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Cancer Biomarkers Collaborative Outlines Potential Guidance

SAN DIEGO (GenomeWeb News) – A multi-agency collaboration unveiled a set of recommendations for biomarker development and oversight at a special session during the American Association of Cancer Research annual meeting here on Sunday.
As researchers chase the goal of personalized medicine, there is an increasing need for adequate biomarkers and a plan for verifying and regulating them, experts say. Now hundreds of collaborators from the AACR, the US Food and Drug Administration, and the National Cancer Institute have identified the barriers and challenges currently hindering biomarker development and have developed a set of strategies to address them.
“It will lead to a major paradigm shift in the way we do business,” Samir Khleif, head of the NCI’s cancer vaccine section and assistant to the FDA commissioner, said during the session.
The idea for the AACR-FDA-NCI Cancer Biomarkers Collaborative was born from a cancer biomarker think tank in 2006. The collaboration — more than 120 members strong — was officially launched last year.
At the AACR meeting yesterday, members of the team described some of the recommendations they plan to present to the FDA — suggestions that could ultimately serve as the cornerstone of the agency’s biomarker oversight, taking biomarkers from basic science to FDA approval.
“The regulatory environment is one that we have the opportunity to build,” outgoing AACR President William Hait, senior vice president and worldwide head of hematology oncology research development at Johnson and Johnson, said during the session.
Currently, the FDA requires researchers to adequately demonstrate a treatment’s safety and efficacy, typically through Phase I, II, and III clinical trials. And randomized control trials have been the “gold standard for the past 45 or 50 years,” Khleif explained.
But, he added, the approach is far from perfect. For every 10,000 promising compounds, only one new drug is approved, at great time and expense. Even then, Khleif noted, approved drugs often don’t work for everyone. That means many people that don’t benefit from treatments are exposed to the drug — and potential side effects — anyway.
“We have an issue of predictability,” Khleif said. “We need to apply new methodologies.” 
Theoretically, biomarkers based on individuals' genetic variability could facilitate personalized medicine, predicting who will benefit most from existing and future drug treatments. They are also promising tools for predicting patient outcomes in general, diagnosing diseases, and understanding an individual’s metabolism.
But while basic research has uncovered many biomarkers, Khleif said, methods for effectively translating and verifying that knowledge are lacking. And, like drugs, the impact of biomarkers needs to be determined and evaluated.
The current regulatory system is not designed to address biomarker development, he added. That’s something the CBC is trying to change. It has developed recommendations related to pertinent issues including biospecimen collection and handling, biomarker analysis and validation, bioinformatics, data standardization and sharing, and regulatory guidelines for biomarker oversight.
While it’s clear that implementing such guidelines could be challenging — particularly with respect to resource sharing and developing appropriate industry business models, the collaborators said the changes are both possible and necessary. “The fear in the industry, if we don’t develop ways to do this, is no one will want to buy our drugs,” Hait said.
The CBC plans to publish several white papers, which it will share with the FDA as soon as possible. “The AACR-FDA-NCI Cancer Biomarker Collaborative is very proud of trying to spearhead this effort,” Hait said.

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