In his recent opinion piece (NIH Pharma Co. Is a Bad Idea, March 15), Henry I. Miller, M.D., argues against the NIH’s proposal to create a new center specifically designed to speed development and testing of novel diagnostics and therapeutics aimed at a wide range of human diseases.1 I disagree with Dr. Miller.
This center, to be called the National Center for Advancing Translational Sciences (NCATS), is not only a good idea, it’s a necessary one. In this article, I will explain why the public sector must participate in the search for new medicines and how NCATS will help. I will also give several examples of how federal funds are already advancing drug development and saving lives.
Dr. Miller believes that drug development is better left in the hands of private pharmaceutical and biotechnology companies. While I am equally frustrated with the slow pace of new drugs coming out of the pipeline, private industry cannot bear this weight alone.
Pharmaceutical companies are extremely good at taking promising new compounds through the later stages of preclinical development, clinical trials, and the FDA approval process. They are even willing to spend billions of dollars and as many as 10 years doing it. However, private companies and venture capitalists are increasingly reluctant to fund the crucial early stages of preclinical development—the research necessary to “translate” promising discoveries made in laboratories into optimized candidate therapeutics ready for testing in clinical trials.
This gap includes many steps in the drug discovery and development process, including assay development, high-throughput screening, medicinal chemistry, exploratory pharmacology, and rigorous preclinical testing of drug efficacy and safety in animal models of disease.
This situation leaves us with the aptly named “Valley of Death”—the large research and funding gap that sets federally funded basic researchers (those of us in nonprofit research institutions, academia, hospitals, and federal laboratories) on one side and the pharmaceutical industry on the other. Few companies are able to reach far enough backward to bridge that gap—the costs and risks are just too high for organizations that are responsible for delivering financial results to their investors and shareholders.
Enter the NIH’s newly proposed center for translational sciences—just the shot in the arm basic research needs to reach forward across that valley.
The goal of this new NIH center is not to make, test, and market drugs in competition with private industry. Rather, this translational science center will provide federal funding to help advance initial laboratory discoveries at least to the point where private industry might be interested in partnering to help carry these findings through the rest of the FDA-required process. NCATS will take the NIH’s investment in basic research closer to helping people who suffer from disease.2
NIH funding to catalyze drug discovery is not a new idea. While Dr. Miller claims that the NIH has a poor track record of producing pharmaceuticals, he cites only one example dating back to 1963. According to a New England Journal of Medicine paper published just this past February, 153 new FDA-approved drugs, vaccines, or new indications for existing drugs were discovered through research carried out in public-sector research institutions over the past 40 years.
These drugs included 93 small molecule drugs, 36 biologic agents, 15 vaccines, eight in vivo diagnostic materials, and one over-the-counter drug. These are medicines that are now saving countless lives—the authors conclude that these drugs discovered by the public sector will have a disproportionately large therapeutic effect on human health.3 Several of these important medicines were delivered through Cooperative Research and Development Agreements and other mechanisms where government funding is leveraged with for-profit company investment. For example, many of the most effective drugs used by oncologists to treat cancer today originally arose from programs funded in whole or in part by the National Cancer Institute, the largest institute within the NIH.