The Golden Age of Medical Innovation: Critics Grouse about a Sluggish Drug Pipeline, but They Are Looking in the Wrong Place. John Calfee Shows That Innovation in Drugs and Medical Devices in the U.S. Is Rampant. New Treatments Are Fighting Breast Cancer, Macular Degeneration, Rheumatoid Arthritis, and Many More Diseases. Why the Success? (Health Innovation Outlook 2007)

Article excerpt

LAST DECEMBER, THE editors of Science magazine--the widely respected journal of the American Association for the Advancement of Science--selected ten "breakthroughs" of the year.

Number one was the solution to the "Poincare Conjecture," a topological puzzle that had confounded mathematicians for a century. Number four was the discovery of a 375-million-year-old fossil that filled an evolutionary gap between fish and birds. But it was breakthrough number six that got my attention--"a ray of hope for macular degeneration patients." It related the stunning clinical-trial results for ranibizumab, a drug sold under the brand name Lucentis by Genentech, its developer and manufacturer.

Lucentis is a monoclonal antibody--made, using biotech methods, from genetically engineered bacteria--that attacks a protein responsible for the leading cause of blindness in seniors. Age-related macular degeneration, or AMD, affects a part of the retina and causes blind spots, which expand over time, in the center of the field of vision. It is a disease that had previously defied all attempts even at delaying the inevitable decline toward blindness. But with Lucentis, the eyesight of about 95 percent of AMD patients either improved or stopped getting worse--a stunning achievement.

Biotechnology has lately provided many other breakthroughs. In January, an article in The Lancet, a British medical journal, described the latest striking results for another Genentech drug, Herceptin, which treats an aggressive form of breast cancer that affects about 50,000 women a year. The cancer is characterized by an overabundance of a protein called human epithelial growth factor receptor-2 (HER2), which stimulates the growth of tumors. In 2005, research published in The New England Journal of Medicine found that, when administered after surgery, Herceptin reduced the odds of a recurrence by half. An accompanying editorial concluded that the key result "suggests a dramatic and perhaps permanent perturbation of the natural history of the disease, maybe even a cure," and that "our care of patients with HER2-positive breast cancer must change today." The new Lancet study went even further, documenting mortality declines after only two years of use, an unheard-of result in treating breast cancer. Other biotech treatments approved recently include Enbrel, for psoriasis, a painful skin condition; Remicade, for Crohn's disease, an inflammatory digestive-tract disorder; Rituxan, for rheumatoid arthritis; and Avastin, for several different cancers.

Enbrel was developed by Immunex, a Seattle company that was purchased in 2001 by Amgen, the world's largest biotech firm. Remicade is a product of Centocor, the biomedical firm that pioneered monoclonal antibody technology. And Rituxan and Avastin are from Genentech, one of the early biotech firms that is now seeing a boom in revenues, up 40 percent in 2006 to $9 billion.

The remarkable treatments these firms have developed seem at odds with the widespread notion that medical innovation has all but come to a halt. A recent report by the Government Accountability Office, for example, has warned, in the words of a popular website that covers drug research news, "that the pharmaceutical industry is not producing enough new drugs despite spending more on research and development." In a similar vein, Democratic Representative Henry Waxman, the new chairman of the House Committee on Government Reform, stated, "Many aspects of the drug development system need to be examined to determine how to encourage research that focuses on breakthrough treatments rather than drug industry profits."

Yet breakthroughs are precisely what are being achieved. Why aren't they more widely recognized? One reason is that none of the treatments I have just described would appear on a list of innovative new drug approvals over the past few years. The results all came from clinical trials involving drugs that had already been approved for something else--often a different stage of cancer, sometimes a different type of cancer, and occasionally a different illness altogether. …