Despite low-quality testing, drugs designed to treat rare cancers—called orphan drugs—are increasingly being made available to patients. On Monday a Florida researcher told Reuters that there are concerns about the safety and efficacy of these drugs because they can come with a high number of serious side effects.
“The scientific validity of these types of studies is very, very limited,” Dr. Thomas J. George, a cancer researcher at the University of Florida, told Reuters Health. “You know less about the safety of the medicine and its quality.”
In a new study researchers looked at evidence evaluated by the FDA from 2004 to 2010 when considering the effectiveness of new cancer medications. The study found that when evaluating orphan drugs—defined as medications which treat diseases affecting fewer than 200,000 Americans—the FDA used a significantly smaller number of people in its studies. Orphan drug trials included 96 patients whereas 290 patients were evaluated in non-orphan drug trials.
“I do think that these studies are a valuable way to provide medication that otherwise wouldn’t be available to patients,” said George. “In the short term it’s a win-win for everybody, but in the long run we have to do due diligence to make sure the drugs are safe.”
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