A collaborative effort between several federal research agencies is trying to change the way clinical trials are done. The motivation behind the initiative is to move away from the traditional, three-phase design in favor of incorporating biomarkers into trials. Officials spearheading this collaboration presented their work-in-progress at this year's annual American Association for Cancer Research meeting.
It's no secret that while many biomarkers have been discovered, very few have been clinically validated and even fewer are currently utilized in clinical trials. The Cancer Biomarkers Collaborative, an association composed of hundreds of researchers affiliated with AACR, FDA, and NCI, officially launched last year. Its main thrust is to formalize addressing specific challenges in biomarker discovery and validation as they pertain to clinical trials design.
"The purpose is mainly to try to put a process [in place] in which we can streamline biomarker development and integrate biomarkers into clinical trials," says Samir Khleif, who is head of NCI's cancer vaccine section and assistant to the FDA commissioner. Khleif assembled the collaborative as a part of FDA's Critical Path Initiative, an effort to modernize the process through which a drug or device goes from lab to clinic.
The CBC will focus on developing recommendations on four main areas of biomarker process development: biospecimens, bioinformatics, assay validation, and information sharing. "For example, in the biospecimen arena, we thought it's very important to establish quality standards, basically which means the biospecimens have to have specific qualities to be able to be integrated, or used for a biomarker assay," Khleif says.
Steve Gutman, director of the FDA's Office of In Vitro Diagnostics, helps co-chair the biomarker validation group. The intent, he says, is to develop "recommendations, guidance, outside perspective, and/or white papers that would help FDA advance the science of biomarker validation." Special attention is to be put on regulating processes and both analytical and clinical test validation.
The largest impact will not necessarily be on researchers or clinical labs, but on companies trying to market a specific biomarker or organizations looking to integrate a biomarker assay into clinical trials. Establishing guidelines for defining what validation means is key to being able to effectively use biomarkers for personalized medicine. "How can [a] company show that the biomarker assay is really validated to be worthwhile and robust enough to enter clinical trials?" Khleif says.