How to reboot the bioscience revolution

By David Ewing Duncan, contributor


FORTUNE -- On a balmy summer day 10 years ago, President Bill Clinton announced an accomplishment that was likened to landing men on the moon: The sequencing of a nearly complete human genome. Flanked in the White House by the two scientists mostly responsible for it, Francis Collins and Craig Venter, the president and other speakers brashly opined that new drugs and treatments would soon flow from this historic achievement.

Ten years later, the public and private sectors have lavished nearly $1 trillion on life science research and development -- double what was spent in the 1990s. The expected cornucopia of new drugs and treatments, however, has not yet arrived. Instead, the number of new drugs approved by the U.S. Food and Drug Administration has actually plummeted, from a peak of 53 new drugs approved in 1996 to an average of 21 a year between 2005 and 2009. And the FDA has approved only a handful of genetic markers associated with disease out of thousands discovered.

One reason is that human biology has turned out to be far more complex than most people imagined. But this isn't the whole story. Another major cause of the delay comes from a failure of the biomedical establishment, and of society, to handle the wave of new science bought by that $1 trillion. This includes not only genetics, but also everything from neuroscience and nanotechnology to the impact of the environment on our DNA.

This "new biology" is nascent, and much of it is years away from making a difference in our lives. Nor has most of it been properly studied to see which parts will actually enhance health or reduce health-care costs -- or who will get access to it in the U.S. and globally.

Personalized health care

Yet the promise is there for a fundamentally different approach to health care based on 1) predicting a person's health future using their genes and other biomarkers, plus traditional diagnostic tests and family history; 2) prevention strategies designed to keep people healthy rather than waiting until they get sick; and 3) a much stronger emphasis on personalized and targeted healthcare and treatments.

The new science is also poised to enable people who want to take more responsibility for their own health and wellness by providing them with more and better information about their health.

This potential biotransformation, however, is running up against a wall of tradition and outmoded infrastructure in science, medicine, government, commerce, and law. These fields are failing to show the same creativity and innovation in adapting to the new science that went into the discoveries themselves.

For example, the legal system in the US has yet to decide if a gene can be patented. This is despite more than 30 years of legal wrangling that has tried to fit DNA into legal definitions that probably don't make sense. Our inability to find a creative solution for how a company can own or license the rights to develop a DNA test - which costs money - is one reason why investors have not paid for human trials to validate thousands of new markers. Nor has the FDA figured out how to regulate these markers, though they are now trying.

The life science community also shares some of the blame, by having a system that has become so specialized and sub-specialized that the various silos even with similar fields seldom talk to each other, much less to physicians and to patients. This has made it difficult for biomedicine to integrate individual discoveries into a holistic understanding of the human body.

A coordinated effort is needed

Lately, the National Institutes of Health has initiated programs to emphasize the translation of discoveries more quickly into applications. But so far it has committed a mere 2% of its $40 billion budget to these programs. Patient advocacy groups like PatientsLikeMe and the Michael J. Fox Foundation are also trying to fill in the gaps to get to cures faster, but the combined budgets of all nonprofits and private institutes last year was less than $2 billion.

Much more is needed, which is why a momentum has been building among many leaders in the life sciences, patient advocacy and wellness communities to call for a coordinated national effort to accelerate the translation of discoveries into applications -- with proper funding and a comprehensive plan at least on the scale of the original Human Genome Project.

One articulation of this effort is a short document called The Personalized Health Manifesto, which will be launched this week in Washington, D.C. The Manifesto was developed with funding from the Ewing Marion Kauffman Foundation, and with the input of 35 experts that represent divergent points of view - scientists, physicians, entrepreneurs, investors, patient advocates, ethicists, lawyers, policymakers, and journalists.

Transforming a system this entrenched will be thorny, although the good news is that many in biomedicine understand the urgent need to change. For the sake of our health -- and to get our money's worth for the trillions we will spend on health R&D -- let's hope their effort succeeds.

David Ewing Duncan is the author of the Personalized Health Manifesto. He is a science and biotech journalist and the Director of UC Berkley's Center for Life Science Policy. To top of page