Article type
Abstract
Background
Off-label drug use, the practice of prescribing medications beyond approved indications, is widespread in clinical settings. Evaluating the supporting evidence is crucial for the safety of patients and effectiveness of treatment.
Objectives
We aim to (1) comprehensively investigate how evidence for off-label drug use can be evaluated in related databases, (2) assess the appraisal of this evidence in guidelines and consensus statements at present, and (3) identify existing frameworks and methods employed for evaluating off-label drug use.
Methods
An extensive review of literature and documents from diverse global sources, including PubMed, Web of Science, and various regional databases and health care websites, was conducted. We incorporated databases, guidelines, consensus statements, and methodological descriptions related to off-label drug use. First, we collated and synthesized standardized evaluation models for guiding off-label drug use that cover the origin and categorization of the evidence and the application of various tools and methodologies. A critical part of this analysis involves detailing the types of evidence (for instance, randomized controlled trials versus observational studies versus expert opinion statements) and the corresponding evidence levels. Moreover, the examination extends to the explicit evaluation tools and methods employed in assessing off-label drug use, such as the GRADE system or the Delphi method. Finally, we will identify frameworks and instruments dedicated to evaluating different aspects of off-label drug use, such as effectiveness, safety, and economic implications, and summarize their main content and methods.
Conclusions
This study intends to clarify the evaluation landscape for off-label drug use and identify existing frameworks and their gaps. The findings will contribute to the development of sophisticated and universally applicable evaluation tools and improve the robustness and reliability of evidence on off-label drug use.
Off-label drug use, the practice of prescribing medications beyond approved indications, is widespread in clinical settings. Evaluating the supporting evidence is crucial for the safety of patients and effectiveness of treatment.
Objectives
We aim to (1) comprehensively investigate how evidence for off-label drug use can be evaluated in related databases, (2) assess the appraisal of this evidence in guidelines and consensus statements at present, and (3) identify existing frameworks and methods employed for evaluating off-label drug use.
Methods
An extensive review of literature and documents from diverse global sources, including PubMed, Web of Science, and various regional databases and health care websites, was conducted. We incorporated databases, guidelines, consensus statements, and methodological descriptions related to off-label drug use. First, we collated and synthesized standardized evaluation models for guiding off-label drug use that cover the origin and categorization of the evidence and the application of various tools and methodologies. A critical part of this analysis involves detailing the types of evidence (for instance, randomized controlled trials versus observational studies versus expert opinion statements) and the corresponding evidence levels. Moreover, the examination extends to the explicit evaluation tools and methods employed in assessing off-label drug use, such as the GRADE system or the Delphi method. Finally, we will identify frameworks and instruments dedicated to evaluating different aspects of off-label drug use, such as effectiveness, safety, and economic implications, and summarize their main content and methods.
Conclusions
This study intends to clarify the evaluation landscape for off-label drug use and identify existing frameworks and their gaps. The findings will contribute to the development of sophisticated and universally applicable evaluation tools and improve the robustness and reliability of evidence on off-label drug use.