Skip to content
Subscriber Only

Tragic Patient Stories Don't Trump Weak Data, Official Says

  • Robust clinical trials needed even as patient focus grows
  • Drugs for Duchenne muscular dystrophy struggle with data
Updated on

Heartrending anecdotes of sick patients in need can’t overcome the shortcomings of weak drug trial data, said one of the U.S.’s top drug regulators, as outside advocacy groups seek a larger role in the drug approval process.

The Food and Drug Administration has worked to better include patients in the development of treatments after a 2012 directive from Congress. While patient support can help a drug’s chances, the key is having evidence the FDA can trust, said Janet Woodcock, director of the agency’s Center for Drug Evaluation and Research. That includes randomized clinical trials where patients receiving an experimental drug are compared with others who don’t -- a step some drugmakers have skipped, especially when the disease is rare or particularly deadly.