For critically ill patients, experimental or investigational drugs, not yet approved, may hold out the best hope for their desperate condition.
Since the 1970s, patients have been able to access experimental drugs outside of a clinical trial, called “compassionate use,” but the rules have been informal and confusing.
Now the Food and Drug Administration (FDA) is trying to make experimental drugs more broadly available.
New rules were posted on the agency’s Web site Wednesday that clarify how patients can access not-yet-approved drugs outside of a clinical trial.
The rules go into effect in mid-October.
Previously if someone wanted access to a drug, not yet approved, they could agree to participate in a study of a drug, however, not all patients qualified for such trials, reports the Wall Street Journal.
Under the newly clarified rules, drug companies will be allowed to charge patients for experimental treatments and it should become easier to obtain FDA approval to have access to experimental drugs outside of a clinical trial or during the late-stage Phase I of a trial.
In order to receive investigational drugs, the patient must have a life-threatening condition for which there is no comparable drug alternative. The companies may seek FDA approval to charge for the drug, though they are restricted from making a profit.
Formalizing the rules, should give more people access to unapproved medications.
"With these initiatives, patients will have the information they need to help them decide whether to seek investigational products," said FDA Commissioner Margaret A. Hamburg.
Janet Woodcock and Lynda Dee, from the FDA’s Antiviral Drugs Advisory Committee appeared at a news conference to announce the new rules Wednesday.
Dr. Woodcock, of the FDA’s Center for Drug Evaluation and Research, is the subject of an investigation by the inspector general of the Department of Health and Human Services for an alleged conflict of interest involving drug approvals at the FDA. #