February 2016, Vol. 5, No. 1

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Personalized Medicine at FDA: 2015 Progress Report

Daryl Pritchard, PhD

The Last Word

Daryl,Pritchard98pxOne year after the initial observation by the Personalized Medicine Coalition (PMC) of an increased rate of personalized medicine approvals at the FDA Center for Drug Evaluation and Research (CDER), the most recent data show that the trend has continued, with oncology leading the way for the field.

A new analysis from PMC, titled 2015 Progress Report: Personalized Medicine at FDA, illustrates the leading role of CDER in the transformation of healthcare from a one-size-fits-all, trial-and-error medicine to a targeted approach that utilizes each patient’s molecular information. CDER approved 45 novel new drugs (NNDs), either new molecular entities or new therapeutic biologics, in 2015. Of these 45 NNDs, 13—or 28%—were personalized medicines as classified by PMC, thus continuing the trend that began in 2014, when 21% of NNDs were classified as personalized medicines (see Figure for the complete list of the 13 personalized medicines approved by the FDA in 2015).


Nowhere is the transformation of healthcare toward personalized medicine more clear than in oncology. Of the 13 personalized NNDs in 2015, 5 are oncology drugs—Alecensa (alectinib), Tagrisso (osimertinib), Cotellic (cobimetinib), Lonsurf (trifluridine and tipiracil), and Ibrance (palbociclib). These drugs account for 35% of the 14 oncology NNDs approved in 2015. CDER also approved a number of significant new indications for previously approved cancer drugs, including Iressa (gefitinib), Opdivo (nivolumab), and Keytruda (pembrolizumab), that redefine and expand the target populations for these drugs, thereby providing patients with more effective personalized treatment options.

The progress is driven in large part by the biopharmaceutical industry’s commitment to personalized medicine in oncology. According to a study released last year by the Tufts Center for the Study of Drug Development, 73% of oncology drugs in the pipeline involve biomarkers in their research and development design. With CDER continuing to recognize the value of personalized treatment strategies, research and development is accelerating. An emerging concept just 10 years ago, personalized medicines have gone from an occasional annual therapeutic product approval to more than 25% of all FDA approvals in 2015. All of this illustrates the prominence of personalized medicine in healthcare.

There remain, of course, many challenges, particularly in the areas of scientific discovery, diagnostic regulatory policy, reimbursement, and integration of new technologies into clinical practice, but these data clearly demonstrate that personalized medicine is more than a passing trend.

PMC’s analysis, which contains a methodology description, is available on the coalition’s website (www.personalizedmedicinecoalition.org).

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