U.S. Food and Drug Administration (FDA)

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The U.S. Food and Drug Administration (FDA) is an agency within the U.S. Department of Health and Human Services (HHS). The FDA protects public health by assuring safety, effectiveness, and security of drugs, vaccines and other biological products, medical devices, and the food supply.

The FDA Critical Path Initiative

In March of 2005, the Food and Drug Administration signaled a shift from its traditional approach to the evaluation and approval of medicines that will affect public health. It announced the approval of a genetic test called the Roche Amplichip that will help doctors determine which drugs will have fewer side effects and work better in patients. In doing so, the FDA noted that the new test was part of the agency’s effort to encourage the use of pharmacogenomics, or personalized medicine, which will “allow medicines to be uniquely crafted to maximize their therapeutic benefits and minimize their potential risks for each patient." [1]

The announcement was part of a larger effort launched by the FDA called the Critical Path Initiative, a program that wants to use genetic tools, approaches to drug evaluation that rely on faster computers, new imaging techniques like molecular PET scans and electronic patient records to make personalized medicine part of the drug development process and the practice of medicine.

The ultimate goal is to speed the development of important new drugs to market and at the same time insure that people get the medicines best for them. it’s 2003 white paper: "Innovation/Stagnation: Challenge and Opportunity on the Critical Path to New Medical Products." The current regulatory approach is focused on ensuring that every product is safe and effective for the general population. Such an approach makes failures likely for products that otherwise might actually be safe and effective for use by specific subpopulations or on an individualize basis.

As FDA Deputy Comissioner, Dr. Janet Woodcock has noted, the vision shaping the Critical Path is that “..we can get better outcomes for people and get a higher percentage of people who actually respond to any given treatment. Instead of your doctor telling you that 40% of the population responds to a drug, he can tell you that if you take this drug, you have a 90% chance of responding.


Our vision is that there aren't bad drugs or good drugs. Instead, some drugs run into bad problems with a small subset of people. Instead of taking all those drugs off the market or putting warnings all over them, we need to make sure that people who are at high risk for a side effect don't get the drug in the first place. Instead of taking all those drugs off the market or putting warnings all over them, we need to make sure that people who are at high risk for a side effect don't get the drug in the first place. “

The tools – and several are already in use and in development - to achieve this vision are already having a profound impact on research and to a less extent, the practice of medicine. At the core of this transformation within the FDA and the medical research community is the development and use of biomarkers. Biomarkers are biological measurement of disease progression, pharmacology, or safety that can be used as basis for decision making in drug development or medical treatments. Because of advances in our understanding of how genetic variations shape response to medicines and disease, biomarkers that identify genetic variations in people has been identified by the FDA and the scientific community as an important new tool for the personalization of medicine. The key to this shift to targeted treatment is the continuing integrating of bioinformatics with electronic health records at the patient level. Over the next decade, this confluence of technologies will they will will help screen out people according to who is likely to be respond to a treatment or possible side effects because of genetic variations. They will be used to both develop new drugs and who should get them. They will allow scientists and doctors to predict disease, prescribe medicines, sort out people according to the mechanism or path a disease uses to strike at patients and monitor the progression of illness. And because the cost and speed of the computers required to collect and share such information is declining exponentially, the ability to use these tools to transform the Critical Path, the practice of medicine and the public health are great indeed.

FDA White Paper Innovation or Stagnation

White Paper Innovation or Stagnation

The Critical Path (C-Path Institute)

The FDA has also released a Critical Path Opportunities List, some of which deal with EPR, biobanking, biospecimens, etc.

Headed up by world re-known pharmacologist and cardiologist Raymond Woosley, the Critical Path Institute was created by Dr. Woosley to serves as a "neutral ground" for scientists from the FDA, academia and industry to work together for the implementation of Critical Path activities as well as efforts designed to promote the use of bioinformatics and EPR to promote better clinical practice.


  1. U.S. Food and Drug Administration Website http://www.fda.gov/