New Center could be Model for Advancing Regulatory Science, Cures

Posted 23 August 2012 By Alexander Gaffney

A new draft report published by the US Institute of Medicine (IOM) on the so-called "Valley of Death"-the gap between therapies exhibiting promise and those actually being approved for use in patients-explores the possibilities for leveraging the newly formed Cures Acceleration Network (CAN).

CAN was formed out of the 2010 Patient Protection and Affordable Care Act (PPACA), and now resides within the National Institutes of Health's (NIH) National Center for Advancing Translational Sciences (NCATS).

It describes itself as a program that aims to, "Advance the development of high need cures and reduce significant barriers between research discovery and clinical trials." Though CAN is still in its infancy, IOM notes the program, "Has the potential to catalyze widespread changes in NCATS, NIH and the drug development ecosystem in general."

Big Plans, Little Funding

Its funding, too, is still small.

It was appropriated just $10 million to operate in fiscal year 2012, and is barred from giving any one project more than $15 million per year. But its financial limitations haven't stopped it-and its advocates, including the US Food and Drug Administration (FDA)-from planning big things for the agency.

At its core, it aims to be something of a research accelerator in the model of the Department of Defense's Defense Advanced Projects Research Agency (DARPA)-a famed research outfit credited with jump-starting the development of the internet.

Through its three award programs, the Cures Acceleration Partnership Program, the Cures Acceleration Grant Program and the Cures Acceleration Flexible Research Program, CAN aims to leverage outside investment and expertise to develop tools that can accelerate product development.

"CAN is designed to be catalytic," notes the report, "which will be essential given its small initial budget." Paired with a collaborative approach, the program could get outsized results, said participants in an IOM workshop in June 2012.

The program will work to leverage existing resources in two additional ways: a mandatory one-to-three matching requirement for all grant disbursements and flexible research awards given in the model of DARPA, with CAN able to set unique conditions for each arrangement.

A 'Model for Interagency Collaboration'

Workshop participants wrote they were looking to assess a number of questions regarding the program, including how it could best interact with FDA's newly launched regulatory science initiative.

"To accelerate the development of cures, CAN will need to function differently rather than emulating other government programs," workshop participants wrote. "At the same time, it needs to work closely with other agencies, including other parts of NIH and FDA. In particular, CAN and FDA will need to communicate early and often."

Officials noted that assisting FDA's regulatory science activities in the future was a strong possibility, particularly if CAN's funding situation improves. Working on regulatory science issues with industry, too, was floated as a possibility, particularly as it would allow companies to bring their products to market faster.

"[CAN] could, as an explicit part of its work, address gaps in regulatory science that, if they were filled, could greatly improve product development," they explained. "In the process, CAN could help develop a regulatory science toolbox that could create a more efficient pathway to develop and evaluate products."

Officials said FDA has expressed "eagerness" to work collaboratively with CAN, and might later become a "model for interagency collaboration."

Specific Proposals

Three specific regulatory science proposals were floated by members of the workshop: developing new approaches to clinical studies, data creation and sharing collaborations, and early and continuous collaboration between FDA and industry to bring products to market.

The committee noted, "Scientific uncertainties can throw off a project for two to three years, and energy devoted up front to anticipating those uncertainties can be a valuable investment."

Officials also said they thought training activities, including for regulatory science, might give outsized returns for advancing cures to market. "Today, unmet needs exist in many of these areas," concluded one committee member.

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Categories: FDA

Tags: Cures Acceleration Network, CAN, IOM, Report, NIH, Latest News, regulatory science, workshop

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