The U.S. Food and Drug Administration (FDA) has announced two new rules related to experimental drug access. The goal of these rules is to clarify existing regulations and provide access of experimental drugs to a broader group of patients.
Until now, the only clear way to access experimental drugs was to take part in a clinical trial. While participation in a clinical trial was not mandatory, FDA regulations outside of the trial system have been muddy and a constant hurdle for those suffering from potentially fatal illnesses.
The first new regulation intends to clarify these rules so that individual patients and small patient groups have improved access to experimental drugs.
Experimental drugs are defined as those that are still in the process of being approved by the FDA. Clinical trials are a key component of approval, and serve to indicate the safety and expected results of new drugs. Typically, they are only available to individuals with serious, life-threatening illnesses, such as cancer and HIV/AIDS.
The second new regulation pertains to the ability for pharmaceutical companies to charge for experimental drugs. As Dr. Janet Woodcock, director of the FDA Center for Drug Evaluation and Research, explains: “Making these drugs available outside of a clinical trial can be costly to manufacturers. By permitting charging for expanded access we hope to stimulate more companies to provide investigational drugs.”
It should be noted that the FDA does not intend to allow a cost charge for all experimental drugs administered outside of clinical trials. Rather, the pharmaceutical company must provide financial data that indicates drug administration would only be feasible with some form of fee attached to the drug.
Regardless of payment approval, the FDA confirmed that drug manufacturers are not allowed to turn a profit off of experimental drugs.
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