The FDA is continuing its push to expedite early-stage research for drug developers and is now accepting applications for a new pilot program aimed at accelerating U.S. clinical trials. The primary goal is to help the U.S. keep up with countries like China, where early-stage drug development can move faster and at a lower cost, said Bill Holtz, a legal and regulatory strategist and counsel at Foley & Lardner.
Called the Expedited Investigational New Drug Pilot, the effort is part of a broader U.S. Department of Health and Human Services initiative dubbed Operation TrialBlazer, a multi-agency effort intended to lure drugmakers back to the U.S. from overseas by removing some of the regulatory hurdles that slow domestic early-stage clinical research.
The TrialBlazer operation includes many regulatory components, and on the FDA side, agency officials are working to clarify expectations to make getting to phase 1 easier, Holtz said.
The Expedited IND Pilot Program is designed to pair up drug developers with qualified research institutions that can then work together to expedite the IND application process. QRIs can include academic centers, CROs, health networks, regulatory advisors or other types of research organizations. Sponsors can apply with a prospective QRI until Oct. 30.
From there the FDA expects to announce the selection of eight to 10 sponsor/QRI teams by December, although the timeline could vary based on the number of submissions. The selected teams will work together to prepare IND applications, and the FDA will review components on a rolling basis as they are completed, allowing sponsors to get real-time feedback.
The pilot doesn’t change regulatory requirements or standards, but it has the potential to move drugs from the lab into human subjects faster. And it’s not only about speed.
“Earlier alignment can give sponsors greater clarity on the key regulatory, clinical and operational requirements of an IND program, enabling better-informed decisions earlier in development,” said Scott Tarrant, executive vice president of drug development advisory at the contract research organization Veristat.
While some details about the program remain uncertain, it will likely be an appealing option for some biotechs or smaller drug trial sponsors in particular. Here are five facts companies should know about the pilot.
Small companies stand to benefit
While larger pharma companies have more experience and resources to navigate regulatory filings, the process often isn’t as easy for small startups that have staffing and budget constraints. This can mean that some companies end up doing more work than they need to, Holtz said.
Gaining early and ongoing feedback from the FDA through this pilot could help small companies avoid missteps and unnecessary effort.
“It's an opportunity for early, earlier and more frequent interaction, which I think can only be a good thing,” Holtz said.
For that reason, larger companies could also benefit from the program, and the FDA said that it wants companies of all sizes in the mix.
It could provide an opportunity for more than just procedural assistance
To participate in the pilot program, sponsors will pair up with a QRI and submit a joint proposal on how they would work together to get to phase 1.
Holtz described this as a formalization of existing QRI sponsor relationships, and the FDA will study interactions to see if they can expedite the path into human trials.
“This pairing up with QRIs is a pretty original concept, and I'm interested to see how it goes,” Holtz said.
Under this pilot program, QRIs can also help guide sponsors in other areas, from pharmacology to manufacturing.
“The QRI component could be especially valuable if companies view these relationships as a source of practical guidance rather than simply another step in the process,” Tarrant said. “Sponsors that engage the right partners early, with experience in the relevant therapeutic area or development approach, will be better positioned to navigate the program effectively and take advantage of the efficiencies it is designed to create.”
The program will likely be competitive and companies may need a unique pitch
The FDA’s published criteria say the agency will prioritize novel products headed for a first-in-human phase 1 trial in the U.S. It also wants a mix of therapeutic areas, products, sponsor sizes and types of QRIs.
The pilot will also likely be competitive given the small number of spots.
“I do think they'll probably get quite a few submissions,” he said. “I don't expect [FDA] to grant people into this program who just have a molecule in mind.”
The program will also test new ways to get trials off the ground
The pilot will help determine whether running trial site setup and Institutional Review Board review, which provides an independent ethics review of study protocols, alongside IND development can save time.
“The other thing that is being tested here is whether setting up an IRB, getting IRB review of a proposed phase 1 trial, and/or setting up the clinical trial sites, can be done during this process,” Holtz said. “If it's being developed with FDA's hand in the mix, perhaps [an IRB] would be willing to complete an earlier review of the study.”
But whether running reviews like this concurrently will shorten trial timelines remains to be seen.
The agency is looking at more than speed
FDA will ultimately evaluate how effective QRI pairings are in multiple ways.
“Let's say that it's faster by some degree. Does it save money?” Holtz said.
FDA pointed to Australia and China as international benchmarks for increasing the speed of early-stage trials.
“It's going to have to be a pretty efficient program to overcome the benefits that going to China currently represents,” Holtz said.
Ultimately, the pilot program could generate evidence that shapes how the FDA supports early research going forward, the agency said.
“I'm really interested to see how it turns out,” Holtz said. “It’s an interesting model that has potential.”
