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FDA s Critical Path Opportunities Rank PGx High; Warfarin, Biomarker ID Efforts Begun

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Biomarkers, especially those useful for warfarin dosing, are top priority for the US Food and Drug Administration's Critical Path Initiative.

The FDA last week made public its Critical Path Opportunities List of 76 projects intended to result in better tools for evaluating candidate medical products, putting those most directly related to pharmacogenomics at the top. The development follows the agency's announcement earlier this month of a request for applications that aims to build research infrastructure for creating a warfarin-dosing algorithm, and it coincides with the establishment of a consortium to identify and qualify safety biomarkers.

The FDA will announce several more Critical Path research opportunities for individual organizations and groups of organizations in the next few weeks, the agency said.

The list's release marks the first major step in the Critical Path Initiative, which the agency kicked off in March 2004 in order to accelerate the development of medical products from biomedical discoveries decrease costs for drug and diagnostic makers.

In surveying opinions about the Critical Path Initiative, the FDA "uncovered a consensus" that biomarker development and streamlining clinical trials are the two most important areas for improving medical product development, the agency said in a statement. The Opportunities List is organized into six topic areas, with the first topic listed as "Better Evaluation Tools — Developing New Biomarkers and Disease Models to Improve Clinical Trials and Medical Therapy."

"Streamlining Clinical Trials" is the second topic in the opportunities list, followed by "Harnessing Bioinformatics"; "Moving Manufacturing Into the 21st Century"; "Developing Products to Address Urgent Public Health Needs"; and "Specific At-Risk Populations — Pediatrics."

The biomarker topic consists of 33 projects that the FDA hopes to undertake, and which are further categorized into five areas, the most immediately relevant to pharmacogenomics being "qualifying disease- and disorder-specific biomarkers" and "safety biomarkers." The other areas under the biomarker topic are: "qualifications and standards"; "advancing the use of imaging technologies"; and "improving predictions of human response from disease models."

Although it is not attached to a specific project within the Critical Path Opportunities List, last week's FDA warfarin dosing RFA is an important part of the initiative. The agency will provide as much as $750,000 in 2006 to develop and infrastructure to support the "Collaborative Cardiovascular Drug Safety and Biomarker Research Program," as well as to conduct research.

Research conducted using this project's infrastructure will support the development and clinical validation of a "genotype-driven warfarin dosing algorithm" and related studies, according to the RFA. The research will mark a first serious effort to prospectively, rather than retrospectively, develop and validate a pharmacogenomic method for treating patients, said Ray Woosley, president and CEO of the Critical Path Institute, in an interview this week (see this week's Perspectives for the complete interview).

The C-Path Institute, a non-profit organization formed under FDA guidance last year in a partnership between the University of Arizona and SRI International, will build the administrative and scientific infrastructure for warfarin-dosing research in collaboration with the University of Utah, the RFA said.

The Predictive Safety Testing Public/Private Consortium is a collaboration of industry, academia, and government for identifying and clinically qualifying safety biomarkers, according to an FDA statement. Its initial members include: Bristol-Meyers Squibb, Johnson & Johnson, Merck, Novartis, and Pfizer. The FDA, while not a member of the partnership, will serve as an advisor, the agency said.

The PSTPPC intends to focus on biomarkers that can improve understanding of a compound's safety profile, serve as tools for troubleshooting compounds failing preclinical testing, predict or prevent post-marketing safety issues, and improve the efficiency of preclinical drug evaluation, the statement said.

— Chris Womack ([email protected])

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