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Talking Personalized Medicine and FDA Reform

Posted by DrLouDeGennaro , 24 July 2014 · 3,293 views

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Today I had the privilege of participating in a roundtable hosted by the U.S. House Energy and Commerce Committee to discuss personalized medicine and FDA policy issues. The roundtable, comprising 12 participants from a range of fields, from patient advocacy, industry, regulatory agencies and academic research, was part of the committee’s 21st Century Cures initiative to remove impediments to therapy approval and development.

I was asked specifically to address what we are currently seeing in personalized medicine, and what the potential is for the future.

There is a need for reform in the regulatory and approval process, as more innovative personalized cancer therapies come into the pipeline that do not necessarily fit the traditional clinical trial path. LLS has been a catalyst for collaboration on a number of cutting-edge initiatives in the area of personalized medicine, and I shared these with the panel. One of the significant initiatives on the personalized medicine front I talked about was our “Beat AML” collaboration with Dr. Brian Druker at Oregon Health & Sciences University. We’ve talked about this initiative before on this blog: the goal is to identify the driver mutations that cause cases of acute myeloid leukemia (AML) and come up with the appropriate combinations of therapies to target those mutations.

I stated that our goal is to eventually do for AML what has been achieved for another leukemia, chronic myeloid leukemia, that with a targeted therapy advanced with help from LLS funding has turned a deadly cancer into a chronic, manageable disease for the vast majority of patients. It is imperative that pharmaceutical and biotechnology companies, and regulatory agencies work together to create clinical trials that co-test multiple drugs from different companies to identify the most appropriate therapy combinations for individual patients.

The panel also discussed what gaps or barriers exist in the current regulatory process that are preventing innovative life-saving therapies from getting to patients fast enough and the need for a clear regulatory pathway for companion diagnostics. Physicians have multiple treatments from which to choose when treating blood cancer patients. It is critical that physicians select the right treatment at the right time for each patient. Companion diagnostics can help identify patients who are more likely to respond to a particular treatment or who are prone to lower or higher risk for a particular side effect, both of which allow for more targeted drug therapy selection. The combination of targeted therapies and diagnostics is more likely to result in the right drug being prescribed for the right patient at the right time in the course of their disease.

The entire two-hour roundtable session can be viewed here. If you’d like to follow the discussion on 21st Century Cures on Twitter, search the hashtag #Path2Cures.

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