They have a tough job, those government doctors, scientists, and bureaucrats who are charged with assessing the safety and effectiveness of proposed new medical products. As you know, they rely largely on studies presented by the applicants.
The U.S. Food and Drug Administration has the power to not approve a new drug or product or even pull it off the market. Right now it is considering limiting or, pulling GlaxoSmithKline’s diabetes drug, Avandia, because of newly discovered data that it may have caused heart attack in some patients – data mysteriously not shown in GSK’s own studies. If the drug is pulled it will cost GSK billions of dollars in lost revenue but, from the FDA’s point-of-view, it will be protecting the public. And, after all, there are safer diabetes drugs on the market as alternatives.
The FDA also is careful when considering approving new drugs. Sometimes applications come from big companies, GSK among them, and other times from small, biotech companies, that have devoted years and hundreds of millions of investors’ dollars to develop a useful, safe, effective new drug.
When it comes to the many forms of cancer, in this age of personalized medicine, increasingly the proposed new drug may be a “niche” product. It may help just a small number of people with a rare condition. And, with an aggressive cancer, it may not cure the cancer but only extend life by weeks or months. Plus, if a drug comes to market, it will be expensive. Biotech drugs are hard to develop and tricky to manufacture.
Over the past few years I have wondered whether the FDA was playing fair as it evaluated these niche drugs from smaller companies that have few resources to challenge or appeal action by the FDA. One such “little guy” is a Seattle company called CTI. A few months ago an FDA panel voted against approving a new drug from CTI called Pixantrone, a drug for an unmet need of treating patients with relapsed or aggressive Non-Hodgkin’s lymphoma. The panel, guided by Dr. Richard Pazdur, effectively the “Czar” of cancer drugs in America, said the drug’s study did not meet the agreed upon endpoint: that it wasn’t effective.