Developing the biomedical century: how to get the treatment from the lab to the patient.

Many expect that history will look back on this century as the biomedical century. New medicines and medical research are transforming our lives more than ever before. There is hardly a disease that has not been touched by modern drugs.

The possibilities for the future are even better. Genomics gives us new insights into the correlation between genes and the likelihood of developing certain diseases. The science of proteomics shows us how proteins interact inside the body to "turn on" complicated pathways that can cause illness. These and other new sciences might result in medical products capable of being individually tailored to treat specific patients with rare diseases, perhaps even to prevent those diseases from occurring in the first place.

We've made much scientific progress toward achieving these goals. But there's still one problem. In contrast to many other fields of applied science, we're not getting any better at the process of moving from the laboratory to the drugstore; that is, developing and refining scientific ideas into products and services that can be used by patients. The plain truth is that despite the dramatic advances and the new fields that have been developed in the last decade--genomics, proteomics, nanotechnology, and others--none of these developments have yet transformed current medical treatments.

In 2002, when researchers celebrated the success of the quest to sequence all of the human genome, the number of applications and approvals for truly new drugs at the Food and Drug Administration reached a 20-year low. Applications for new biologics (drugs based on human proteins such as hormones and enzymes) and medical devices are also down dramatically. While applications to the FDA have rebounded this year, they are not up to their highest levels of the 1990s, and they still don't reflect all the investments and advances in biomedical science over the last few decades.

The potential for achieving greater innovation in the development of medical products is certainly there. Spending on biomedical research and development is higher than ever. Right now, the National Institutes for Health has completed a five-year doubling of its budget, to more than $27 billion. Less well-known is that spending on research and development by private companies has also risen dramatically. R&D budgets of pharmaceutical companies have doubled since 1995--to more than $30 billion. Add in funding from private foundations, biotech companies, and academic institutions and the total comes close to $100 billion.

The real problem is that it continues to get more expensive and riskier to transform discoveries in basic science into useful treatments. If recent history is any guide, the vast majority of treatments that show promise in the lab will not ever make it to a human-trial phase. Of those products that are tested on people, less than one in five will result in a product application to the FDA. And those applications will come more than a decade after the drug is first developed. People who are suffering from diseases today can't wait all those years for fresh treatments to eventually make their way to the market. They need help now.

Just as the time it takes to bring a new drug to market has risen, so have the costs. According to the Tufts Center for the Study of Drug Development, it now takes more than $1 billion in private money--in addition to the substantial NIH contributions to laying the scientific foundations--to develop a drug. All in all, the cost of developing a new drug has doubled over the past decade.