U.S. physician knowledge of the FDA‐approved indications and evidence base for commonly prescribed drugs: results of a national survey

  title={U.S. physician knowledge of the FDA‐approved indications and evidence base for commonly prescribed drugs: results of a national survey},
  author={Donna T Chen and Matthew K. Wynia and Rachael M. Moloney and G. Caleb Alexander},
  journal={Pharmacoepidemiology and Drug Safety},
The Food and Drug Administration (FDA) regulates prescription drug marketing, not prescribing. However, off‐label use is common, often lacks supporting evidence, and may expose patients to unwarranted risk. We sought to determine physicians' knowledge of the FDA‐approved indications of commonly prescribed drugs, and to assess whether physicians' belief that an indication is FDA‐approved increases with level of evidence supporting such use. 

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

  • R. Stafford
  • Medicine, Political Science
    Clinical pharmacology and therapeutics
  • 2012
Given the contradictory and unresolved expectations of major stakeholders, off‐label use remains problematic and 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.

Experts’ Views on FDA Regulatory Standards for Drug and High-Risk Medical Devices: Implications for Patient Care

Experts identified advantages of expediting development and of FDA flexibility in applying its standards to new drugs and medical devices, but highlighted concerns that patients may not be adequately informed about the risks of shorter review times or about uncertainties in the evidence that result.

First Amendment Protection of Evidence-Based Promotion of Prescription Drugs: A Study of Published Clinical Evidence Supporting Off-Label Promotion in the USA

Some of the off-label uses alleged in FCA complaints investigated and settled by the federal government were supported by published clinical evidence; however, many of the alleged uses were unsupported by any clinical evidence and, thus, false and misleading.

Prescription Drug and Pharmaceutical Policy

This chapter is a primer for medical trainees that highlights the ways in which drugs are developed, approved, and paid for in the USA as well as the important differences between brand and generic medications.

Off‐label drug use in oncology: a systematic review of literature

The aim of this review was to provide an overview of off‐label drug use prevalence in oncology and to address ethical concerns around the lack of informed consent.

Analyzing Patterns of Drug Use in Clinical Notes for Patient Safety

This work applied term extraction tools on the clinical notes of a million patients to compile a database of statistically significant patterns of drug use, and validated some of the usage patterns learned from the data against sources of known on-label and off-label use.

Off-Label Prescribing of Medications for Pain: Maintaining Optimal Care at an Intersection of Law, Public Policy, and Ethics

  • J. Ruble
  • Medicine
    Journal of pain & palliative care pharmacotherapy
  • 2012
In many circumstances, an off-label medication may be in the patient's best interests; however, where there is a lower level of clinical justification, the informed consent of the patient and shared decision making of the patients is essential to optimize outcome.

Off-Label Use and Prescribing What Does It Mean for the Foot and Ankle Surgeon?

This edition of In the Profession examines what it means to be off label in terms of regulations, clinical studies, industry marketing, and common areas of use.

Clinical trial evidence supporting FDA approval of novel therapeutic agents, 2005-2012.

The quality of clinical trial evidence used by the FDA as the basis for recent approvals of novel therapeutic agents varied widely across indications, having important implications for patients and physicians as they make decisions about the use of newly approved therapeutic agents.

Using patients to promote evidence-based prescribing: comment on "Communicating uncertainties about prescription drugs to the public".

A patient-oriented approach to promote use of drugs with proven clinical benefit and a well-established track record of safety is reported on, with results suggesting that a simple warning improved patient decision-making.



Report of the Council on Scientific Affairs*: Unlabeled Indications of Food and Drug Administration-Approved Drugs

The AMA's Council on Scientific Affairs reviews and makes recommendations on four important subjects related to unlabeled uses: clinical significance and prescribing; coverage and reimbursement; dissemination of information by pharmaceutical manufacturers; and improving the Supplemental New-Drug Application (SNDA) process.

Off-label prescribing among office-based physicians.

Off-label medication use is common in outpatient care, and most occurs without scientific support, so efforts should be made to scrutinize underevaluated off-label prescribing that compromises patient safety or represents wasteful medication use.

‘Off-label’ drug use: an FDA regulatory term, not a negative implication of its medical use

The doctor's decision to inform the patient of the ‘off-label’ status of the prescription is not relevant to the physician's standard of care for an informed consent case, and doctors should not be branded with the additional duty of disclosing non-pertinent information, such as the FDA's medically irrelevant distinction, to their patients.

The Major Role of Clinicians in the Discovery of Off‐Label Drug Therapies

The role of clinicians in the discovery of off‐label use of prescription drugs approved by the United States Food and Drug Administration is investigated.

Off-label drug use in human immunodeficiency virus disease.

  • C. BrosgartT. Mitchell D. Abrams
  • Medicine, Political Science
    Journal of acquired immune deficiency syndromes and human retrovirology : official publication of the International Retrovirology Association
  • 1996
The prevalence of off-label drug use in routine clinical practice and the development of newer and more costly drugs for treatment of HIV and its medical complications argues for the articulation of an explicit national reimbursement policy for off- label uses of prescription drugs so that medically appropriate therapies will be available to those with insurance in a rational, consistent way.

Off-label drug prescribing on a state university obstetric service.

Off-label drug use occurs when physicians prescribe a drug recommended for indications other than those listed on the prescription labeling. The purpose of the present investigation was to describe

Off-label drug prescribing on a state university obstetric service.

Off-label drug use occurs when physicians prescribe a drug recommended for indications other than those listed on the prescription labeling. The purpose of the present investigation was to describe

National trends in cyclooxygenase-2 inhibitor use since market release: nonselective diffusion of a selectively cost-effective innovation.

Marked increases in COX-2 inhibitor use have occurred since their release, primarily among patients at low risk for adverse events from NSAIDs, demonstrating the challenge of limiting innovative therapies to the settings in which they are initially targeted and maximally beneficial.

Prioritizing Future Research on Off‐Label Prescribing: Results of a Quantitative Evaluation

A prioritized list of individual drugs for which future research regarding off‐label uses is warranted is developed to develop a prioritized List of Individual Drugs.

Unlicensed and off label prescribing of drugs in general practice

This is the first study to show that a significant number of drugs prescribed for children by general practitioners are off label and highlights the anomalies and inadequacies of drug information for prescribers.