Off-Label Promotion Reform: A Legislative Proposal Addressing Vulnerable Patient Drug Access and Limiting Inappropriate Pharmaceutical Marketing

Off-label promotion occurs when pharmaceutical manufacturers engage in promotion of unapproved or “off-label” uses of their drugs. These off label uses may lack adequate clinical data to substantiate marketing claims, have led to corporate investigations and penalties, and can endanger public health. However there is adequate evidence to suggest that off-label uses are entirely appropriate for some vulnerable patient populations, and that physicians have accepted such uses as standard. Historically, U.S. law has prohibited direct off-label promotion to physicians and patients. However, failed government guidance, industry-based litigation, and the diminished capacity of regulators to police illegal practices have had dire consequences for patient safety and the prevention of healthcare fraud and abuse. Worse still, because of these policies, vulnerable disease patients and their physicians are often unaware of appropriate off-label treatments, and the lack of information places these patients at risk. To address these concerns, we propose the creation of a targeted and regulated off-label promotion system that enables vulnerable patient populations to access life-saving treatments and simultaneously creates clear incentives to avoid inappropriate off-label promotion. This federal legislation would create FDA-targeted exemptions of off-label prohibitions for vulnerable patient populations, if certain requirements of enrollment, risk management, and pharmacovigilance are met. Any proposed off-label promotion would also be pre-reviewed by the FDA to ensure that the program was safe and properly monitored. To create incentives for appropriate off-label marketing and avoid the problem of repeat corporate offenders, additional penalties would be available without preempting other causes of action. This system of carrots and sticks would increase drug access for vulnerable patient populations while discouraging illegal marketing that could threaten patient safety and public health.