How Recent FDA Actions Impact Sponsors

One of FDA’s main focuses at the moment is streamlining the drug approval process. In June of this year, the agency announced a new pilot program designed specifically to shorten the early-stage drug trial timelines.

A key factor of this is the growing acceptance of RWD in trial design. The agency hopes that by allowing RWD collected by qualified research institutions, drug developers will not only be able to move through early stages faster, but also reduce the need for late-stage trials as well.

Pharmaceutical Executive spoke with Jen Lamppa, VP of commercial strategy, how RWD impacts trial design, specifically during the patient eligibility screening phase. The addition of this data is expected to impact recruitment timelines, although it may have other unintended impacts as well. As such, pharma and biotech companies will likely need to adjust their recruitment models to properly take advantage of the new environment.

Pharmaceutical Executive: What recent FDA actions are impacting sponsors?
Jen Lamppa: The FDA's recent guidance encouraging sponsors to leverage real-world data is a wholly positive signal. That guidance largely focuses on the use of real-world data in the evidence package — specifically, the ability of sponsors to use real-world data in some cases without providing the identified patient record. That is a fair limitation of many real-world data sources that have been aggregated for registry and research use, and the FDA's increasing tolerance and acceptance of de-identified real-world data as part of the evidence package is, in effect, an invitation for sponsors to explore how to also use that data upstream to streamline trial starts and patient enrollment.

If sponsors are investing in real-world data and making it part of critical study workflows, the most effective approach is to start by profiling patients and designing studies with a higher likelihood of completion — enrolling diverse and representative patient populations without the undue restrictions that legacy study designs have historically imposed. From there, that investment carries through to finding where those patients are, pre-screening them, and providing supplemental information to validate that a patient belongs in that study. All of that ultimately feeds into the eventual collection of patient-specific data for the study data package.

The FDA's movement to encourage real-world data use through downstream acceptance is, in effect, a call to action for sponsors to begin exploring — if they haven't already — how to use it upstream as well. Doing so will make the entire process more efficient and generate greater value from an investment that is already being made.