NCI BRB Translational Research

Translational Research is the movement of novel concepts from the research laboratory into the clinic in order to establish a proof-of-principle. The BRB strives to accomplish this part of its mission through myriad avenues: Intramural and peer-reviewed extramural research that funnel promising projects into the program; special competitions such as NExT that allow the program to choose the most developed projects for biopharmaceutical development; and other government and non-government collaborations that provide a mechanism for biopharmaceutical development of non-cancer related entities such as vaccines and therapies for bio-terrorism threats. The current capacity for the BRB is approximately 20 projects each year. Questions pertaining to the access of BRB resources in translational research may be directed to the BRB Program Directors.

Projects are selected for novelty and innovation over derivatives of existing approaches. Many concepts previously have been considered by Big Pharma and Biotech, but declined due to uncertain technology, regulatory hurdles, and/or lack of market potential. Because of the technological and regulatory uncertainties, only 5-10% of the projects that the BRB accepts will eventually make it to licensure. This “success rate” of one to two projects each year is not the only measure of success in the BRB. Success for a BRB project is defined as reproducibly manufacturing a well-characterized, stable biopharmaceutical that is safe and potentially efficacious for administration to humans in a Phase I or II clinical study. Additionally, the project is successful when a larger entity, such as Big Pharma becomes interested in accepting the product and taking it to licensure.

The selection process helps to maximize the success rate by eliminating as many of the technological and regulatory uncertainties as possible. In addition to the prerequisites of the various programs listed above, the BRB requires that potential investigators complete a Generic Questionnaire that delves into the specifics of their project/application. Responses to the questions give the BRB and other reviewers a clearer understanding of the level to which the project is clinically developed, i.e., the more thorough the responses, the closer the project will likely be to the clinic. The BRB has provided below a downloadable questionnaire that it provides to potential investigators.

Biologics Product Development Questionnaire