If we learn nothing else from BP’s recent unpleasantness, it’s that being able to identify an obvious problem (like when oil is gushing uncontrolled into the Gulf of Mexico) is one thing. Identifying that there is a potential problem is tougher. And toughest of all is designing a solution that addresses a need early in the curve.
Case in point, Alzheimer’s Disease. As Gina Kolata writes in today’s New York Times, “The failure of a promising Alzheimer’s drug in clinical trials highlights the gap between diagnosis — where real progress has recently been made — and treatment of the disease.”
Alzheimer’s Disease is a healthcare oil spill of draconian proportion.
Recent significant steps forward in early diagnosis of the disease are important. And frustrating -- because there is still precious little that can be done when this devastating condition is identified either late in the game or in its nascent stages.
To say that the science is “hard” (while true) is not particularly helpful. What needs to be addressed are the twin issues of drug development and regulatory science. Both are lagging. Biomarkers notwithstanding, more needs to be done. We need better tools. Too many programs (almost 50%) are failing in late Phase III. Too many programs are mired in regulatory treacle. The economics are unsustainable from a corporate R&D standpoint and the impact of Alzheimer’s Disease on patients, their families and American healthcare economics is devastating.
Better, more current and predictable scientific research and standards must be developed and devoted to streamlining the critical path. Investment in basic research is not enough. Specifically new development tools are needed to improve the predictability of the drug development cycle and to lower the cost of research by helping industry identify product failures earlier in the clinical trials process.
25 years ago, the success rate for a new drug used was about 14%. Today, a new medicinal compound entering Phase 1 testing—often after more than a decade of preclinical screening and evaluation—is estimated to have only an 8% chance of reaching the market. For very innovative and unproven technologies, the probability of an individual product’s success is even lower. We have got to work together to turn that around.
When Thomas Edison was asked why he was so successful he responded, “Because I fail so much faster than everyone else.” Consider the implications if FDA could help companies to fail faster. Using the lower end of the Tufts drug development number ($802 million):
* A 10% improvement in predicting failure before clinical trials could save $100 million in development costs.
* Shifting 5% of clinical failures from Phase 3 to Phase 1 reduces out of pocket costs by $15-$20 million.
* Shifting of failures from Phase 2 to Phase 1 would reduce out of pocket costs by $12-$21 million.
Investment in basic research is not enough. Specifically new development tools are needed to improve the predictability of the drug development cycle.
For all that modern science has to offer, developing new treatments is still very much an art—in which hunches, intuition, and luck play a critical role. The odds are long. For medicine that is affordable and innovative, we need more well-understood science and we need regulatory predictability. And that’s precisely the mission of the FDA’s still moribund Reagan-Udall Foundation.
To quote the late Senator Ted Kennedy, the Reagan-Udall Foundation “will make new research tools and techniques available to the entire research community, shortening the time it takes to develop new drugs and reducing costs for patients.”
Shortly before his death, I had the privilege of a private meeting with Nobel Laureate Joshua Lederberg. The topic of conversation was the future of the FDA and the agency’s Critical Path initiative. We talked about the state of applied research and “the texture” of the agency, the prioritization of development science, biomarkers and a host of other future-oriented issues. He talked. I took a lot of notes. At the end of the meeting he put everything into perspective in a single
I hope so and so should we all. Innovation = Hope.
