Off-Label Use of Drugs and Medical Devices: A Review of Policy Implications

Abstract

Off-label use" occurs when the use of a medication or device deviates from what is mentioned in its US Food and Drug Administration (FDA) product label. Off-label use is common, legal, and an important source of innovation; however, it can be costly, and strong evidence of the efficacy and safety of such use may be lacking. 1 Given the contradictory and unresolved expectations of major stakeholders, off-label use remains problematic. It requires a new policy paradigm that can successfully balance the need for innovation against the imperatives of evidence-based practice and finite health-care resources.

Fifty years of preventing and treating childhood behaviour disorders: a systematic review to inform policy and practice

“… 16 43 As well, prescriptions for behaviour problems have often involved ‘off label’ use—raising additional risks for children. 44 The lack of long-term follow-up studies on these medications is a further cause for concern given the evidence amassing on cardiovascular, cognitive and other harms associated with the long-term use of antipsychotics such as risperidone and quetiapine in children. 26 45 More research is therefore needed on pharmacological treatments for childhood ODD/CD, better delineating long-term risks and benefits.…”

Off-label use of the expensive orphan drug eculizumab in France 2009–2013 and the impact of literature: focus on the transplantation field

“…1 Despite the very low prevalence of its on-label indications, eculizumab is classed among the highest drugs based on expenditures in France in 2013 among the list of products reimbursed separately from the diagnosis-orientated case group fees (DRG) 2 [16]. Even if its high per-unit cost could partly explain this phenomenon, this scenario is suggestive of off-label use, especially as eculizumab's pharmacological properties make it susceptible to be used off-label in multiple inflammatory or immunological diseases, including graft rejection [17].…”

“…Therefore, the study of the use of medicines for off-label indications offers the opportunity to improve patient care and the use of health resources [1][2][3][4][5].…”

The Many Sides of Off-Label Prescribing

“…This issue of the Journal includes papers that cover the waterfront of issues related to off‐label prescribing. Two articles provide updates on the status of off‐label use of drugs and devices 5 and the prevalence of off‐label use in special populations such as pediatrics 6 . To address the conflicting needs of regulatory agencies, payers, providers, patients, insurers, patient safety advocates, and others related to the appropriate use of drugs or devices, various strategies are suggested.…”