The Wall Street Journal ran an interesting article yesterday about Nisha Gupta, a medical resident who accidentally poked herself with a needle while treating a patient. Unfortunately, that incident infected Dr. Gupta with hepatitis C and led her on a harrowing multi-decade path of liver transplantation, encephalopathy, and cancer. "We were all stressed out. I was going to die soon and I was prepared," she remembers.
That was her prognosis then, but Dr. Gupta is alive today, thanks to a drug that isn't commercially available. Dr. Gupta, through her physician, contacted Bristol-Myers Squibb to acquire compassionate use--really "expanded access"--doses of BMS's daclatasvir, while daclatasvir was still in early clinical trials. The BMS drug worked miracles and there is no longer a trace of the hepatitis C virus in her body.
Interestingly, Dr. Gupta had first contacted Merck and Vertex to obtain expanded access doses of boceprevir and telaprevir, but both companies were worried that their unapproved drugs would interact with the other drugs the woman was taking. "As a company, and a physician, we were taught not to do harm. We thought we would do harm," Vertex's chief medical director, Dr. Robert Kauffman explained.
We have been told for decades that without the FDA to protect us, drug companies would sell us all manner of dangerous and ineffective drugs. Yet here is a gravely ill patient begging two drug companies to let her try their hepatitis C drugs before she dies, and they refrain. Does that make sense? Yes it does. Drug companies are run by people, often physicians, who want to help patients, not hurt them. Executives and employees alike hold their heads high when they consider their progress in the war against disease. Drug companies make money when they successfully treat medical conditions and they risk going out of business if they don't, or even worse, if they hurt patients. Just look at the Vioxx fiasco, the army of lawyers that took on cases, and Merck's near brush with corporate death. Vioxx was a misstep that Merck hopes to never repeat and Merck didn't need a government agency, such as the FDA, to state the obvious.
Anyone who thinks that established, reputable pharmaceutical companies would foist dangerous products on the American public absent the FDA hasn't studied the situation very carefully. Pharmaceutical companies clearly understand the situation. It's time policy makers joined them.