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Realities of real-world evidence

How regulators, drug companies and payers can expand RWE from safety to efficacy

After countless conferences, white papers and speeches, stakeholders across the healthcare ecosystem are finally coming together to figure out how to extend the use of real-world evidence beyond pharmacovigilance and into drug development.

The payoffs from creating efficient systems for generating real-world evidence (RWE) as a complement to evidence generated in randomized, controlled trials (RCTs) have long been clear.

RWE is derived from analysis of real-world data (RWD), which may include observational studies, registries, retrospective database studies, case reports, administrative and healthcare claims, electronic medical records (EMRs) and pragmatic trials conducted with broad enrollment criteria in the setting of routine clinical practice (see “Spectrum of Reliance on RWD”).

Studies conducted in these real-world settings could generate data faster and more cheaply than randomized controlled trials to support the addition of new indications to approved products.

The EMA’s adaptive licensing pathway, developed with support from a precompetitive collaboration that includes industry, regulators, payers, patient advocates and academics, seeks to use RWE for this purpose following an initial, narrow approval that was based on randomized controlled trials (see “Real-World Applications”).

Real-world studies that enroll broad patient populations also could fill persistent gaps in the evidence produced by RCTs, leading to labels that are more relevant to more patients. Data from these studies also would support evidence-based coverage and reimbursement policies that enable access to medicines for the patients who are most likely to benefit -- potentially including patients not typically treated in the academic medical centers that typically participate in RCTs.

“There’s often no single truth standard when it comes to the evidence used to support medical decisions.”

Scott Gottlieb, FDA

There are also clear applications of RWE early in drug development, including to validate biomarkers and explore alternative dosing.

Industry, patient groups and FDA had these benefits in mind when they pushed Congress to include requirements in the 21st Century Cures Act for FDA to smooth the path for RWE to become a routine part of drug development, and when they included complementary provisions in PDUFA VI.

So far, though, not many real-world studies aimed at supporting regulatory decisions have made it into submissions for drug approvals.

At two workshops in September, FDA leaders, biopharmaceutical executives, payers and healthcare providers met to discuss practical ways to lift the barriers that have stood in

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