Washington – Greater numbers of seriously ill patients could get experimental medicines under proposed federal guidelines released Monday that also would clarify when drug companies could charge for the medicines.
The revised guidelines lay out in greater detail when unapproved drugs would be available to patients with no other options to treat a life-threatening or serious disease or condition, the Food and Drug Administration said.
The FDA would have to be satisfied that the potential benefit of the treatment justifies its potential risks.
If adopted, the guidelines would spell out that drugs could be available during all stages of drug development, including during initial, phase-one testing. During that step, experimental drugs are tested in as few as 20 people.
Just 10 percent to 15 percent of phase-one drugs ultimately win FDA approval.
Since the 1970s, the FDA has made experimental drugs available to either individuals or large groups of patients under compassionate-use programs.
The revised guidelines, subject to comment for 90 days, would further describe all the circumstances in which access would be allowed, including to small groups of patients.
