The issue of off-label prescribing is heating up again. A recent article in the New England Journal of Medicine by Randall Stafford made the case for greater regulation. I am concerned that the benefits of off-label prescribing are not fully appreciated. Dan Klein and I wrote a letter to the NEJM – which they declined to publish – in response. Here’s the letter:
R.S. Stafford writes that off-label prescribing “permits innovation in clinical practice … offers patients and physicians earlier access to potentially valuable medications and allows physicians to adopt new practices based on emerging evidence.” Nevertheless, he calls for greater FDA regulation.
In contrast, we argue that the efficacy of off-label usage suggests that less FDA regulation of first or on-label usage would increase innovation and offer patients earlier access to new medications.
Off-label prescribing is regulated by the judgments of doctors, medical researchers, industry, the patient community, and patients. This system offers patients a more nuanced approach to care than a top-down approach. We should extend this approach to new drugs as well as to new uses for old drugs.
Our perspective is bolstered by a large survey of physicians which demonstrates strong support for off-label prescribing and considerable support for reducing FDA regulations on new drugs.
George Mason University