Program Connects Patients With Experimental Drugs: FDA Program Gives Seriously Ill People Path to Obtain Drugs Still in Testing
Nisha Gupta was working as a medical resident at a Syracuse, N.Y., hospital when she accidentally poked her finger with a needle while caring for a patient. She remembers feeling a tiny prick at the time. But the incident triggered a two-decade-long struggle against an incurable disease that involved two liver transplants, a near brush with death and a last-ditch gamble with a drug still in the experimental stage.
Dr. Gupta contracted hepatitis C, a disease whose symptoms typically take years to develop but then become debilitating as the virus attacks the liver. For Dr. Gupta, fatigue and itching became so severe she would need several ice packs to numb her skin. She developed a brain disorder called encephalopathy in 2008 as her liver started failing and could no longer filter toxins from the blood. A year later, she was diagnosed with liver cancer. Treatments with standard antiviral drugs stopped working.
Today, Dr. Gupta, 51 years old, has been clear of hepatitis-C symptoms for more than a year. She attributes her remarkable recovery to the experimental drug she eventually took, daclatasvir, which is still in testing by its manufacturer, Bristol-Myers Squibb Co. BMY +0.68% The drug hasn’t yet been approved for public use by the Food and Drug Administration. Instead, Dr. Gupta received daclatasvir through a little-known FDA program called “compassionate use” that helps patients with serious or life-threatening conditions gain access to experimental drugs if they have exhausted all other options.
Last year, nearly 1,200 patients received treatment with experimental drugs through the compassionate-use program for conditions including hepatitis C, cancer and rare diseases like cystic fibrosis, the FDA says. That is up from about 1,000 patients in 2010, the first year the agency compiled data on the program. The FDA says it has been trying to increase participation, including by helping to set up a Web-based seminar that trains doctors how to make use of the program.