March 09, 2006
The Biotech Conundrum
I'm off this week to speak at the BioAgenda conference, an annual gathering of biotechnology industry supporters. I'm on a panel debating drug pricing and pharmaceutical innovation. Here's one of the points I plan to make:
The biotechnology revolution is a major pillar of America’s claim to global high-tech preeminence. Its medical and economic promise has enthralled two successive generations of biologists and medical entrepreneurs, so much so that today the Biotechnology Industry Organization (BIO) claims nearly 2,000 member companies and most cities and towns have pinned their economic futures on grabbing a piece of what is presumed to be an ever expanding biotech pie.
But has this revolution paid off for investors, communities, scientists, and patients? Beyond the early big winners who picked the low-hanging fruit of the biotechnology revolution – companies like Amgen, Genentech and Genzyme – have the thousands of companies populating this industry filled the therapeutics pipeline with promising new biotech drugs that will revolutionize the practice of medicine?
This week’s Food and Drug Administration hearing on a new treatment for multiple sclerosis demonstrated both the promise and the perils facing these biotech firms. The 12-member paneled voted unanimously to let Biogen/Elan’s Tysabri back on the market despite rare occurrences of a fatal brain disease. At the same time, it voted just 7-5 to make it a drug of first choice for “some patients,” reflecting the sad truth that this monoclonal antibody is far from a slam dunk in treating the progressive, neurological disorder.
Tysabri is a symptom of the general disease affecting the biotech industry. FDA data reveals that the number of new biotech products has fallen from more than 30 a year in the early 1990s to less than 15 per year in 2003, and many of those that come on the market have only marginal effects on the medical conditions they seek to treat.
This trend bodes ill for both patients and biotech investors. The vast majority of the 2,000 firms that belong to BIO have no products on the market, minimal sales and a long string of investment-driven losses to show their investors. Not for nothing is this field known as the rich man’s lottery.
How about patients, like the more than a dozen multiple sclerosis patients who showed up for the Tysabri hearing? They hear the hype. They have the hope. And they suffer. Only they don’t suffer in silence. Often organized into patient advocacy groups funded by industry, they’re more than willing to raise their voices in support of even marginal new drugs.
Why has the promise of the biotech revolution not been fulfilled? There are many contributing factors. For instance, the low-hanging fruit has been picked. There are only so many diseases where one protein is missing and replacing it leads to a cure. Then there’s the corporate focus on “me-too” drugs, which has even infected successful biotech firms. Look at Amgen, whose biggest product in recent years is Aranesp, a follow-on drug to its blockbuster Epogen, which treats anemia.
But most significantly, the most pressing medical problems today (Alzheimer’s; obesity; cancer; chronic degenerative diseases like Parkinson’s, rheumatoid arthritis, etc.) result from either social causes or a biological cascade of events. There is no single triggering factor. Therefore, they cannot be easily “cured” by a single drug or biologic intervention.
The sad truth is that major innovations in medicines are rare, and usually result from intense collaboration over many years among dedicated scientists and teams of scientists focused on understanding and curing a disease. Yet today’s innovation system encourages atomization of scientists. They're encouraged to patent their scientific insights and pursue proprietary products through start-up biotech firms.
The future of innovation in biotechnology and biomedicine will require greater resources and greater collaboration than ever before. It’s not clear to me that atomized biotech firms, each working on their own intellectual property in the hope that it will turn out to be a financial home run, is the best way to get the job done.