By Keith L. Black, MD

Researchers and clinicians must work together to bring discoveries from the laboratory bench to the patient's bedside. Clinicians can then provide valuable feedback to refine treatments and suggest new pathways for researchers to pursue. This relationship between the lab and clinic, however, requires support in the form of funding. Without adequate funding, discoveries that may save many lives languish in the lab, their clinical potential unexplored. Increased funding from both public and private sources is required to offset the high risk, long-term and capital intensive nature of such ventures.

Even with U.S. drug sales in 2004 at $200 billion and the pharmaceutical industry ranking first among major industries in profitability since the early 1980s, only slipping to third in the Fortune 500 list of 47 industries in 2003, there is a gap between industry funding, focused on producing usable drugs, and National Institutes of Health funding, focused on basic science.

A series of laws in the 1980s, especially the Bayh-Dole Act, launched the current pharmaceutical boom. The law allowed universities and small businesses to patent discoveries emanating from NIH-sponsored research and then grant exclusive licenses to drug companies. Before that, publicly funded research discoveries were in the public domain. Under the new laws, the NIH itself was also allowed to make deals with drug companies to translate NIH discoveries into useful drugs and treatments.

This change led to a boom in biotechnology and big pharma. It also meant that the big pharmaceutical companies did less of their own basic research. At least one third of the drugs marketed by major drug companies are now licensed from universities or small biotech companies, which are often started by researchers from universities and hospitals. Furthermore, a majority of the most innovative drugs have been developed via this route, and not by big pharma research.

In the 1990s, another series of laws extended monopoly rights for brand-name drugs, which proved to be another boon for the pharmaceutical companies. The effective patent life of a brand-name drug grew from eight years in 1980 to fourteen years in 2000. Those extra six years of patent life provided billions of dollars in sales for major drugs, such as Zoloft®, Lipitor® and Celebrex®.

With the changes in the laws, profits for big pharma grew. In 2002, the profits from the ten drug companies in the Fortune 500 ($35.9 billion) were more than the profits for all the other 490 businesses combined ($33.7 billion). Research and development spending by the companies, however, remained at between 11 and 14 percent of sales, with the companies spending far more (in 1990 for example) 36 percent of sales revenue, on "marketing and administration."

On the other side of the equation, NIH funding now exceeds $13 billion a year, still mostly focused on basic research. The need for greater support for translational research is acute because researchers are on the cusp of developing lifesaving medical treatments as they pursue the study of molecular pathways (the next step in the human genome project), fusing diagnostics with therapeutics, combining biotechnology with nanotechnology, and furthering research into cell and gene therapy, with the use of adult stem cells to treat disease.

Many, if not most of the areas in which medicine is on the brink of new therapeutic developments are now conducted by researchers in academia. In order to quickly translate research discoveries into clinically effective treatments, increased translational funding is required. Unfortunately, the funding gap between fundamental research and clinical application is shrinking. In the past, drug discovery was based on serendipity or trial-and-error, while today it is crucially dependent on basic knowledge of genes, proteins and associated biochemical pathways. Basic research is now at the core of the development of new drugs.

Besides funding, researchers and clinicians must routinely and constantly communicate. Without such communication, the full potential of discoveries remains unfulfilled and the vital feedback from clinicians back to researchers to refine drugs is missing.