title: Designing a strategy to promote safe, innovative off-label use of medications.
abstract: Innovative off-label medication use (defined as prescribing with reasonable rationale for use, but insufficient evidence to allay safety, efficacy, and cost-effectiveness concerns, yet is not clinical research) is common practice and provides challenges to ensuring high-quality health care and patient safety. This article describes a strategy to promote policy and standardization of innovative off-label medication use, ensure oversight of patient safety, and prospectively assess efficacy. A multidisciplinary group developed a policy and process to regulate innovative off-label medication use that standardizes formulary review, maximizes peer expertise input, and minimizes institution liability by evaluating the effectiveness of use, promoting evidence-based practices, and ensuring ethical obligations to patients and society. This strategy has been implemented through institutional staff structure. The review process balances benefits/risks for biologically plausible therapy that lacks rigorous data support. The authors' strategy illustrates collaboration that enables a priori consideration for innovative off-label medication use while providing safety surveillance and outcomes monitoring.
authors.affiliation: University of Pittsburgh School of Pharmacy, Pittsburgh, PA, USA. nicole.ansani@pfizer.com
authors.forename: Nicole
authors.initials: N
authors.lastname: Ansani
authors.forename: Carl
authors.initials: C
authors.lastname: Sirio
authors.forename: Thomas
authors.initials: T
authors.lastname: Smitherman
authors.forename: Bethany
authors.initials: B
authors.lastname: Fedutes-Henderson
authors.forename: Susan
authors.initials: S
authors.lastname: Skledar
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Weber
authors.forename: Nathalie
authors.initials: N
authors.lastname: Zgheib
authors.forename: Robert
authors.initials: R
authors.lastname: Branch
mesh_terms: D004348:Drug Labeling / Q000592:standards; D006801:Humans; D008508:Medication Errors / Q000517:prevention & control; D019982:Organizational Case Studies; D010414:Pennsylvania; D004364:Pharmaceutical Preparations; D016730:Program Development; D011786:Quality Control; D017751:Safety Management / Q000458:organization & administration*
chemical_list: D004364:Pharmaceutical Preparations