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.
Rare and Neglected Diseases
Another reason why the proposed NCATS is so timely is found in rare and neglected diseases. Here, the market opportunity is often too small to entice for-profit company investment, obligating the public sector to fill the void. The NIH’s Office of Rare Diseases Research estimates that approximately 8% of the U.S. population (25–30 million Americans) suffers from a rare disease. Historically, persons suffering from rare diseases, including many hereditary disorders affecting children, have probably benefited the most from government-funded drug development.
Thanks to the Orphan Drug Act, a 1983 law mandating the FDA to fast-track approval for rare disease therapeutics, the regulatory path exists to aid this objective. Since the act was passed, more than 325 orphan-designated products received marketing approval, a big improvement over the fewer than 10 that reached the market in the previous decade.4
With NCATS, even more progress could be made. If the NIH, through NCATS, funds the early stages of discovery and development of candidate therapeutics for rare diseases, chances are much greater that an industry sponsor will come forward to carry the ball across the FDA-approval goal line.
Plenty of other diseases are largely neglected by the pharmaceutical industry for reasons other than market size, leaving the burden to the public to find solutions. Take, for example, traumatic brain injury (TBI) and post-traumatic stress disorder (PTSD), leading causes of death and long-term disability among military personnel serving in combat. Few (if any) large pharmaceutical companies are currently developing countermeasures specifically for TBI or PTSD, and the handful of small companies working toward therapeutics for these disorders are mostly funded not by investors but by the government—namely the U.S. Department of Defense.