RAPS is closely monitoring developments in the Coronavirus (COVID-19) outbreak. See our public safety page for the latest updates.

The RAPS store will be under maintenance Saturday, 13 June between 6 AM and 12 PM EST. Store functionality may be unavailable at times during this window.
We apologize for any inconvenience caused during this time.

Regulatory Focus™ > News Articles > FDA Says Focus on Regulatory Science Key to Advancing Personalized Medicine and Helping Patients

FDA Says Focus on Regulatory Science Key to Advancing Personalized Medicine and Helping Patients

Posted 29 October 2013 | By Alexander Gaffney, RAC

A new report published by the US Food and Drug Administration (FDA) seeks to further advance its frequent narrative that good regulation is good for both business and the American public, this time focusing on how its efforts in the area have advanced personalized medicine.


The term "regulatory science," which FDA defines as the "science of developing new tools, standards and approaches to assess the safety, efficacy, quality and performance of all FDA-regulated products," has been central to many of FDA's efforts in the last three years.

In issue after issue and appearance after appearance, FDA Commissioner Margaret Hamburg has touted the approach, saying it's one way for regulators to better accomplish their jobs and avoid the scrutiny of skeptical politicians.

"Frankly, these days, at least in Washington, you are far more likely to hear the word "regulatory" linked to the phrase "job-killing" than to "science," quipped Hamburg in an April 2012 appearance before a New England Healthcare Institute (NEHI) conference . "But believe me, this is a short-sighted view."

Efforts to advance public understanding of the term has taken center stage in the last two years at FDA, with FDA going as far as to launch a campaign in November 2012 intended to promote regulatory science to the public in plain terms. Elsewhere, FDA has made a point of partnering with industry and academic groups to advance regulatory science, such as with LifeScience Alley and the Brookings Institute.

In all, FDA says it wants to spend as much as $50 million on regulatory science-related activities during the current fiscal year.

Benefits of Regulatory Science

But how does these efforts benefit the public? FDA is glad you asked. In August 2013, for example, FDA touted the role of regulatory science in helping it to assess new medical countermeasures to guard against radiation sickness in the event of a nuclear or radiological attack or disaster in the US.

Countermeasures, though, are unlikely-thankfully-to ever be used by the majority of the American public. What they are likely to use are medications for various ailments, such as cancer or cystic fibrosis.

In its 29 October 2013 report, Paving the Way for Personalized Medicine: FDA's Role in a New Era of Medical Product Development, FDA said its efforts to recognize cutting-edge regulatory science have allowed highly-targeted medicines to benefit patients in new ways, giving them access to treatments that might not have been approved in years past.

"The concept of personalized medicine is not new: clinicians have long observed that patients with similar symptoms may have different illnesses, with different causes; and similarly, that medical interventions may work well in some patients with a disease but not in others with apparently the same disease," wrote Hamburg. "What is new is that advances in a wide range of fields from genomics to medical imaging to regenerative medicine, along with increased computational power and the advent of mobile and wireless capability and other technologies, are allowing patients to be treated and monitored more precisely and effectively and in ways that better meet their individual needs."

High-Profile Approvals

The report highlights several high-profile approvals, including Vertex Pharmaceutical's Kalydeco (ivacaftor), a treatment for cystic fibrosis that specifically targets the G551D gene associated with an even rarer subset (4% of patients) of the rare disease, treating its underlying cause instead of just its symptoms. For FDA, the drug's approval was notable for several reasons. First, it was approved in just three months, an incredibly short time period for any drug. Second, it was co-developed with the Cystic Fibrosis Foundation (CFF), a patient group who has been pivotal in gathering patient genetic information to build a robust patient registry. It was this same genetic data that permitted FDA and Vertex to approve the drug in record time.

Similar success stories have been common in the oncology space, with FDA highlighting the approvals of crizotinib, vemurafinib, dabrafenic and tremetinib, all of which were approved based on patients' unique genetic characteristics.

An evolution in FDA's understanding of how to "bridge" its regulatory processes to meet these scientific developments-pharmacogenomic-related advances, mostly-has permitted those same products to be approved, ushering in a new era of personalized medicine, regulators wrote.

The intent of the report, Hamburg added, is to serve as a "compendium of FDA's many recent efforts to advance regulatory standards, methods and tools in support of personalized medicine and to further refine critical regulatory processes and policies in order to bring about personalized medical product development."

Paving the Way for Personalized Medicine: FDA's Role in a New Era of Medical Product Development

Focus Articles on Regulatory Science

Regulatory Focus newsletters

All the biggest regulatory news and happenings.