Regulatory Focus™ > News Articles > Report: Compassionate Use Exemptions Being Granted in Increasing Numbers

Report: Compassionate Use Exemptions Being Granted in Increasing Numbers

Posted 24 October 2012 | By

A so-called "compassionate use" program launched by the US Food and Drug Administration (FDA) is helping increasing numbers of patients access potentially life-saving drugs at early stages in their development process, reports the Wall Street Journal.

The compassionate use program is relatively straightforward: FDA allows manufacturers to provide some patients-generally only those with highly advanced and likely fatal diseases-with drug compounds still under early-stage clinical investigation, but they are not required to do so. Previously,  manufacturers would not be allowed to do so without explicit permission from FDA because the terms of an investigational new drug approval only provides for the release of the product in controlled, clinical settings.

Some 1,200 patients benefited from the program in 2011-up from around 1,000 in 2010, said the Journal.

The program is not without its detractors, however. Some companies are hesitant to let their products out of their tightly-controlled supervision, fearful that an erroneous or poorly-understood adverse event could sink their regulatory prospects or harm their stock. Further, some companies are worried that widespread adoption of compassionate use guidelines could harm their ability to populate clinical trials, delaying the entry of a drug and ultimately harming other patients who are still in need of the drug's therapeutic benefits.

Regulators, too, are still wary of the program. "There's this sense [from patients that] these are miracle drugs," said Richard Klein, director of FDA's Office of Special Health Issues, to the Journal. Even if the drug works-which is far from certain in early-stage clinical trials-the benefit might not amount to much.

But it's the shot at that miracle that keeps patients and their physicians clamoring for more access to experimental drugs, and if the data are to be believed, such requests are being granted in increasing numbers.

Read the Wall Street Journal's report:

WSJ - Connecting Patients With Experimental Drugs

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