In a report released on October 29, 2013, the U.S. Food and Drug Administration (FDA) announced its continued commitment to advancing personalized medicine. “Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development” (“Report”) outlines the FDA’s unique role and responsibilities that it has in helping usher in medical products and technologies that are key to personalized medicine. The Report also outlined the FDA’s ongoing work with health care professionals, researchers, medical device and drug manufacturers to bring safe and effective personalized treatments to the public. The Report is a comprehensive overview of the rapid growth of personalized medicine and its unique challenges.

The FDA’s View of, and Commitment to, Personalized Medicine

Personalized medicine is often defined as the tailoring of medical treatment to the individual characteristics, needs, and preferences of an individual patient during all stages of care, including prevention, diagnosis, treatment, and follow-up. The Report noted that personalized medicine is not limited to pharmaceutical therapy, but also  includes advances in computational power, medical imaging, mobile medicine and regenerative medicine.

In addition to reviewing the internal structural and administrative changes that the FDA recently implemented to advance personalized medicine, the Report noted that the following significant challenges remain:

  • limited understanding of the intrinsic biology of disease;
  • common conditions involving multiple genes/biomarkers;
  • an outdated disease classification;
  • lack of agency infrastructure;
  • clinical implementation of new diagnostics;
  • investment uncertainties; and
  • limitations on access to personalized therapeutics.

The Report concludes by noting that while many of the challenges are beyond the scope of the FDA’s set of roles and responsibilities, the agency is committed to working in concert with all key stakeholders to find solutions that will help move this promising field forward. Moreover, the FDA announced its continued commitment to facilitate the development of personalized medicine by advancing the science and tools that will drive innovation and collaboration in the field while continuing to identify opportunities to streamline regulatory processes.

A copy of the Report is available here.