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FDA and NIH to Collaborate on Regulatory Science Initiative to Advance Personalized Medicine


Originally published Jan. 20.

By Turna Ray

The National Institutes of Health and the US Food and Drug Administration will work together to update FDA's regulatory initiatives to advance personalized medicine, representatives from both government entities said this week.

According to comments from Kathy Hudson, NIH deputy director for science, and Carlos Peña, director of emerging technology programs at FDA's Office of the Commissioner, leaders from the two government bodies will be putting their heads together to help FDA bring its regulations up to speed with technological and scientific advancements.

"We have created a joint leadership program with FDA and the expectation is that we would be able to provide them with some of our scientific expertise, be able to help them streamline some of their processes," Hudson said this week at the Personalized Medicine World Conference in Mountain View, Calif.

In June, NIH Director Francis Collins and FDA Commissioner Margaret Hamburg co-wrote an article in the New England Journal of Medicine in which they discussed their "shared vision of personalized medicine and the scientific and regulatory structure needed to support its growth.

"Together, we have been focusing on the best ways to develop new therapies and optimize prescribing by steering patients to the right drug at the right dose at the right time," Collins and Hamburg wrote in NEJM. They outlined several policy and scientific challenges hindering broad adoption of personalized medicine, and vowed to engage their two agencies to overcome these hurdles.

"To make progress, the NIH and the FDA will invest in advancing translational and regulatory science, better define regulatory pathways for coordinated approval of codeveloped diagnostics and therapeutics, develop risk-based approaches for appropriate review of diagnostics to more accurately assess their validity and clinical utility, and make information about tests readily available," Collins and Hamburg wrote.

Now, it seems their joint vision is being implemented into a formal inter-agency collaborative framework.

In October, the FDA released a report called Advancing Regulatory Science in which it outlined research areas to which it plans to apply "the bulk" of its $25 million FY 2011 budget increase designated for regulatory science initiatives. Under the regulatory science initiative, the FDA plans to form external collaborations with industry, academia, and other government entities, to "transform product development" in the area of toxicology and personalized medicine. Other collaborations will concentrate on advancing the science of regulating emerging technologies; information sciences for health outcomes; and addressing unmet public health needs (PGx Reporter 10/06/10).

Among the various regulatory science collaborations, an NIH/FDA leadership council will work together to advance the initiatives under the regulatory science framework.

Discussing FDA's regulatory science initiative at the meeting in Mountain View this week, Peña acknowledged that the laws guiding the agency's regulation of medical products "were not written with personalized medicine in mind." As such, the FDA must work together with other government institutions, industry, and academia to figure out the best way forward.

Peña noted that under FDA's regulatory science initiative, NIH leaders can advise the FDA from a research perspective, so the agency can formulate submission requirements that advance personalized medicine without being overly burdensome to industry. "It really helps for FDA to become more engaged in these [joint] forums, especially in the research side, because it helps the FDA understand some of the challenges and make more of a predictable path for all the stakeholders who have submissions before the agency," Peña said.

Hudson, during her talk at the conference, said "there is the possibility of [NIH] doing more for [FDA] than we have historically."

As an example, Hudson mused that a potential area where NIH can help FDA is in conducting some of the post-marketing studies that aren't currently being done. "There are these unique situations where drugs are approved … under an accelerated review with an agreement from the company that they will collect more data, and more often times than not the companies are unable or unwilling to collect that data," Hudson said. "Yet, [these products] are out there on the market, and benefiting individual patients, so FDA is reticent to snag those products off the market."

Post-marketing studies to investigate drugs in smaller, genomically defined subpopulations of patients are precisely the types of trials that industry might be unable to do since they are costly, or may be unwilling to conduct since such analysis might constrict the market for a profitable drug.

"Maybe that's the role for the NIH, to do those types of trials, so the products can reach important patient populations, and [allow drugs to] stay on the market," Hudson noted.

There are other changes happening within NIH focused on downstream translational research efforts, many of which may advance personalized medicine.

Recently, NIH announced it would create the National Center for Advancing Translational Science, which will build a national infrastructure for translational medicine, support the development of new diagnostics and therapeutics, and provide resources for collaborations between academia and the private sector.

Therapeutics for Rare and Neglected Diseases, or TRND, is an NIH program launched in 2009 with $24 million in funding that would fall under NCATS. The program is NIH's first foray into creating a drug development pipeline at the institute by collaborating with researchers working on treatments for rare and neglected diseases.

"We … will continue to have the majority of our work focused on understanding disease, target identification, and basic biomedical research," Hudson said. "But increasingly we're focusing our attention further down the developmental pipeline in assay development and high-throughput screening through our molecular libraries initiative, in preclinical projects such as TRND, and we are also starting to move into the clinical trials space through partnerships with pharma and biotech."

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