ONE OF the great challenges of our age is to connect the astounding advances in biology with medicines that can detect and fight disease. The success of the Human Genome Project and the explosion of data flowing from genomics and other disciplines have offered a look as never before at the basic building blocks of life. But the data explosion has raced far ahead of new diagnostics and drugs to improve human health.
Now, the National Institutes of Health, under Director Francis S. Collins, has launched an ambitious and unconventional project aimed at bridging the gap with respect to four diseases: Alzheimer’s, type 2 diabetes, rheumatoid arthritis and lupus. Dr. Collins has corralled 10 drug companies, the federal government and several nonprofit organizations and research foundations to collaborate on what they could not do alone — find promising targets in biology for new drug therapies. The pilot projects, which Dr. Collins announcedlast week, will bring scientists from rival companies and organizations to work side by side, navigating through the mountains of data in hopes of finding clues and signposts to new therapies and to do it sooner and cheaper than if companies were working alone. The project will make all the discoveries public, similar to the open-source movement in computing.
This is a major change from the model that has dominated drug development, in which pharmaceutical companies each worked alone in walled-off research divisions, hoping to find a blockbuster drug that would lead to lucrative profits. But the existing model entails enormous risks. Currently, taking a drug candidate from early discovery through Food and Drug Administration approval takes a decade and has a 95 percent failure rate. The cost of developing one drug can exceed $1 billion. No wonder that companies grew cautious in how they placed their bets, some of which even so turned out to be duds.
The five-year, $230 million Accelerating Medicines Partnership suggests a different approach in the early stage of research. Scientists will be free to explore without the nagging worry of corporate return on investment hanging over their heads. Among other things, they will be looking for biomarkers, those pathways in the data that could show a specific gene or protein can be altered by a drug to effectively fight disease. Should the effort succeed, it might be wise to consider other realms and diseases that could use help, such as the dwindling pipeline of antibiotics.
Once the collaborative effort has published the data in the open, competition will resume among the drug companies to develop new therapies. The FDA’s three-phase process of trials will remain in place to ensure the safety and efficacy of new products, although in some cases the new data might allow a shorter testing period. Dr. Collins is confronting a complex set of scientific and economic problems. We hope his approach succeeds in translating the surge of scientific data into concrete advances for human health.