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FDA Report Describes Agency’s Recent and Forward-Looking Initiatives to Advance the Development of Personalized Medicine

Susan S. Lee

Susan S. Lee,

Washington, D.C.

12 November 2013

On October 29, 2013, FDA released a report titled Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development.  In a post on FDA’s blog, FDA Voice, Commissioner Hamburg noted that the report “describes many of the exciting developments and looming advances in personalized medicine, lays out the historical progress in this field, and examines FDA’s regulatory role: from ensuring the availability of safe and effective diagnostic devices, to addressing the challenges of aligning a drug with a diagnostic device, to post-market surveillance.”

The report offers many interesting statistics.  Among them, the report indicates that over 100 approved drugs contain information in their labeling about genomic biomarkers, including gene variants, functional deficiencies, expression changes, and chromosomal abnormalities.  These drugs span a broad range of therapeutic areas.  Although personalized medicine is often perceived as particularly relevant in oncology, the report shows that 62 percent of products with biomarker information in their labeling are in therapeutic areas outside of oncology, including psychiatry (17%), infectious diseases (10%), and cardiology (7%).  See Paving the Way for Personalized Medicine at 36-37.  Of these 100+ drugs that contain pharmacogenomic biomarker information in their labeling, the report identifies 34 drugs that provide specific actionable guidance in their labeling based on the biomarker information.  23 of the 34 products are oncology products, 7 are psychiatry/neurology products, and the remaining four are pulmonary, analgesic, antiviral, and hematology products.  Id. at 38.

The report also provides indicators of how FDA’s review activity in personalized medicine has accelerated in recent years.  The report notes that of the new drugs approved since 2011, approximately one-third have included some type of genetic or other biomarker data in their applications to characterize efficacy, safety, or pharmacokinetics.  The number of inter-center consults has also increased as targeted drug products must be reviewed with companion diagnostics to identify appropriate patients.  A chart in the report shows that the number of inter-center consults by the Office of In Vitro Diagnostics and Radiological Health increased from 38 in FY 2010 to 51 in FY 2011, to 117 in FY 2012.  Id. at 54.

In addition to reviewing products, FDA has been taking proactive steps to support the development of personalized medicine by developing tools and collaborating in research.  The report describes a broad range of initiatives in which the agency is involved, including a formal process to qualify biomarkers for use in drug development, and assessments of the technical performance and practical utility of emerging molecular biomarker technologies.  Id. at 42-43.  On FDA Voice, Commissioner Hamburg noted that “[o]utside collaboration and information sharing is essential for this field to flourish.”  Indeed, some of FDA’s activities in this area seem to go beyond FDA’s traditional purview.  For example, the report describes a project in which FDA personnel are working with scientists at Booz Allen Hamilton to compare different statistical methods for analyzing genomic data, in order to determine which methods may provide the highest predictive accuracy in different circumstances.  Id. at 46-47.

Overall, this expansive report provides a useful overview of FDA’s efforts in personalized medicine to date, as well as some indications of the areas that the agency is focusing on as it continues to develop its regulatory approach to the briskly evolving field of personalized medicine.

The author thanks James Allred for his assistance

Susan S. Lee

Susan S. Lee,

Washington, D.C.

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