Last Friday, I attended the funeral of a remarkable 13-year-old girl named Anna Tomalis. For the past three years, Anna had been battling terminal cancer and, more recently, trying to get the Food and Drug Administration to grant a “compassionate use” exemption so she could try an experimental cancer drug now being jointly developed by the pharmaceutical companies ARIAD and Merck. Unfortunately, FDA rarely grants exemptions. If too many exemptions are granted, it would become harder to enroll patients in clinical trials, where they have as much as a 50-50 chance of getting a placebo. Anna was too young and too sick to be admitted to any of the clinical trials, so that wasn’t at issue here. But, of course, the whole point of FDA is to keep individuals from making their own decisions about which drugs to take. So, eventually, after months of delay, FDA finally approved Anna’s exemption, but it came too late. She died just three weeks after beginning treatment — too little time for the drug to have worked.
I got to meet Anna and her mother Liz a few months ago, through a patient advocacy organization called the Abigail Alliance For Better Access To Developmental Drugs, with which CEI works occasionally. And, I continued to correspond with them both by e-mail ever since. Though I certainly did not know Anna very well, the service was quite moving. Her father, Ron, for example, explained that Anna realized all along that her chances of survival weren’t good. But, keeping a good attitude about the whole thing, Anna insisted that she be buried in a hot pink casket. Since no one actually makes a hot pink casket, her parents had to buy a non-descript one and take it to an auto body shop to have it painted pink.
Anna also tried to use her impending death in order to promote a change in the law that would make it easier for critically ill patients to get compassionate use exemptions. Even though she realized she wouldn’t last long enough to benefit personally from such a change, she visited Washington several times in order to advocate for such a policy. My op-ed on the subject appeared in this past weekend edition of The Wall Street Journal, and can be read here.