FDA Issues Rule on Patient Access to Experimental DrugsBy...

FDA Issues Rule on Patient Access to Experimental Drugs
By JENNIFER CORBETT DOOREN

WASHINGTON -- The Food and Drug Administration finalized new regulations Wednesday designed to provide broader access to experimental drugs for seriously ill people who have exhausted all other commercially available treatments.

The new rule, which was posted on the FDA's Web site Wednesday, clarifies existing regulations explaining how patients can receive access to drugs in development for various conditions as well as providing additional so-called expanded-access programs to allow more patients to have access to therapies under development.

"The final rule is intended to improve access to investigational drugs for patients with serious or immediately life-threatening diseases or conditions who lack other therapeutic options and [who] may benefit from such therapies," the agency said.

Currently, large patient groups with cardiovascular diseases, cancer and human immunodeficiency virus, or HIV, have long been able to access drugs that are currently being developed through clinical trials. However, outside of clinical trials, FDA regulations have been confusing and potentially a barrier to small groups and individual patients, or small patient groups who want to receive access to experimental treatments.

The rule also allows companies and researchers to charge patients for the experimental treatments, potentially allowing more start-up or smaller firms to participate in expanded-access programs. However, the FDA said the cost of an investigational drug used in clinical trial "is an anticipated cost of drug development and should ordinarily be borne by the sponsor."

If companies or researchers charge for the cost of manufacturing and providing a drug as part of an expanded-access program, they aren't allowed to make a profit.

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