Eric Topol M.D., director of the Scripps Translational Science Institute and author of The Creative Destruction of Medicine, describes what’s next in the continuing saga of technology-based industry disruption as the great inflection of medicine.
While moderating a California Innovation discussion for expediting drug discovery and cures, and as a long-time Silicon Valley participant, I couldn’t help but notice parallels in medical discovery to the evolution of the Internet as a platform. It appears that a key milestone igniting Topol’s predicted future is emerging—the formation of a Health Innovation Highway.
The Health Innovation Highway has the potential to do for medicine what the Internet did for business. The widespread adoption of the Internet in the 1990s unleashed a flood of entrepreneurial activity by dramatically reducing the cost of innovating, launching, and growing a startup.
Today we are experiencing the equivalent of the pre-Internet 1980s in health care. Innovation is slow, expensive, and out of reach for most organizations. Almost everything from drug discovery to clinical trials are ad-hoc and recreated from the ground up for each new project; learning is not easily applied to the next innovation. It typically takes 15 years and more than $1.5 billion to develop a new drug in the U.S., and that doesn’t count the many projects which fail to ever pay off.
It’s no wonder that even promising new treatments can fall into a "Valley of Death" because of the high cost and uncertainty involved. One participant in the California roundtable observed, "With today’s hurdles, a drug like Tylenol would not make it to the market."
Recognizing that there has got to be a better way, The California Biotechnology Foundation and The California Endowment recently organized and hosted an innovation dinner in San Francisco at the Public Policy Institute of California with 22 diverse thought leaders ranging from senior representatives of the California governor’s office and a state senator to biotech CEOs, VCs, and research foundations.
The goal of this forum was to to explore creative solutions for expediting drug discovery and delivery as a way to make health happen for all Californians. "There is too much uncertainty, delay, and duplication in the current model," said Joseph Panetta, chairman of the CBF, and president and CEO of BIOCOM, a Southern California biotech association of 550 firms. "We need much greater collaboration among the financers, researchers, patient advocates, and regulators to create more effective and faster pathways."
The discussion with these thought leaders made one thing clear: there is a huge opportunity to develop a Health Innovation Highway that can serve as a common infrastructure to expedite development for drugs and other treatments.
Work on several elements of what could become the Health Innovation Highway has already begun. Here are a few of the steps being pursued that were shared by roundtable participants and from my observations of the industry:
Shared Clinical Testing Platforms: Dr. Laura Esserman of the University of California San Francisco indicated that precompetitive collaboration among researchers for clinical trials has potential to take 10 years off of the development process. Esserman is a principal investigator for the I-SPY 2 Trial, a public-private partnership and collaboration between the NIH Biomarker Consortium, the FDA, the National Cancer Institute, 20+ academic research centers, and others with a goal of reducing the cost, time and number of patients required to bring new drug therapies to breast cancer patients.
According to Paul Hastings, President and CEO of OncoMed Pharmaceuticals, a traditional early-stage clinical trial for a cancer treatment costs a company around $40,000 per patient, with a large portion of that cost devoted to developing baseline data separate from the drug being developed. Hastings says it’s difficult to get VC backing to test promising but unproven treatments when the costs are so substantial. This leaves many possibilities behind. With I-Spy 2, OncoMed has the potential to test a drug target more cost effectively and more quickly, enabling them to identify patient attributes in which a treatment works most effectively.
Leveraging the success of I-SPY 2 work, QuantumLeap Healthcare Collaborative is currently building shared informatics platforms for researchers to use in other disease areas such as Alzheimer’s and cardiovascular diseases. Similar efforts are underway to share other types of precompetitive data by Sage Bionetworks and Genetic Alliance. "Although precompetitive consortiums are new to life sciences, some industries have realized a 10-times return," says David Dilts, Ph.D, of the Knight Cancer Institute.
Disease-in-a-Dish Research: Sharing basic research, driving down the cost, and improving quality of genome sequencing and stem cells from skin cells (iPS) can exponentially accelerate economic viability. What is perhaps the crown jewel in state-led efforts is the California Institute for Regenerative Medicine (CIRM), a unique initiative for developing stem cell therapies, chaired by Jonathan Thomas, Ph.D. Scientists supported by CIRM recently discovered a new brain stem cell that could aid in therapies such as autism.
Topol and others have observed that mass medicine studies and animal models are imperfect predictors for how an individual will respond to a new drug. With stem cells, a doctor could test a potential treatment on copies of your own neurons made from stem cells with no need to perform brain surgery or expose you to risk. With disease-in-a-dish research, scientists can now study difficult illnesses such as bipolar disorder, ALS, and Parkinson’s on live human tissue to better understand root causes and develop viable treatments. Stem cell and genomic testing have the potential to also reduce years of trial and error (and life-threatening side effects) for patients seeking the right treatment "cocktail" customized for their genetics.
Precision Medicine through Common Patient Data Structures: John Wilbanks, a senior Kauffman Fellow, has long advocated for sharing scientific research and personal health data to detect disease patterns in big data. One example is through precision medicine as proposed by the National Academy of Science, in which diseases will be defined analogous to a Google map with layers of information to define diseases—symptoms, genome, epigenome (changes to gene expression caused by the environment), and other attributes. Although privacy remains an issue, Wilbanks and others suggest possible incentives for sharing personal information include laws to protect against discrimination based on health data, securing data from personal identification, and tax credits for contributors.
Disease Foundations Building Bridges: Some disease foundations are developing platforms to bridge the "Valley of Death" in-between academia and pharma as an industry resource. The Myelin Repair Foundation assembled its own translational lab staffed with experienced biopharma researchers to increase the odds of success for new MS treatments by ensuring academic discoveries are commercially robust and industry ready, an emphasis markedly different than funding traditional university or private company research.
Without short-term Wall Street pressures, disease nonprofits are not subject to shifting corporate research priorities in big pharma, enabling them to steadfastly pursue their mission during a critical but vulnerable early stage of R&D. In addition to partnering with academia and private industry as a resource, disease foundations can rally patients for clinical trials reducing time-to-market. In a prior article, I described how One Mind for Research is acting as a catalyst across the industry for cures to brain diseases.
Common Frameworks, Tools and Standards: At the federal level, efforts are also underway as DARPA (inventors of the Internet) is working on elements that could contribute to the Health Innovation Highway via their Living Foundries Project. The Living Foundries Program is working with Caltech, The J. Craig Venter Institute, MIT, and others to build a library of modularized parts analogous to LEGO pieces and an engineering framework for biology, with a goal of speeding the biological design-build-test cycle by 10 times. Such common frameworks are akin to technologies like TCP/IP, HTML, and web browsers that allow web apps to be built without redeveloping each underlying element.
The Health Innovation Highway Is a Collaborative Race for the Human Race
The Health Innovation Highway is not a zero sum game where winner takes all. Of course, whoever builds the ecosystem will generate jobs and tax revenue as the "next Silicon Valley." One could also envision disease hubs forming in distinct communities with disease experts and foundations leveraging this infrastructure.
There is much work to do and private industry and foundations cannot do this alone. As one roundtable attendee astutely noted, "Eisenhower built highways, not trucking companies."
What’s unique is if just one startup or pharmaceutical company on this highway—just one company—discovers a cure to a devastating disease like cancer, diabetes, or schizophrenia, everyone in the world wins, from quality of life for patients to reducing the economic burden on social services. The Health Innovation Highway is not a race between countries or states, it’s a collaborative race for the human race.
Who else is building infrastructure to support rapid biotech and medical innovation? Please share your thoughts with me.
To learn more:
The California Biotechnology Foundation
The California Endowment
—Adrian C. Ott is a corporate advisor, speaker, and executive facilitator who was retained to moderate the California Health Innovation Roundtable. During the 1990s, as leader of a corporate innovation program for a Fortune 100 technology company, she was recognized in the annual report for infusing the company with "new revenue streams, new technologies and new business models." Reach her @ExponentialEdge.