In our global industry, the FDA does not want to fall behind countries like China and Australia where first in human studies have an expedited pathway. Operation Trialblazer is designed in part to do that for US companies. It is described by HHS and FDA as an “initiative to modernize and accelerate clinical drug development by reducing unnecessary regulatory burden while maintaining FDA’s standards for safety and effectiveness.” The pilot program recently announced will accept up to 10 participants from now until October 30 (Happy Halloween or Trick or Treat, take your pick) and will include “an expedited Phase 1 IND pilot; streamlined Chemistry, Manufacturing, and Controls (CMC) requirements for early development; greater use of computational tools to support dose selection; more flexible approaches to late-stage clinical trials; and new resources to help sponsors navigate the regulatory process.”
The pilot will pair sponsors with qualified research institutions (QRIs) that may include contract research organizations (CROs), CMC expert organization, clinical pharm-tox experts, certain health networks or the like to provide FDA with a more comprehensive (and hopefully complete) IND package when submitted to the FDA. This will expedite initiation of first in human trials by hopefully having all of the necessary information vetted by not only the sponsors but also the expert review of the QRIs. There will also be various FDA touchpoints and communications during the pilot program to ensure the adequacy of the documentation and quality of the data.
While the QRI and sponsor will be responsible for providing the package, and the QRI will act as a surrogate FDA with regards to providing additional expertise in data review and generation, with the goal of having a more complete package that FDA will be willing to accept when the IND is filed. FDA has said that the initial run up to first in human trials can take up to 2-3 years. The Agency hopes this program will help significantly reduce the current typical time for preparation and evaluation of the INDs. The FDA will also use a rolling review of the submissions to decrease the delays that have been seen in traditional pursuit of INDs and hopefully support an earlier in-human dosing.
The FDA webpage titled FDA Expedited Investigational New Drug (IND) Pilot Program (here) provides detailed information on program applicant prioritization, conflict of interest requirements, and the selection considerations the agency will use to determine selection of the sponsors and qualification of the selected QRI.
