FDA’s Expedited IND Pilot: what will make it work for sponsors?

When a small biotech plans its first-in-human (FIH) clinical study, the date on the development plan depends on decisions across the company. A change to the manufacturing program can affect the evidence needed for the application. A site contract may depend on finance being released. Those connections matter when assessing the overall time efficiencies of FDA’s new pilot.

The US Food and Drug Administration launched the final design of its Expedited Investigational New Drug (IND) Pilot on 15 September 2026. It tests whether working with qualified research institutions, or QRIs, can help sponsors reach their first studies in people sooner. FDA expects an initial cohort of eight to ten sponsor–QRI pairs.¹

QRIs advise on the strategies and preparation of an IND submission. FDA retains authority over whether a trial may proceed.²

VCLS submitted its response to the consultation in July. We supported the ambition and asked how the arrangements would work for the companies preparing these applications. Our questions covered the timing of FDA feedback, the responsibilities of advisers, the cost of development and the evidence needed to judge success.³

Referring to public feedback, FDA’s launch announcement says, “we have incorporated that feedback into the final pilot design”.¹ While rolling review was already part of FDA’s June proposal, VCLS’s July questions concerned how it would work in practice.⁴ Having reviewed the published program against our submission, there is now useful clarification on several points. Some of the harder questions will need to be answered through implementation.

Earlier feedback needs to inform decisions

One of our questions was whether FDA would review completed components before the sponsor submitted its final IND, allowing issues to be addressed while other work continued.

The published process now describes component review during the pre-IND phase, followed by submission of the complete IND and the start of the 30-day review window. FDA describes each component as “a discrete, self-contained package”.⁵

A sponsor therefore needs to decide when a body of evidence is sufficiently complete to support a meaningful FDA review and feedback. Sending work early will be helpful if the resulting advice arrives while the team can still make a sensible change, which FDA seems cognizant of as they described the pilot.

For example, consider a hypothetical team deciding how to address an outstanding manufacturing question. If the team can clarify the concern before doing further work, it may avoid repeating an activity or revising documents late in development. Whether that happens consistently is part of what the pilot needs to establish.

While the opportunity lies in making important decisions with fewer unresolved questions, sponsors will still need a coherent application at the end of the process. Such an IND submission should enhance FDA’s efficiency during the formal IND review as well.

The QRI relationship needs to simplify the work

An IND draws together nonclinical evidence, clinical plans, and chemistry, manufacturing and controls, usually shortened to CMC. A decision in one area can affect the others.

VCLS’s July submission supported identifying advisers with expertise across those disciplines. It also warned that “the addition of independent QRIs should not burden the process and create more work for a sponsor”.³

FDA’s current pilot program provides for one designated QRI. It allows that QRI organization to supplement its capabilities with external expertise and recognizes documented partnerships for clinical trial infrastructure.²

Sponsors should agree who will reconcile advice across disciplines and how the QRI will work with the sponsor’s providers already involved. If a toxicology recommendation changes the clinical plan, someone needs to take responsibility for its effect on the other components. Otherwise, several reasonable recommendations may fail to result in a workable program.

A useful QRI relationship should make responsibilities easier to understand. The sponsor needs to be able to act on the advice and understand its consequences for the wider program. An additional review that leaves conflicting recommendations unresolved would be difficult to reconcile with the purpose of acceleration.

Sponsors need the resources to act

The funding question in our response remains particularly relevant to smaller companies:

“Engaging CROs and clinical sites requires substantial upfront investment, and early feedback alone may not meet the tangible milestone requirements needed for funding.”

CROs are contract research organizations. Bringing them and clinical sites into a program involves commitments that a development team must be able to finance.

For a company whose next investor funding payment depends on its study being permitted to proceed, encouraging regulatory feedback may still arrive before the money is available. The team could understand its next steps more clearly and remain unable to commission them.

Before relying on a compressed schedule, a sponsor and its investors should agree which activities can be funded before the final regulatory decision. The plan should identify the work that remains conditional and what a delay would mean for the available cash.

These questions do not undermine the FDA’s pilot initiative and the value of earlier advice. They help establish whether a company can use it. A development plan is more credible when the financial assumptions behind the proposed dates are explicit.

The evidence still has to support the study

VCLS’s submission also raised the time and cost involved in preparing manufacturing and nonclinical evidence. Those concerns deserve a sponsor’s attention alongside changes to the review process.³

For manufacturing, the discussion should be precise. FDA already provides for phase-appropriate current good manufacturing practice, or CGMP. Most Phase 1 investigational drugs are exempt from the detailed requirements of 21 CFR Part 211 while remaining subject to the statutory CGMP requirement. That is an existing distinction, rather than a new concession under this pilot.⁶

The practical task is to establish what evidence and controls are appropriate for the particular product and study. A small trial still needs adequate support for participant safety. A sponsor also needs to understand which work is essential now, and which work belongs at a later stage of development. Such considerations may be added by the QRI.

Earlier agreement on the scope of work could prevent a sponsor spending funds on activities that are unnecessary for the proposed FIH trial, or discovering too late that essential evidence is missing. That is the discussion to have with the QRI.

Judge the pilot through the work and time it saves

VCLS’s response proposed measures that would help distinguish a useful process from one that simply adds activities. These included how quickly FDA raises questions, the proportion resolved before the final review window, clinical holds, time from an effective IND to the first patient entering the study, and the resources used by QRIs and FDA. VCLS also asked for attention to repeated document revisions and participants’ experience of the process.³

“The pilot should be judged by the work it saves and the studies it helps sponsors start.”

FDA describes plans to gather feedback throughout the pilot. The published process does not establish that our particular set of measures has been adopted.⁵

VCLS would want the evaluation to answer several practical questions:

• Are important issues being identified early enough to change the development plan?
• Does the process reduce avoidable revisions and duplicated work?
• Can sponsors translate regulatory progress into earlier trial initiation?
• What does that progress require in adviser time, sponsor effort and expenditure?
• Is the benefit sufficient to make the approach workable for smaller companies?

These questions need context. Programs differ in their complexity and readiness. A small, selected cohort will provide useful experience, but comparisons should account for those differences before attributing a faster start to the pilot itself.

For sponsors, this is also a useful way to assess their own plans. Ideally, the pilot program should identify the decisions that are holding the program back, establish who can resolve them and check that the company has the resources to follow through.

VCLS welcomes the opportunity to test a more collaborative approach. The pilot should be judged by the work it saves and the studies it helps sponsors start. That is the outcome our July comments were intended to support.

BLOG POST BY

Sandra Cottrell, PhD, MA

Head of Regulatory and Client Strategy

Mamta Puri-Lechner, PhD.

Director, Regulatory Science