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. Off label uses often lack adequate clinical data to substantiate marketing claims, may endanger the public health and have led to corporate investigations and penalties. On the other hand, off label uses are entirely appropriate for some vulnerable patient populations, since there is adequate evidence to suggest such a clinical approach and physicians have accepted such use as standard. Off-label promotion directly to physicians and patients has historically been prohibited by U.S. law. However, failed government guidance, industry-based litigation, and the resulting diminished capacity of regulators to police such illegal practices has created an environment that has had dire consequences for patient safety and healthcare fraud and abuse. Yet concomitantly, vulnerable disease patients and their physicians are often ignorant about the appropriate off label treatment that is best applicable to them, and policy efforts to prohibit such information flow places risks of the current off label policy directly on these patients. What is needed is an appropriately targeted and regulated off-label promotion system that benefits vulnerable patient populations who lack information and access to life-saving treatments while simultaneously creating a clear disincentive to engage in inappropriate off label promotion. We propose such as system through federal legislation. This legislation would create FDA targeted exemptions of off-label prohibitions for certain vulnerable patient populations, provided that certain requirements of enrollment, risk-management, and pharmacovigilance are met. As well, any general proposed off label promotion would also be pre-reviewed by the FDA before release as marketing, again with information and justification documented and post-approval safety monitoring plans in place. A relevant FDA Advisory Committee would review the materials for adequacy and accuracy, and provide a recommendation to the FDA Commissioner as to approval. Because this system would also provide drug companies with specific guidance on acceptable forms and contents of off label marketing, anything outside of this system would easily be deemed fraud and abuse by regulators and courts. In addition, to incentivize appropriate off label marketing approval and avoid the problem of repeat off label promotion offenders, the legislation would also provide for significant fraud and abuse penalties for these crimes, without preemption of other causes of action. Such penalties would include banning an offending pharmaceutical company from off label marketing programs as defined by the proposed legislation as well making more than one off label violation grounds for mandatory exclusion from federal health care programs by amending the Social Security Act. This systemic confluence of carrots and sticks will hopefully serve to increase drug access for vulnerable patient populations who lack information on treatment while disincentivizing illegal industry marketing that threatens the public health.