Management guideline for the off-label use of medicine in China (2021)

ABSTRACT

Background

Off-label drug use embodies a thorough clinical diagnosis and evaluation of treatment needs and should not be confused with unreasonable drug use, but it also faces potential risks with drug safety and legal issues.

Research design and methods

We first established a guideline working group. Following the guideline development process recommended by the World Health Organization Handbook and the Chinese Medical Association, the key questions were determined through literature searches of PubMed, CNKI (Chinese National Knowledge Infrastructure) and other databases. Both the evidence and the clinicians’ diagnosis and treatment workload were considered to formulate the initial recommendations. Finally, two rounds of Delphi surveys and one expert seminar were organized to determine the final recommendations of this guideline. Meanwhile, we graded the recommendations based on the body of evidence.

Results

We determined nine questions and proposed a total of 23 recommendations regarding the definition of off-label use of drugs, applicable circumstances, classification of evidence, informed consent, legal basis, adverse drug reaction monitoring and evaluation, management procedure, responsibilities and obligations of different stakeholders, medical insurance reimbursement, and the national approval system.

Conclusions

This guideline standardized clinical off-label drug use and provided suggestions and references for the management of off-label drug use.

KEYWORDS:

• Off-label use of drugs
• management
• evidence-based medicine
• hierarchical management
• guideline

Acknowledgments

We acknowledge the experts from the guideline external review group for their suggestions on this guideline and Janne Anton Markus Estill for the language polishing and revision of this manuscript.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

W Zuo and Y Sun wrote this manuscript which was then revised by all authors. B Zhang, Y Chen, and S Zhang proposed the conception of this guideline, supported the formulation of the guideline working group, and designed the framework of this guideline. All the listed authors participated in the guideline development. In addition to this, W Zu, Y Sun and R Liu analyzed the data of two rounds of the Delphi survey.

Ethics statement

This research follows the principles of the Declaration of Helsinki and was reviewed and approved by the Ethical Committee of the Peking Union Medical College and the Chinese Academy of Medical Sciences (Beijing, China).

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/17512433.2022.2120468

Additional information

Funding

This manuscript was funded by CAMS Innovation Fund for Medical Sciences (CIFMS 2021-I2M-1-003), National Natural Science Foundation of China Grants [No. 81974183].