Frequently Asked Questions

Real-World Data/Real-World Evidence

Real-word data (RWD) and real-world evidence (RWE) are considered by the FDA (as part of the 21^st Century Cures Act) to be highly credible and critical sources of information to help guide clinical decision-making for life-saving therapeutic interventions. This data represents the missing link between the prescribed clinical intervention and the patient’s real-time, unique, physiological response.

Clinical research trials are the gold standard for many conditions, however, especially in the case of new or rare diagnoses, we don’t have the ability to engage in a controlled trial. Creating a control group in a traditional clinical setting would fail to provide those patients with the appropriate standard of medical care. It’s unreasonable to expect patients who present to the hospital with life-threatening symptoms to participate in a study that very well may include them receiving a placebo rather than a life-saving treatment.

In this sense, real-world data can provide insights that even a clinical trial cannot; the information garnered represents outcomes of various treatments prescribed by qualified medical professionals using their best clinical judgment to treat their patients in a real-world setting. These unique findings support the reason Congress mandated that the FDA use RWD in making regulatory decisions.

What exactly is real-world data (RWD)?

Real-world data are the data relating to patient health status and/or the delivery of health care routinely collected from heterogeneous patient populations in real-world settings from a variety of sources, including:

Electronic health records (EHRs)
Claims and billing activities
Product and disease registries
Patient-generated data including in-home use settings
Data gathered from other sources that can inform on health status, such as mobile devices

What is real-world evidence (RWE)?

Real-world evidence is the clinical evidence obtained from real-world data regarding the usage and potential benefits or risks of a medical product. These are observational data, generated from clinical practice/experience, obtained outside the context of stringent clinical research involving randomized controlled trials.

How are real-world evidence (RWE) and real-world data (RWD) used?

Real-world evidence and real-world data are being increasingly analyzed by stakeholders across the healthcare continuum who need immediate access to outcomes data based on current, real-time interventions for specific patient cohorts.

Clinicians, including physicians, nurses, pharmacists and others are interested in real-world evidence to:

Inform clinical decision-making on treatment protocols
Assess patients’ treatment compliance
Monitor the safety of newly approved interventions
Learn about adverse events associated with interventions
Develop guidelines and decision support tools for clinical practice

Payers employ real-world evidence from claims and billing data to:

Evaluate the cost–effectiveness of interventions
Help make drug coverage decisions
Improve the affordability of healthcare

Pharmaceutical manufacturers utilize real-world data to:

Improve their ability to acquire useful evidence about products
Introduce drugs to market more quickly
Expand drug usage to new indications more quickly
Answer critical questions to guide the safe and appropriate use of drugs

What is the FDA’s position on using RWD to help inform medical decision-making?

In 2016, Congress passed the 21st Century Cures Act. In that Act, Congress defined RWE as data regarding the usage, and the potential benefits or risks, of a drug derived from sources other than traditional clinical trials. The Act places additional focus on the use of these types of data to support regulatory decision making, including approval of new indications for approved drugs. According to

the FDA, the health care community is using these data to develop guidelines and decision support tools for use in clinical practice, and the FDA uses RWD and RWE to make regulatory decisions.

How do real-world data compare with randomized controlled trial data obtained from clinical research trials?

Randomized controlled trials are considered the “gold standard” for determining whether treatments or interventions are effective. However, these trials include relatively small patient populations that represent only a fraction of a disease population. The data derived from these clinical trials are often used to acquire regulatory approval for new drugs or interventions that make them readily available for patients.

By contrast, real-world evidence provides insights on treatment outcomes, for real people in real-world environments, outside of tightly controlled, unnatural clinical trial settings accounting for such variables as age, gender, race and comorbidities, and the results pertain to a much larger, heterogenous patient population.

Medical decision-making with and without clinical trial studies

Scenario A: Patient presents to the ED with chest pain. The medical team assesses him for a possible heart attack and orders vital signs, an EKG and labs. The patient is diagnosed with a heart attack and is treated with interventions recommended by long-standing clinical research.

Scenario B: Patient presents to the ED with fever, cough and shortness of breath. The medical team assesses him for one of several lung conditions but rules out common diagnoses and classifies patient as ‘suspected COVID-19’. Patient’s health begins to deteriorate. With no clinical research trial data, the physicians have 2 choices to determine the most efficacious treatment plan:

Assess anecdotal evidence from colleagues that’s based on limited experience and lacks reliable and statistical value
Analyze real-world data based on current prescribing patterns and the resulting outcomes across distinct patient subgroups

See how Agilum is using RWD to impact COVID-19 treatment protocols

By leveraging our Comparative Rapid Cycle Analytics™ (CRCA™) platform, Agilum has written protocols to analyze real-world data (RWD) from the inpatient care setting taking place in hospitals — both nationwide and specific to New York state — treating patients with COVID-19.