A report from, of all places, the US General Accounting Office1 appears in this issue of JAMA documenting that the majority of American oncologists are prescribing for cancer patients drug regimens that lack sufficient evidence of effectiveness for Food and Drug Administration approval. It further shows that most oncologists find that the Health Care Financing Administration and private insurance carriers are increasingly resistant to reimburse for such "off-label" drug uses. This problem, as well as a parallel issue of perceived overly rigid standards for approval of new anticancer drugs, has led organized groups of oncologists to plead for redress through their publications and through paid congressional lobbyists. These pleas have been supported by activist groups, as well as by officials of the National Cancer Institute, Bethesda, Md.
Simplistically, one might ask: If there is no sound scientific evidence that a therapy is effective, ie, that
Moertel CG. Off-Label Drug Use for Cancer Therapy and National Health Care Priorities. JAMA. 1991;266(21):3031-3032. doi:10.1001/jama.1991.03470210099042