Innovation at a Crossroads
Can we discover? This basic question drives thousands of researchers in the public and private sectors pursuing the discovery of newer, better medicines.
Today, at the beginning of the 21st century, pharmaceutical research has come to a critical juncture in the answer to this question.
In this new century, researchers are on the cusp of a new era in pharmaceutical discovery. Our knowledge of the genome and molecular workings of the body is opening up a new world of opportunities that was closed to us only a few years ago. In addition, powerful new research tools like pharmacogenomics, high-throughput screening and combinatorial chemistry are making drug discovery more promising and challenging than ever before.
Even as researchers are advancing into these new frontiers of science, they are tackling more complex diseases than ever before and the regulatory process is becoming more intensive.
A growing number of experts are pointing to the important implications that this crossroads in innovation holds for patients, for research, and for public policy.
"We're at a stage of scientific progress when a large range of new technologies such as genomics and proteomics are now becoming widely used in the development of new treatments, and these new sciences just might bring about new breakthroughs. Many new drugs currently in clinical trials are no longer scattershot one-size-fits-all treatments, but carefully targeted to be highly effective given the molecular features of a disease in a particular type of patient. These same new sciences also hold promise for making the process for developing new medical technologies faster and more predictable."
- Dr. Mark McClellan, Center for Medicare and Medicaid Services Administrator, September 25, 2003
"[M]edicine is poised for a great leap forward. For all our amazing advances in the last 50 years, we are still working with the tools of the first pharmaceutical revolution. That is, we are still mostly using advanced chemistry to treat disease symptoms. In the new age we are now entering, we will increasingly use advanced biology to actually cure or even prevent disease from occurring. The fruits of genomics and other new disciplines in biomedicine will clearly take some time, longer than we first thought, to transform therapeutics. But that transformation will come if we do not interdict it with short-sighted controls."
- Sidney Taurel, Chairman, President and Chief Executive Officer, Eli Lilly and Company
"Medical science stands at the threshold of research advances not even imagined a decade ago."
- HHS Secretary Tommy Thompson, January 19, 2001
Advancing Science to a New Era in Medicine
A number of emerging fields are changing the way that new treatments are researched and the kinds of breakthroughs being pursued. Biotechnology advances in genomics and proteomics are helping identify new treatment targets. Combinatorial chemistry and high throughput screening make it possible to rapidly create and test many thousands of compounds for desirable properties. Advances in therapeutic "biologics" such as monoclonal antibodies and the emerging field of gene therapy are expanding the definition of what a "medicine" is. And the new field of "personalized medicine," which uses molecular analysis of individuals and their diseases to determine which treatment approaches are likely to work best for each group, holds potential to change pharmaceutical research as well as clinical practice. (See "Next Wave of Innovation" for more detailed descriptions of new areas of research and technologies.)
As powerful and exciting as these new tools are - and despite the impact they are likely to have on medicine - their benefits are not immediate. In the short term, they add complexity and expense to the research enterprise.
Targeting More Complex Diseases
Today, scientists are increasingly confronting more complex degenerative and chronic diseases (such as Alzheimer's, cancer, and HIV/AIDS) that pose new challenges. Complex diseases often require much more basic research to identify novel treatment targets and results of these trials often prove more difficult to evaluate. An analysis of Phase III clinical trials by disease type and procedures shows a dramatic increase in highly complex disease types (as measured by procedural complexity) in just the past few years, from 28% of overall projects in 1995 to 49% in 2000.
Meeting More Demanding Regulatory Requirements
An increasingly rigorous regulatory environment has emerged over the last 25 years. More stringent requirements call for greater understanding of how each new drug works in the body and also testing in more patient subpopulations. These changes improve safety, but require more time and investment for each new medicine.
Facing Higher Costs of Discovery
As scientific and regulatory requirements have increased, the cost of developing a new medicine has risen dramatically, going from $138 million in 1975 to $318 million in 1987 to $1.3 billion in 2006 (2005 dollars). To finance this longer, larger, and more expensive process, PhRMA member companies have multiplied their investment in R&D from $2 billion in 1980 to $44.5 billion in 2007(the biopharmaceutical industry as a whole invested over $58.8 billion in 2006).
Yes. The tremendous push being made into new frontiers of science is beginning to bear fruit. Earlier this year, for example, FDA approved the first anti-cancer medicine based on angiogenesis inhibition, which kills tumors by restricting their blood supply. It took three decades of research in this field before the first new medicine emerged. The number of truly novel drugs approved by FDA in 2003 was up 20% from the year before, and the number of investigational applications for novel therapies also increased.
Pharmaceutical researchers are on the verge of a new era in medicine that holds great promise for patients and our healthcare system. We can discover, if we make a sustained, substantial investment in research and development, and create a public policy environment that supports continued innovation.
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