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U.S. Department of Health and Human Services

Science & Research

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Personalized Medicine

FDA’s Unique Role and Responsibilities in Personalized Medicine

 photo montage depicting personalized medicine applications

The term "personalized medicine" is often described as providing "the right patient with the right drug at the right dose at the right time."   More broadly, "personalized medicine" may be thought of as the tailoring of medical treatment to the individual characteristics, needs, and preferences of a patient during all stages of care, including prevention, diagnosis, treatment, and follow-up.

Consistent with FDA's core mission, the agency is working in collaboration with researchers, manufacturers of drugs, medical devices and biologics, health care professionals and others to better understand and adapt to the promise of personalized medicine.  FDA's ongoing efforts to make personalized medicine possible touch on various facets of product development and use including:

 

Early Stage Development

FDA is helping to speed the development of promising new therapeutics by developing regulatory science standards, reference libraries, research methods, and tools that are needed for integrating genetic and other biomarker information into drug and device development and clinical decision-making. Below are some examples of how FDA is doing this.

Regulatory Pathways and Policies

More than a decade ago, FDA began putting in place the regulatory processes, policies and infrastructure that can support the challenges of regulating personalized medicine products.  Certain offices within the agency’s National Center for Toxicological Research, the Center for Drugs, the Center for Biologics and the Center for Devices were restructured to better adapt to the scientific and regulatory challenges posed by personalized research. In addition, a new position of Deputy Commissioner for Medical Products and Tobacco and accompanying office were established to provide high-level coordination and leadership for these efforts.

FDA has also issued guidance documents and regulations that seek to clarify regulatory requirements, coordinate premarket reviews, delineate the activities and responsibilities of the different centers and provide consistency and timeliness in the oversight of personalized medicine products. They
provide guidance on a broad range of topics, such as incorporating genetic and other biomarker information in drug development programs, designing clinical trials to incorporate biomarker data, coordinating cross-labeling activities, evaluating pharmacogenomics data, and demonstrating companion diagnostic test performance.

Product Use

Given the nature of personalized medicine, the FDA places high priority on helping to ensure that the agency, drug manufacturers, physicians and patients have adequate information about the product and its use. Product labeling and tracking of use in the marketplace are critical to the proper application of personalized medication tools.

Product Labeling

The FDA requires product labeling to be balanced, scientifically accurate and not misleading, and that clear instructions be communicated to healthcare practitioners for drug prescribing and/or administration.  Personalized medicines that may only be safe and effective in particular sub-populations, or must be administered in different doses in different sub-populations, must be labeled accordingly. To date, the labeling of more than 100 approved drugs contain information on genomic biomarkers (including gene variants, functional deficiencies, expression changes, chromosomal abnormalities, and others). 

Post-market Surveillance

While personalized medicine will likely allow for more focused clinical trials by increasing the proportion of responders in the trial or increasing the average effect size, or both, one implication of dramatically smaller pre-market exposure is a general increase in the importance of and emphasis on post-market monitoring, because relatively rare adverse events, in particular, are unlikely to show up when a drug is being tested in a small population, may arise when a broader population is treated. Post-market surveillance, then, is critical to the success of personalized medicine.

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