Off-label prescribing: Comparative evidence, regulation, and utilization

标签外处方：比较证据、监管和利用

基本信息

批准号：
8507154
负责人：
AARON SETH KESSELHEIM
金额：
$ 15.93万
依托单位：
BRIGHAM AND WOMEN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2009
资助国家：
美国
起止时间：
2009-09-30 至 2014-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8507154
关键词：
Off label prescribing Comparative evidence

项目摘要

Off-label prescription of drugs is a routine practice among physicians. The Food and Drug Administration (FDA) approves new drugs based on their effectiveness for a particular indication, but once a drug enters the market, physicians can prescribe drugs for other indications that they deem appropriate. Sometimes, such prescribing is based on rigorous randomized trial data that has not (yet) led to formal FDA approval for a given indication; other off-label use is based on less well-established evidence. Because of concerns about cost and/or quality of care, payors in both the private and public sectors have sought to prohibit off-label use, limit its coverage, and/or require patients to assume an increasing fraction of its cost. Off-label drug use thus involves prescriptions that may or may not be sound therapy, that may involve unnecessary risk to patients, and that may be cost-effective or cost-ineffective-or a mix of all three. Though off-label use has been estimated to account for nearly three-quarters of the use of some drugs, the practice remains poorly understood. Few studies have detailed extent to which newly approved drugs are prescribed for off-label uses, or the evidence supporting such utilization. In addition, scant data exist regarding the patient or physician or drug characteristics that predict when an off-label use is more likely to occur. Though promotion of drugs for off-label uses is strictly regulated, there have been several recent high-profile instances of violation of these regulations. No studies have systematically evaluated the impact of marketing or other legal, regulatory, or financial forces on the practice of off-label drug use. We propose to develop a comprehensive scheme for studying off-label drug use to categorize the different types of off-label use and their clinical, economic, and policy implications. We will apply this typology to evaluate the frequency and patterns of off-label drug use in three important categories: oncology drugs, neuropsychiatric drugs, and drugs for other rare diseases. We will identify target drugs and review the medical literature, as well as expert opinion, regarding specific uses of each product to define the quality of evidence supporting each off-label use. We will then evaluate the characteristics of off-label drug use in large population databases of prescriptions and diagnoses, including Medicaid patients, Medicare patients, and those covered by a large health insurer. Using these datasets, we will use multivariable regression to determine predictors of use of specific off-label drugs for particular purposes, and identify characteristics of patients, physicians, and medications that are associated with off-label use. Finally, we will use time-trend analysis to determine the impact of changes in legal, regulatory, or financial factors that impact off-label use. A better understanding of the properties and predictors of off-label use can inform evidence-based prescribing of these products. Studying the benefit-risk-cost relationships associated with such uses can lead to more enlightened approaches to prescribing and policy decisions in this increasingly contentious area.

药物的标签外处方是医生的常规做法。食物和药物管理（FDA）根据其特定迹象的有效性批准了新药，但曾经批准药物进入市场，医生可以开药，以确定其认为适当的其他迹象。有时，这种处方是基于严格的随机试验数据，但尚未（尚未）导致正式FDA 批准给定指标；其他标签的使用是基于较不公平的证据。由于对成本和/或护理质量的担忧，私营部门和公共部门的付款人都试图禁止标签外使用，限制其覆盖范围和/或要求患者承担其成本的越来越小。因此，使用标签的药物使用涉及可能或可能不是合理疗法的处方，可能涉及对患者的不必要风险，这可能是成本效益或成本效益，或者是这三者的混合。尽管据估计，标签外的使用估计可以说明使用某些药物的近四分之三仍然很了解。很少有研究详细规定了新批准的药物的详细范围标签外使用或支持这种利用的证据。此外，关于患者或预测何时使用标签的医师或药物特征更有可能发生。虽然促进标签外用途的药物受到严格监管，最近有一些侵犯了一些备受瞩目的实例这些法规。没有研究系统地评估了营销或其他法律，监管的影响或金融部队在标签非毒品使用方面的实践。我们建议制定一项全面的计划，用于研究标签外药物的使用，以分类不同类型的非标签使用及其临床，经济和政策影响。我们将应用此类型学评估三个重要类别中标签非标签药物使用的频率和模式：肿瘤药物，神经精神药物和其他罕见疾病的药物。我们将确定目标药物并审查医疗关于每种产品的特定用途以定义证据质量的文献以及专家意见支持每个标签的使用。然后，我们将评估大量人群中标签外药物使用的特征处方和诊断的数据库，包括医疗补助患者，医疗保险患者以及涵盖的数据库由大型健康保险公司。使用这些数据集，我们将使用多变量回归来确定用于特定目的的特定标签外药物，并确定患者，医师和与标签外使用相关的药物。最后，我们将使用时间趋势分析来确定影响标签外使用的法律，法规或财务因素的变化的影响。更好地了解非标签使用情况的属性和预测因素可以为循证提供信息这些产品的处方。研究与此类用途相关的利益风险成本关系可以领导在这个日益有争议的领域中，更开明的规定和政策决策的方法。