Despite a 2012 directive from Congress to better include patients in the development of treatments for disease, the Food and Drug Administration (FDA) says that the key to a drug's approval is still scientific evidence.
A recent Bloomberg report noted that a drug to treat Duchenne's muscular dystrophy, Kyndrisa from BioMarin Pharmaceutical, failed to gain FDA approval in January 2016 despite pleas from patients because the FDA said it failed to provide clear scientific evidence that it helped patients. Duchenne's is a rare form of muscular dystrophy that affects 1 in 3,600 boys.
Another Duchenne's drug -- Translarna from PTC Therapeutics -- was also rejected by the FDA for failure to show that patients on the drug fared better than those who were given a placebo.
According to the Bloomberg report, drug companies sometimes skip randomized clinical trials for drugs to treat rare or particularly deadly diseases. However, it is these trials that the FDA depends on to approve or reject a drug.
“I would prefer seeing randomization very, very early” in the drug testing process, FDA's Center for Drug Evaluation and Research director Janet Woodcock told Bloomberg. “Even if there's a small, tiny effect, it may be meaningful to that patient population. If they can show there is definitely a small effect in a terrible disease, we will approve that drug.”
There have been drugs that have been approved in part due to patient advocacy groups. Addyi, a drug used to treat low female libido, was rejected twice before it was approved last year following a patient advocacy campaign that was funded in part by the drug's manufacturer, Sprout Pharmaceuticals.
The FDA is currently working with patient organizations on identifying key issues in drug development for muscular dystrophy. The Parent Project Muscular Dystrophy organization has suggested that the agency use different measures to determine a drug's benefit.
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