Twelve-year-old Anna Tomalis was diagnosed with a rare form of liver cancer, and she waited months for the FDA to grant a “compassionate use” exemption for her to take an experimental drug. This video discusses Anna’s plight and the problems dying patients experience when seeking access to unapproved medicines.
UPDATE: On Friday, August 15, 2008, Anna Tomalis died at home, surrounded by her family. Although there was no way to know whether the experimental treatment would be successful even if Anna had gotten permission right away, the tumors spread aggressively while she waited for FDA’s green light. In the end, the compassionate use exemption was too little, too late.