BIO applauds efforts targeting early drug development efficiency, provides suggestions

A Food and Drug Administration (FDA) pilot program to expedite approval of new drugs contains potentially impactful initiatives but risks adding burdens, says the Biotechnology Innovation Organization (BIO).

BIO’s Aug. 24 comments respond to FDA’s request for information (RFI) on its Expedited Investigational New Drug Pilot Program. The pilot is part of the FDA’s Trialblazer initiative to test potential efficiency enhancements that could enable more rapid clinical trials and drug approvals.

BIO lauds the concept of “rolling review,” which lets a drug sponsor provide individual modules of their investigational new drug (IND) application instead of holding the application until all modules are complete. But BIO expresses concerns that the pilot’s proposal to make applicants partner with a Qualified Research Institution (QRI) adds a “third-party layer” to the process without clear benefits.

QRI questions

FDA’s pilot envisions “enlisting expert Qualified Research Institutions (QRIs), such as academic medical centers, contract research organizations, and other organizations with deep subject matter area expertise, to take on the role of a scientific partner during IND preparation.” FDA says it wants to test the “hypothesis” that QRIs would ensure applicants provide complete data, meet FDA standards, and provide submissions with recommendations from external experts.

BIO acknowledges that a QRI might help review an application but questions the benefits, given potential delays in an IND application.

“The principal risk is that the pilot could add an additional layer of review rather than reduce time to safe-to-proceed, particularly if FDA must review both the QRI’s output and the underlying components,” BIO says. “Unless QRI review is clearly decoupled from FDA’s own review, the model risks increasing workload rather than delivering efficiency gains.”

BIO lists several additional concerns about QRIs, including the new body’s unclear regulatory status, potential for variable QRI quality, over-reliance on QRI recommendations, and new bottlenecks related to a QRI’s limited resources or FDA questions about QRI qualification.

Support for rolling review

BIO supports the pilot program’s concept of rolling review, which FDA describes as follows: “FDA plans to review and accept individual components of an IND submission as they are completed, rather than waiting for a complete package.”

FDA adds that rolling review makes more sense regarding preparation for a first-in-human (FIH) clinical trial.

“In a well-designed FIH program, the nonclinical package is typically the first body of evidence ready for review, establishing the scientific rationale and safety basis for proceeding to humans. CMC (Chemistry, Manufacturing and Controls) data follows as product characterization matures. Clinical protocols and safety information come last, informed by what the nonclinical and manufacturing data have established,” according to FDA. “Today’s process ignores this natural sequence, requiring sponsors to hold everything until the full package is assembled.”

The pilot envisions QRIs overseeing rolling review, but BIO recommends that the pilot focus on proving the potential benefits of rolling review alone.

“Rolling review offers a more direct mechanism to enable earlier issue resolution and parallel preparation of nonclinical, CMC, and clinical components, and warrants continued exploration independent of the QRI framework,” BIO says. “Rolling reviews would likely result in significant time savings by enabling earlier FDA engagement and parallel preparation and earlier resolution of nonclinical, CMC, and clinical issues.”

Other considerations and recommendations

Other aspects of the pilot include clarifying IND requirements, but BIO’s comments question the benefit of “incremental improvement” and call for broader process improvements.

“In practice, delays in FIH initiation arise from a combination of factors, including front-end uncertainty, iterative rework, and downstream bottlenecks such as IRB (Institutional Review Board) review, contracting, site activation, and FDA protocol review,” along with other cost and logistics considerations, BIO says. “Meaningful impact will require a comprehensive approach that addresses the full ecosystem.”

BIO identifies a wide range of potential FDA efficiencies in recent research, including its October report on “Strategies for Enhancing FDA as Global Gold Standard,” produced in consultation with its members. BIO’s collaboration in an industry-FDA roundtable earlier this year produced detailed recommendations published in June by the Reagan-Udall Foundation for the FDA. Some recommendations from these reports are mentioned in BIO’s Aug. 24 comments.

For the current pilot program as envisioned by FDA, BIO recommends a methodology that identifies what works and what does not.

“Given the number of variables FDA appears to be testing—including the QRI model, rolling submissions, IRB review, and site activation—BIO urges FDA to design the pilot with clear success metrics that can distinguish the impact of each component,” BIO says. “Critically, success should reflect net improvement, not just faster IND clearance, and should account for whether gains are offset by delays or burdens elsewhere.”

Read BIO’s full comments for the RFI.

The post BIO applauds efforts targeting early drug development efficiency, provides suggestions appeared first on Bio.News.