The Food and Drug Administration is trying to make it easier for doctors to get approval of using experimental drugs on patients with life-threatening conditions.
The application physicians will now have to submit for a "compassionate use" drug consists of 11 questions, rather than 26. The agency estimates that doctors will be able to fill out the form in 45 minutes, on average.
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The simpler application comes in the context of the "right to try" movement that has advocated to give terminally ill or other seriously ill patients easier access to experimental drugs aimed at either curing a disease or easing pain. National Public Radio reports that 20 states currently have such laws in place.
The FDA approves almost every request it has received, rejecting only 14 of the more than 1,400 submitted last year.
But in addition to gaining approval from the FDA, doctors must get permission from the company that makes the drug. Some drugmakers may not feel the drug is ready to be used on people or that it is not appropriate for the specific case. They worry about negative publicity or even lawsuits in the event of unexpected side effects.
The FDA also recently announced that drug manufacturers will not be able to profit off the sale of experimental drugs for compassionate use. They will only be allowed to charge patients the cost of manufacturing them. Private insurers and government health care programs cannot be forced to pay for them, either.
The agency is under a lot of pressure from patients' groups to approve a new drug, Eteplirsen by Cambridge, Mass.-based Sarepta Therapeutics Inc., to treat muscle wasting. In light of a recent study conducted by an agency advisory board, which determined that the medicine is not effective, the drug is likely to be rejected for mainstream use. In response to the study, the FDA announced it would delay its decision.
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