Aetion, the global leader in real-world evidence (RWE) technology and analytics, is pleased to announce that ‘A Screening Tool to Evaluate whether Using Real-world Data to Support an Effectiveness Claim in an FDA Application has Regulatory Feasibility (SURF)’ was published today in Clinical Pharmacology & Therapeutics. SURF intends to help sponsors make an initial feasibility assessment for using real-world data (RWD) to provide substantial evidence of effectiveness to support a U.S. Food and Drug Administration (FDA) approval.

In recent years, FDA has made considerable progress in advancing its RWE program. From FDA’s RWE framework and associated guidance to recent approvals that rely partially or entirely on RWE, the Agency has demonstrated its openness to evidence generation approaches beyond randomized controlled trials (RCT). As mentioned in the SURF publication, sponsors may be able to most successfully use RWE when RCTs are not feasible, and the unmet medical need represents an urgent public health issue, thus increasing efficiency in clinical development and improving the chance of regulatory success.

“The SURF tool is based on Aetion’s experience, and our review of FDA guidance and RWE uses in Agency approvals. We have seen sponsors struggling with decisions about whether to conduct an external control arm (ECA), and in publicly available use cases, we have seen many ECAs disregarded by FDA. We applied these learnings to create SURF, which intends to provide sponsors with straightforward and actionable guidance around the use of RWE for effectiveness claims,” said Nicolle Gatto, Ph.D., M.P.H., Chief Science Officer at Aetion. “This actionable guidance can help sponsors distinguish early on between clinical development programs that are good candidates for RWE use from those that are not, and prioritize resources accordingly.”

The SURF screening tool consists of a short series of questions about clinical development program characteristics that collectively represent circumstances in which an RWE approach for providing evidence of effectiveness in an application may be acceptable. While success ultimately depends on robust study design, data, and interpretable findings, sponsors can use SURF to determine whether an RWE approach is worth pursuing, given the considerable time and cost associated with assessing study design and data feasibility.

SURF adds to the set of resources—including SPACE, SPIFD, and SPIFD2—Aetion scientists have led and co-authored to support the successful use of RWE in regulatory applications and to meet regulatory commitments using RWE.

Source: Company Press Release