA’s use of risk-benefit analyses in policymaking involving medical product labeling or marketing authorization decisions.


Real-World Evidence is a powerful tool for assisting physicians and patients in making informed treatment decisions. It is critical to remember that RWE does not replace clinical trials, but it can often supplement and compliment them in order to further advance clinical knowledge.