Personalized medicine, which involves tailoring medical treatment to an individual patient’s characteristics, needs, and preferences, is advancing because of new technology and discoveries in various fields, including genomics, medical imaging, and devices. FDA’s report, “Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development,” published in October 2013, describes FDA’s recent efforts to advance regulatory standards, methods, and tools in support of personalized medicine, said FDA Commissioner, Margaret Hamburg, in the introduction to the report.
The report describes the concept and various definitions of personalized medicine and notes that it typically involves both a diagnostic device (e.g., assays to measure genetic factors and imaging equipment) and a therapeutic product. FDA’s role and responsibilities in this field include considering benefits and risks when evaluating medical products, providing guidance to encourage development, being informed about advances and ensuring that information is used appropriately throughout the industry, and working with stakeholders to evaluate and validate new diagnostics and therapeutics. FDA is responsible for “identifying opportunities for streamlining regulatory processes and advancing the science and tools that will help drive innovation,” notes the report.
FDA is building an infrastructure to support personalized medicine. The Center for Devices and Radiological Health (CDRH), for example, created the Office of In Vitro Diagnostic Device Evaluation and Safety (OIVD) in 2002 as a single organizational unit for comprehensive regulation of in vitro diagnostic devices (IVDs) that includes divisions of Immunology and Hematology, Chemistry and Toxicology, and Microbiology. In 2013, OIVD incorporated products related to radiological health and was renamed as the Office of In Vitro Diagnostics and Radiological Health (OIR). Also in 2013, the National Center for Toxicological Research, a laboratory research center that supports FDA, established a new Division of Bioinformatics and Biostatistics that will strengthen personalized medicine and pharmacogenomics efforts.
A challenge for FDA in the personalized medicine field is ensuring the availability of safe and effective diagnostics, which are considered medical devices and thus are regulated by FDA. Evaluating these tests and developing standards to demonstrate their validity can be complicated, particularly when the understanding and meaning of a genetic association is still changing. Another challenge is the interdependency of products that are regulated by different authorities, such as a drugs and devices, including companion diagnostics, for which FDA issued a draft guidance.
Postmarket surveillance is crucial to the success of personalized medicine, and the report describes some of FDA’s efforts in this area. The report also discusses FDA projects in support of advancing the fundamental science, research, technology, and tools needed by FDA to assess personalized medicine products. “The era of personalized medicine has clearly arrived,” conclude the report’s authors, who reaffirm FDA’s commitment to working with stakeholders to move the field forward.