March 20, 2006
Biomarker Development and Clinical Trial Design Greatest Areas for Impact
Health and Human Services (HHS) Secretary Mike Leavitt and HHS' Food and Drug Administration (FDA) today released an initial list of priority research projects that could advance innovation in medical products. The announcement of the Critical Path Opportunities List signals the next major step in FDA's Critical Path Initiative, aimed at modernizing medical product development, so new medical discoveries are brought to patients faster and at a lower cost.
The Opportunities List outlines an initial 76 projects to bridge the gap between the quick pace of new biomedical discoveries and the slower pace at which those discoveries are currently developed into therapies. The release of the list marks a starting point in identifying priorities to be accomplished under the Critical Path Initiative. Government, industry and academic experts estimate that, if accomplished, the new tests and tools developed under the Critical Path Initiative will modernize the drug development process by 2010 and help to get new medical discoveries to patients faster and at a lower cost.
"This Opportunities List enhances the health and well-being of Americans by fostering strong, sustained scientific advances in medicine to better public health," Secretary Leavitt said. "Medical product development relies on cutting-edge scientific tools to help identify promising medical discoveries and target testing for benefits and risks when they are used by patients."
The Critical Path Opportunities List, the first specific blueprint for this nationwide modernization initiative, was developed based on feedback to the agency's 2004 Critical Path Report. The report diagnosed a slowdown in the development of innovative medical therapies, and proposed an FDA-sponsored long-term initiative to address the problem. The research projects in the Opportunities List, designed to deliver smarter tools to evaluate candidate medical products, were identified through numerous sources including public comment, inter-agency consultations, and FDA product reviewers who know first-hand the obstacles encountered by medical product development.
"Right now, researchers are trying to bring 21 st century medical innovations to market using 20 th century tools to evaluate them. Under the Critical Path Initiative, we anticipate being able to dramatically increase the success rate in moving products from the lab to the patient," said Dr. Andrew C. von Eschenbach, Acting FDA Commissioner. "The keys to a smarter more modern medical product development process are the standardization of new tools to test potential products along with the unprecedented integration of information within government, industry and academic partnerships."
The Critical Path Opportunities Report is organized into six broad topic areas: development of biomarkers; clinical trial designs, bioinformatics, manufacturing, public health needs and pediatrics. FDA's outreach efforts uncovered a consensus that the two most important areas for improving medical product development are biomarker development (Topic 1 ) and streamlining clinical trials (Topic 2).
Biomarkers are measurable characteristics in animals or humans that can help predict the performance of a product during development, reducing uncertainties about safety or effectiveness.
"Most researchers agree that a new generation of predictive biomarkers would dramatically improve the efficiency of product development, help identify safety problems before a product is on the market (and even before it is tested in humans), and facilitate the development of new types of clinical trials that will produce better data faster," said Janet Woodcock, MD, Deputy Commissioner for Operations and head of FDA's Critical Path Initiative. "Similarly, researchers stressed that reforming the clinical trial process -- both trial design and trial conduct -- would dramatically improve the efficiency of product development, which means getting products to patients faster at less cost."
Accomplishment of the research priorities will require unprecedented collaboration among public and private sector partners. To facilitate completion of these projects in a timely manner, the FDA will bring together partnerships and consortia to accomplish a majority of the projects. FDA's regulatory experience and knowledge of the hurdles in scientific research will enable the agency to play a consultative role in many of the projects. In addition, the initiative will require a new, cooperative partnership among the primary research, evaluation, approval and medical treatment delivery and reimbursement divisions of HHS, including the FDA, National Institutes of Health, (NIH), Centers for Medicare and Medicaid Services (CMS), and Agency for Healthcare Research and Quality (AHRQ).
"Coordinated leadership is essential to illuminate and align this pathway to personalized medicine to an era when doctors will eliminate -- or even prevent -- dozens of today's most-devastating diseases," said von Eschenbach. "Instead of taking a pill and hoping it works effectively and safely, patients will receive treatments designed just for them. Results will be more certain and more affordable."
Over the next few weeks, the FDA will identify several priority Critical Path research opportunities. Some of the projects in the list could be undertaken by one organization; some will require collaborations coordinated and supported by the FDA. For example, a major Critical Path undertaking announced today, which seeks to develop guidance on the use of standard biomarkers to predict safety in drug development, will be coordinated by the Critical Path (C-Path) Institute and carried out by a newly formed Predictive Safety Testing Partnership including: Bristol Meyers Squibb, Johnson & Johnson, Merck, Novartis and Pfizer. The FDA, while not a member of the Partnership, will assist it in an advisory capacity (see a separate press release).
"It is important to note that the list released today is meant to spur a continued dialog among industry, academia, patient and professional groups and government organizations about the research priorities that need to be accomplished in our effort to modernize the medical product development process," added Woodcock. "We believe it is crucial to build a national infrastructure to support and continually improve the Critical Path Initiative. Therefore, we must reach beyond specific opportunities and build collaborations to work together to encourage continued development of the Critical Path sciences."
About the Critical Path Initiative
Critical Path is the FDA's premier initiative to identify and prioritize the most pressing medical product development problems and the greatest opportunities for rapid improvement in public health benefits. Its primary purpose is to ensure that basic scientific discoveries translate more rapidly into new and better medical treatments by creating new tools to find answers about how the safety and effectiveness of new medical products can be demonstrated in faster timeframes with more certainty, at lower costs, and with better information.
Today's report, as well as other information on the Critical Path Initiative, is available at http://www.fda.gov/oc/initiatives/criticalpath/.