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Status Quo Not an Option for Developing New Drugs Faster

By Peter Winter

Despite the availability of advanced molecular technologies and the investment of billions of dollars in research and development every year, biopharmaceutical companies have not yet managed to improve their efficiency rate for bringing novel therapies to the marketplace.

Not much has changed since this state of affairs was first identified. It has been more than eight years since the FDA released a report titled "Innovation or Stagnation? Challenge and Opportunity on the Critical Path to New Medical Products," which noted that despite advances in fields such as genomics, proteomics and nanotechnology, "there has been a downward trend in the number of innovative medical product applications to the FDA and its counterpart agencies throughout the world." The FDA's analysis was critical of the industry – identifying a slowdown instead of the expected acceleration, in innovative medical therapies reaching patients. (See BioWorld Today, March 19, 2004.)

The seminal report led the agency to launch its Critical Path Initiative designed to modernize and accelerate the drug development process by making it "less empirical, more scientific and rational."

Since the introduction of this initiative there have been significant changes in the drug discovery process – moving from a focus on discovering blockbuster drugs to creating targeted therapies, companion diagnostics and biomarkers that are contributing to a new era of personalized medicine.

However, according to statistics compiled by the FDA, the number of new molecular entities (NMEs) approved each year by the FDA has changed very little – averaging around 23 per year over the last decade.

Accelerating Drug Development

It is clear that much more needs to be done to improve upon this statistic. This is why the federal government is hoping that funding the National Center for Advancing Translational Sciences (NCATS), the newest center at the National Institutes of Health (NIH), will help accelerate the development of new drugs. The new center will encompass a range of preclinical and clinical translational science capabilities drawn from a variety of centers within NIH. One of these programs is the Cures Acceleration Network (CAN), created by the 2010 Affordable Care Act as a means of closing off the "valley of death" for early stage biotech and drug discovery. (See BioWorld Today, Dec. 28, 2011.)

In June the Forum on Drug Discovery, Development, and Translation of the Institute of Medicine (IOM) held a workshop to explore options and opportunities in the implementation of CAN in which various stakeholders discussed the potential of CAN and its mission going forward. To accelerate the development of cures, the participants at the workshop acknowledged that to be effective, CAN has to operate "out of the box" rather than merely emulating other government programs. "CAN has to have a very low tolerance for the status quo," said Joshua Boger, of Vertex Pharmaceuticals Inc., at the meeting.

(A summary of the workshop was published by the National Academies Press: Accelerating the Development of New Drugs and Diagnostics: Maximizing the Impact of the Cures Acceleration Network: Workshop Summary.)

Under CAN, NCATS may make large grant awards of up to $15 million per fiscal year, partnership awards that require 1:3 matching funds, and flexible research awards using a special funding mechanism called other transactions (OT), which allows projects to be actively managed.

New Therapeutic Uses

One of the new initiatives that NCATS recently has created is the Discovering New Therapeutic Uses for Existing Molecules collaborative pilot program, which aims to tackle problems that are beyond the scope of any one organization or sector. By giving access to compounds that have already moved several steps down the development process, including into clinical trials, the research community will have the opportunity to contribute their expertise to advancing these resources even further toward new disease indications.

For the pilot phase of the program, NCATS will provide up to $20 million to fund two- to three-year staged, cooperative agreement research grants. If specific milestones are met, funded researchers will conduct preclinical validation and clinical feasibility studies in the first stage, and proof-of-concept clinical trials in the second stage, to test whether one of the compounds may be effective against a previously unexplored disease target.

To date NCATS reports that eight participating companies, including Abbott, AstraZeneca plc, Bristol-Myers Squibb Co., Eli Lilly and Co., GlaxoSmithKline plc, Janssen Pharmaceutical Research & Development, LLC, Pfizer Inc. and Sanofi SA, have agreed to make 58 compounds available for the pilot program.

"If researchers funded through this effort can demonstrate new uses for the compounds, they could significantly reduce the amount of time it takes to get a treatment to patients in need," said Kathy L. Hudson, NCATS acting deputy director in a statement.

The pilot program also will review the use of special template agreements designed to streamline the legal and administrative process for participation by multiple organizations. The agreements provide a roadmap for handling intellectual property used in or developed through the program. According to terms of the project, participating industry partners will retain the ownership of their compounds, while academic research partners will own any intellectual property they discover through the research project with the right to publish the results of their work.