A billion-dollar U.S. government drug development center, which will focus on moving potential new discoveries out of the lab and into early-phase trials, could help biopharmaceutical companies improve their success rate in developing new treatments by reducing the risk of failure in early-stage research.

According to the Pharmaceutical Research Manufacturers of America (PhRMA), the biopharmaceutical research sector invested an estimated $65.3 billion to discover and develop new medicines in 2009. Yet, on average, one medicine was approved for every 10,000 molecules studied. As a result, it has become increasingly difficult for the pharmaceutical industry to continue supporting the cost and time required for new drug discovery.

The center, to be called the National Center for Advancing Translational Sciences, was proposed to address widespread concerns about the slowing pace of new drugs coming out of the pharmaceutical industry. Health and Human Services Secretary Kathleen Sebelius told Congress the center, which will be set up within the National Institutes of Health (NIH), will be up and running by October.

The federal research center could have a significant impact, in particular, on the development of new treatments for rare and orphan diseases. Many discoveries have been made recently in this area, but drug sponsors haven’t shown interest in spending the time and money necessary to pursue these treatments since the diseases affect a limited number of patients. To support development in these areas, NIH Director Francis Collins said NIH-funded researchers could explore compounds through early stages in the drug-development pipeline to “de-risk” projects that would otherwise be disregarded.

The idea behind the center, which Collins endorsed, will be for NIH investigators to evaluate novel potential drug targets and develop promising compound leads, doing some work that has traditionally been done by drug companies, in order to provide enough information for pharmaceutical companies to invest in further drug development. According to Collins, the goal is to develop each project far enough to interest industry in finishing the job. For a new molecular entity to treat a common disease, drug-sponsor interest would be high early in the project. But a treatment for a rare disease, on the other hand, might not interest a drug sponsor until later in development.

David Wheadon, senior vice president of PhRMA, said the biopharmaceutical industry is “encouraged” by Collin’s goal to focus on early-stage research in the hope of improving ultimate success rates in drug development.

In addition, the new center won’t try to compete with drug companies in developing medications for common diseases. Instead, the NIH website said it will focus on neglected diseases and on drugs for diseases that “attract little commercial” attention. Companies including Pfizer, Novartis and Sanofi-aventis recently have shown interest in developing drugs for rare diseases.

Wheadon said the collaboration between industry, NIH and academia can help drive innovation in drug development, particularly in early-stage research. “Bold and ambitious proposals, such as Dr. Collins’, will be key to how we collectively progress in discovering novel compounds for addressing patients’ unmet medical needs,” he said.

While Wheadon called the new center worthwhile, he also noted that biopharmaceutical companies create the “vast majority” of new medicines from start to finish, saying, “The fact remains that biopharmaceutical companies today and in the future will play a pivotal role.”

 —Karyn Korieth