Under current U.S. law, physicians may prescribe drugs and devices in situations not covered on the label approved by the Food and Drug Administration. Those supporting this system say that requiring FDA approval for off-label uses would unnecessarily impede the delivery of benefits to patients. Patients do benefit from off-label prescribing that is supported by sound scientific and medical evidence. In the absence of such evidence, however, off-label prescribing can expose patients to risky and ineffective treatments. The medical community and federal authorities should more actively promote patients' interests in receiving beneficial off-label treatments. To exercise responsible self-regulation, members of the medical community must determine whether available evidence justifies specific off-label uses and must promote information-gathering when the evidence is inadequate. Physicians should also discuss with patients the uncertainties accompanying off-label uses. Federal authorities should more closely monitor the effects of off-label prescribing and adopt other measures to reduce harm and enhance benefits produced by off-label prescribing.

Off-label prescribing: a call for heightened professional and government oversight.

Objective

To investigate to what extent and why new rheumatology patients access medical information online prior to first appointments and secondarily to ask whether they discuss information gained from the Internet with physicians.



Methods

Research was conducted in a teaching rheumatology clinic with a nonrandom sample of 120 English-speaking adults presenting for first appointments in rheumatology. Quantitative and qualitative data were gained in pre- and postappointment patient surveys and interviews, including online information gathering prior to first appointment, demographics, health status, information usage in patient-physician interactions, and satisfaction. Data were analyzed for significant relationships across variables and for qualitative insights into quantitative outcome measures.



Results

Of all patients, 87.5% looked up their symptoms or suspected condition prior to their first appointment and 62.5% of all patients sought that information on the Internet. Only 20% of online information seekers discussed that information with their physicians. Age and sex were significant predictors of Internet information seeking. Physician and patient appointment satisfaction was significantly higher when Internet information was discussed; however, most patients did not discuss their information seeking because they primarily feared being perceived as challenging their physician.



Conclusion

The majority of patients research their conditions online prior to initial appointments, but are unlikely to discuss that research with physicians even though discussion is related to higher satisfaction. Physicians may want to consider strategies for enabling communication about online research.

Prepared patients: Internet information seeking by new rheumatology patients

Health-care professionals who prescribe medicines have the professional duty to choose medicines that are in the best interest of their individual patient, irrespective if that patient is an adult or a child. However, the availability of medicines with an appropriate label for pediatric use is lagging behind those for adults, and even available pediatric drugs are sometimes not suitable to administer to children. Consequently, health-care professionals often have no other option than to prescribe off-label medicines to children. An important reason for use of off-label medicines is to improve access to (innovative) treatments or to address medical needs and preferences of patients, especially when no other options are available. However, off-label use of medicines is in general not supported by the same level of evidence as medicines licensed for pediatric use. This may result in increased uncertainty on efficacy as well as the risk for toxicity and other side effects. In addition, liability may also be of concern, counterbalanced by professional guidelines. Conclusion: The purpose of this joint EAP/ESDPPP policy statement is to offer guidance for HCPs on when and how to prescribe off-label medicines to children and to provide recommendations for future European policy.

/pdf/off-label-use-of-medicines-in-neonates-infants-children-and-1he6ksee90.pdf

Off-label use of medicines in neonates, infants, children, and adolescents: a joint policy statement by the European Academy of Paediatrics and the European society for Developmental Perinatal and Pediatric Pharmacology.

In the past two decades, many legislative and regulatory initiatives were taken globally to improve drug use in children. However, children are still found to be prescribed with off-label drugs. This study was conducted to provide an overview of the worldwide trend in off-label prescribing in children from the year 1996 to 2016. The articles published in PubMed, MEDLINE and Google Scholar were searched using text words: off-label, unlicensed, paediatric and children. Additional articles were identified by reviewing the bibliography of the retrieved articles. Full-text articles published in English which reported on the prevalence of off-label prescribing in children between January 1996 and December 2016 were included. A total of 101 studies met the inclusion criteria. Off-label prescribing definition included four main categories: age, indication, dose and route of administration. The three most common reference sources used in the studies were summary of product characteristics, national formularies and package inserts. Overall, the off-label prescribing rates in children ranged from 1.2 to 99.7%. The most common category of off-label prescribing in children was dose and age. This review highlighted that off-label prescribing in children was found to be highly prevalent throughout the past two decades, persistently in the neonatal intensive care units. This suggests that besides legislative and regulatory initiatives, behavioural, knowledge aspects and efforts to integrate evidence into practice related to off-label prescribing also need to be evaluated and consolidated as part of the concerted efforts to narrow the gaps in prescribing for children.

Two decades of off-label prescribing in children: a literature review

F OLLOWING US FOOD and Drug Administration (FDA) approval, clinicians may lawfully prescribe a drug or biologic agent for treatment regimens not specified in the approved labeling or package insert. This is known as off-label use. A recent study found that for 160 common drugs in the United States, off-label use accounted for 21% of all prescriptions. Many off-label uses are accepted as standard of care, for example, use of -blockers for congestive heart failure. Many other offlabel uses, however, lack evidence of clinical efficacy. In light of growing concerns about appropriateness and cost, off-label use has been identified as 1 aspect of problematic drug prescribing. Currently, there is little guidance to distinguish clearly between of f label uses that are wel l supported by evidence and those that are not. In this article, we address that gap at the level of the practicing physician. First, we identify characteristics of off-label use that signal the need for greater scrutiny. Second, we propose a framework that differentiates types of off-label use based on the available evidence and links the level of evidence to specific requirements for ethical prescribing. Although specifically targeted to off-label use, this framework is applicable to the general issue of appropriate prescribing in light of adequate evidence of risks and benefits.

Going off-label without venturing off-course: evidence and ethical off-label prescribing.

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.

Designing a Strategy to Promote Safe, Innovative Off-Label Use of Medications

Data are limited regarding how academic medical centers (AMCs) deal with medication use that represents a departure from product labeling; has reasonable rationale for use, but insufficient evidence to allay safety, efficacy, and cost-effectiveness concerns; yet is not clinical research (defined as innovative off-label medication use). This report describes national trends in management of innovative off-label medication use. A cross-sectional survey of US AMCs was conducted. Survey questionnaires were directed to drug information centers or pharmacy directors. Of 469 AMCs contacted, 104 responded (22%). Fifty-nine AMCs identified innovative off-label use as a challenge. Only 18 AMCs developed strategies to address this issue: 12 requiring initial reviews and 8 requiring clinical monitoring. Sixty-five AMCs indicated interest in data sharing of clinical outcomes for innovative off-label protocol(s). Innovative off-label medication use is a widely recognized challenge; however, few prospectively active AMC responses exist. The authors suggest development of systematic structured approaches within and across AMCs.

United States medical practice summary: innovative off-label medication use.

One important function of a pharmacy and therapeutics (P&T) committee is to provide judicious formulary review, with recommendations for management and selection of optimal treatment agents, and advice and strategies in therapeutic algorithms. The rapid pace of innovation (e.g., introduction of new

Practical approach to grading evidence for formulary recommendations

Purpose A large hospital’s systematic and evidence-based approach to adjudicating, monitoring, and ensuring the safety of off-label medication use is described.

Summary In 2003 the University of Pittsburgh Medical Center (UPMC)–Presbyterian implemented a policy that created a formal process for the systematic evaluation of formulary requests and drug-utilization patterns indicating or suggesting off-label use. Explicit criteria were developed for differentiating “innovative off-label use” (i.e., use based on a reasonable rationale yet lacking definitive scientific support in the form of fully published randomized controlled trials) from medication use more appropriately classified as clinical research. The UPMC–Presbyterian policy also outlined a process for the development, implementation, and evaluation of guidelines on innovative off-label use, including the collection of efficacy and safety outcomes. As of October 2012, 31 proposals for off-label medication use had been evaluated by the medical center’s pharmacy and therapeutics committee and formulary subcommittee. Thirteen requests resulted in a determination of innovative off-label use and the development of prescribing guidelines, and 10 prompted the extension of an agent’s current formulary status; in 6 cases, proposed off-label uses were determined to constitute clinical research. In some instances, innovative off-label medication use generated safety and outcomes data that led to changes in local standards of care. An algorithm to guide decision-making with regard to requests and proposals for off-label medication use is provided.

Conclusion The UPMC–Presbyterian experience indicates that off-label medication use can be effectively managed using evidence-based principles and peer review mechanisms.

Addressing innovative off-label medication use at an academic medical center.

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Designing a Strategy to Promote Safe, Innovative Off-Label Use of Medications

United States medical practice summary: innovative off-label medication use.

Practical approach to grading evidence for formulary recommendations

Addressing innovative off-label medication use at an academic medical center.