Maihöfner Christian, Mallick-Searle Theresa, Vollert Jan, Kalita Pranab, Sood Sethi Vidhu
Department of Neurology, General Fürth Hospital, University of Erlangen, Fürth, Germany.
Division of Pain Medicine, Stanford Health Care Pain Management Clinic, Palo Alto, CA, USA.
Pragmat Obs Res. 2025 Jan 23;16:7-18. doi: 10.2147/POR.S504709. eCollection 2025.
In recent years, regulatory authorities have signaled a willingness to consider real-world evidence (RWE) data to support applications for new claims and indications for pharmaceuticals. Historically, RWE studies have been the domain of prescription drugs, driven by the fact that clinical data on patients are routinely captured in medical records, claims databases, registries, etc. However, RWE reports of nonprescription drugs and supplements are relatively sparse due to methodological gaps in this area. The objective of this narrative review is to identify which RWE methodologies have been used to study nonprescription products. A total of 49 articles were included based on literature searches. Label comprehension studies, used to support prescription-to-nonprescription switches, are useful in determining how nonprescription products will be used; however, they provide no actual clinical data. The most common RWE studies of nonprescription products were cross-sectional surveys, which investigated a broad range of indications and were conducted in an array of settings, including online, by phone, point-of-sale (pharmacy), outpatient clinics, and shopping malls. However, while this type of study is effective for identifying use patterns and attitudes in the general population, recall bias limits the ability to collect safety and effectiveness data. Studies of electronic medical records and claims databases are hampered by incomplete or absent capturing of data on nonprescription products. As a result, most RWE studies to date have provided limited useful information. Although case reports and expert opinion should not be discounted, in the absence of other information they provide few actual data. Novel approaches using smartphone apps and artificial intelligence may provide new opportunities to collect RWE for nonprescription products, but these areas of research are in their infancy. Overall, there is a need to develop standards for execution of RWE studies of nonprescription products in terms of endpoints, study design, and study quality.
近年来，监管机构已表明愿意考虑使用真实世界证据（RWE）数据来支持药品新适应症和新用途的申请。从历史上看，RWE研究一直是处方药的领域，原因是患者的临床数据通常记录在医疗记录、理赔数据库、登记处等中。然而，由于该领域存在方法学上的差距，非处方药和补充剂的RWE报告相对较少。本叙述性综述的目的是确定哪些RWE方法已被用于研究非处方产品。基于文献检索共纳入了49篇文章。用于支持处方药转换为非处方药的标签理解研究，有助于确定非处方产品的使用方式；然而，它们不提供实际临床数据。最常见的非处方产品RWE研究是横断面调查，该调查研究了广泛的适应症，并在一系列环境中进行，包括在线、电话、销售点（药店）、门诊诊所和购物中心。然而，虽然这类研究对于识别普通人群的使用模式和态度很有效，但回忆偏倚限制了收集安全性和有效性数据的能力。电子病历和理赔数据库的研究因非处方产品数据的不完整或缺失而受到阻碍。因此，迄今为止，大多数RWE研究提供的有用信息有限。尽管病例报告和专家意见不应被忽视，但在缺乏其他信息的情况下，它们提供的实际数据很少。使用智能手机应用程序和人工智能的新方法可能为收集非处方产品的RWE提供新机会，但这些研究领域尚处于起步阶段。总体而言，有必要在终点、研究设计和研究质量方面制定非处方产品RWE研究的执行标准。
