The floodgate has opened for molecularly targeted antitumor agents, and with each novel compound the cost of treating cancer soars ever higher. Approval by the US Food and Drug Administration (FDA) is usually based on a delay in disease progression, but just a 2-month benefit may be statistically significant in large randomized phase 3 trials. In contrast, the United Kingdom’s National Health Service (NHS) and its advisory branch, the National Institute for Health and Clinical Excellence (NICE), have been less supportive of their use.
NICE provides guidance to the NHS on the effective use of ever-limited resources. In the past year or so, it has recommended against approval (although some decisions are preliminary and still open to appeal; see www. nice.org.uk) of the following targeted agents, which have received FDA approval in the United States: