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FDA Reviews Approval Issues With Cancer Advocates

FDA Reviews Approval Issues With Cancer Advocates

WASHINGTON—Data from patients granted "compassionate use"
of an investigational cancer drug are examined but not usually pooled with
controlled clinical trial findings when the Food and Drug Administration (FDA)
considers whether to approve a new drug application (NDA), according to Richard
Pazdur, MD, director of the FDA’s Division of Oncologic Drug Products.

Dr. Pazdur discussed the evaluation of "compassionate use" data
and a number of other topics during a meeting between several FDA officials and
representatives of cancer patient advocacy groups.

Although commonly used, "compassionate use" is not a preferred
regulatory term, he said. It refers to the treatment use, as opposed to the
investigational use, of unapproved drugs.

Treatment use of unapproved drugs may be divided into two main groups:
single-patient treatment use and expanded access use, Dr. Pazdur said. Expanded
access refers to multiple patients treated under a single protocol. In the
single-patient use, individual treatment plans are drawn up for each patients.

The requirements for single-patient treatment include the existence of a
drug supplier and sponsor, a qualified investigator, institutional review board
(IRB) approval and informed consent, and FDA concurrence, Dr. Pazdur said.

There are two mechanisms for handling single-patient use of investigational
drugs, he said. In the first mechanism, the single-patient investigational new
drug (IND), a new sponsor files a separate IND for each patient.

In the second mechanism, called the single-patient exemption, there is
already an IND, an existing sponsor, and an investigational protocol. With this
mechanism, a patient who is ineligible for an investigational protocol is
treated under a plan that is a slight modification of the existing protocol.
"This is a more efficient mechanism for single-patient treatment,"
Dr. Pazdur said.


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