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ABSTRACT Background Methods Results Conclusion Off‐label drug use (OLDU) is a common practice in health care institutions, and numerous guidance documents have been developed to guide the management of OLDU in China. This scoping review aims to compare these documents and identify existing issues.PubMed, EMbase, three Chinese databases, the National Public Service Platform for Standards Information and the official websites of pharmaceutical‐related associations were searched to identify guidance documents relevant to the management of OLDU for Chinese health care institutions. We extracted and compared the recommended practices for various aspects of OLDU management, including management systems, organizational structure, prerequisites for OLDU, approval processes, evidence‐based evaluation, informed consent, and other related aspects.A total of 16 guidance documents were included, comprising 12 expert consensuses, 2 practice guidelines, and 2 group standards. Only six documents provide specific requirements for the establishment of management systems. Management of OLDU requires involvement from multiple departments or committees, yet only a few documents explicitly delineate the supervisory authority, and the responsibilities of the parties involved. These documents also show significant disparities in their approval process, evidence‐based evaluation, and informed consent recommendations. Furthermore, only a minority of the documents provide specific requirements for training and assessments focused on OLDU and improving adverse reaction monitoring.These guidance documents differ significantly in their specific recommendations for the management of OLDU and lack sufficient emphasis on certain critical aspects. It may be beneficial for health administrative authorities to promote the development of unified national guidelines. [ABSTRACT FROM AUTHOR]

Drug package inserts are a crucial foundation for clinical medication practices and serve as the legal basis for guiding rational drug use and ensuring patient safety and efficacy. As rare disease treatments evolve, current package inserts often need to meet the clinical requirements for treating such conditions, frequently resulting in off-label drug use. This consensus is derived from discussions between Guangdong Pharmaceutical Association Hematologic Rare Diseases Group experts. The consensus aims to provide a framework and reference for the clinical application of off-label drug use in treating rare hematologic diseases. [ABSTRACT FROM AUTHOR]

OBJECTIVE: To establish the pharmaceutical care pathways of nanoparticle albumin bound paclitaxel ( nab-PTX), so as to provide reference for clinical rational drug use. METHODS: Data of patients who used nab-PTX from Jan. 2020 to Jun. 2023 in the hospital were retrospectively analyzed and evaluated. The pharmaceutical care pathways of rational use of nab-PTX was established based on the instructions of nab-PTX, evidence-based medicine, and the real-world data of nab-PTX use in the hospital. RESULTS: Data analysis of 279 patients included in this study showed that 42 cases of metastatic breast were in line with the drug indications of nab-PTX, and 237 cases (84. 9%) were off-label. There were 209 off-label cases with evidence-based medical evidence (including cervical cancer, pancreatic cancer, stomach cancer, non-small cell lung cancer, nasopharyngeal cancer, ovarian cancer or fallopian tube cancer, esophageal cancer, endometrial cancer, and cholangiocarcinoma), which were evaluated as rational drug use. However, some off-label cases (oral cancer, mediastinal tumor, bone carcinoma, soft-tissue carcinoma, prostatic cancer, and malignant duodenal neoplasm) were evaluated as irrational due to insufficient evidence-based medical evidence. The most common adverse drug reactions of nab-PTX were myelosuppression and peripheral neuropathy, which were consistent with the drug instructions. The pharmaceutical care pathways of nab-PTX were established from various aspects, including pre-chemotherapy, chemotherapy, post-chemotherapy and follow-up. CONCLUSIONS: Offlabel drug use of nab-PTX is common, and the management for application of nab-PTX should be strengthened. The development of pharmaceutical care pathways of nab-PTX can improve the work efficiency of pharmacists and ensure the safe and rational drug use of cancer patients. [ABSTRACT FROM AUTHOR]

Background: Children with medical complexity (CMC) often require multiple medications, leading to polypharmacy, which seems to be linked to adverse effects, administration errors, and increased caregiver burden. This study aimed to describe the prevalence of polypharmacy, medication burden, off-label drug use, and associated costs. Methods: Conducted at the Pediatric Palliative Care Center of Padua, Italy, from August to October 2021, this cross-sectional observational study included patients up to 23 years old with at least one prescribed drug. Data were collected from medical records and caregiver interviews. Drug costs were collected from the Italian Medicine Agency. Descriptive statistical analysis was performed. For comparisons among categorical variables, the Chi-square test was used, and for those among continuous variables, the ANOVA test was used. Results: This study analyzed treatment regimens of 169 patients with a median age of 12.5 years (0.3–23). Polypharmacy was present in 52.7% of patients, and medication burden was observed in 44.4%, both varying significantly by primary diagnosis (p < 0.001). The median daily cost per patient was EUR 2.2 (IQR 0.9–7.1), with significant variation among subgroups. Only 34.6% of prescriptions were off-label. Conclusions: polypharmacy and medication burden are frequent among our CMC population, with some differences according to primary diagnosis. [ABSTRACT FROM AUTHOR]

Drug package inserts are a crucial foundation for clinical medication practices and serve as the legal basis for guiding rational drug use and ensuring patient safety and efficacy. As rare disease treatments evolve, current package inserts often need to meet the clinical requirements for treating such conditions, frequently resulting in off-label drug use. This consensus is derived from discussions between Guangdong Pharmaceutical Association Hematologic Rare Diseases Group experts. The consensus aims to provide a framework and reference for the clinical application of off-label drug use in treating rare hematologic diseases.

Relevance. Off-label use of medications is a common practice in clinical settings. To address the growing interest in this issue, the International Society for Pharmacoepidemiology (ISPE) has developed new general guidelines for off-label prescribing in clinical practice that emphasize the importance of evidence-based medicine and promote transparent communication between healthcare professionals and patients.Objective. The aim of this work was to analyze and present key trends for optimizing off-label prescribing decisions based on the ISPE recommendations (2023).Results. The report highlights five main recommendations: seeking strong scientific evidence, including the use of real-world data; using expert knowledge to evaluate and summarize evidence; developing recommendations with rigorous consistency; aligning the use of off-label medications with research; and strengthening collaboration among regulators, researchers, clinicians, and the pharmaceutical industry.Conclusions. A comprehensive approach is required to address the problem of off-label use of medicines. Implementation of these initiatives will reduce the risks associated with off-label use, generate sustainable scientific evidence, and improve the quality of patient care.

OBJECTIVE: To investigate the management status of off-label drug use in 146 medical institutions, so as to provide reference for improving the management level of off-label drug use. METHODS: Off-label drug use management in 146 medical institutions was investigated by questionnaire survey, including 104 tertiary hospitals and 42 secondary hospitals. Basic situation, management status, existing problems and successful experience were statistically analyzed. RESULTS: There were different degrees of off-label drug use in 146 medical institutions, of which 125 (85. 62%) carried out off-label drug use management. The management measures included establishment of management system (125 / 125, 100. 00%), formulation of off-label drug use catalogue (106 / 125, 84. 80%), and supervision for rationality of use process ( 112 / 125, 89. 60%). Totally 97. 26% ( 142 cases) of the respondents believed that sufficient evidence-based basis was the premise of off-label drug use. The proportion of medical institutions with sufficient evidence of off-label drug use was “about 50%” in 33. 56% (49 institutions), “almost all” in 34. 25% (50 institutions), and “few” in 30. 14% (44 institutions). Off-label drug use management had problems such as clinicians’ lack of cooperation / attention, non-use in accordance with prescribed procedures ( 116 / 146, 79. 45%), widespread off-label phenomenon, and difficulty in comprehensive management ( 73 / 146, 50. 00%). Management of off-label drug use can be improved by multi-department cooperation, clear responsibilities, hierarchical management according to the level of evidence or risk, and the introduction of information management. CONCLUSIONS: The phenomenon of off-label drug use is widespread, and medical institutions have made some success in the management of off-label drug use, yet there are also difficulties. Successful experience should be promoted to improve the management level of off-label drug use in medical institutions, so as to standardize the behavior of off-label drug use. [ABSTRACT FROM AUTHOR]

Background: Children with medical complexity (CMC) often require multiple medications, leading to polypharmacy, which seems to be linked to adverse effects, administration errors, and increased caregiver burden. This study aimed to describe the prevalence of polypharmacy, medication burden, off-label drug use, and associated costs. Methods: Conducted at the Pediatric Palliative Care Center of Padua, Italy, from August to October 2021, this cross-sectional observational study included patients up to 23 years old with at least one prescribed drug. Data were collected from medical records and caregiver interviews. Drug costs were collected from the Italian Medicine Agency. Descriptive statistical analysis was performed. For comparisons among categorical variables, the Chi-square test was used, and for those among continuous variables, the ANOVA test was used. Results: This study analyzed treatment regimens of 169 patients with a median age of 12.5 years (0.3–23). Polypharmacy was present in 52.7% of patients, and medication burden was observed in 44.4%, both varying significantly by primary diagnosis (p < 0.001). The median daily cost per patient was EUR 2.2 (IQR 0.9–7.1), with significant variation among subgroups. Only 34.6% of prescriptions were off-label. Conclusions: polypharmacy and medication burden are frequent among our CMC population, with some differences according to primary diagnosis.

Background: Thrombopoietin receptor agonists (TPO-RAs) have been widely used to treat thrombocytopenia, however, a scientometric profile of TPO-RAs research is lacking. Methods: This study uses VOSviewer, CiteSpace, and R software to provide an overview of current research, highlight study hotspots, and predict future research directions of TPO-RAs. Results: One thousand seven hundred and nineteen relevant studies from 1993 to 2022 with 43962 citations were identified from the Web of Science Core Collection. Over three decades, the USA has been leading TPO-RAs publications. Industries and academic institutions have been actively involved in TPO-RAs research, with funding provided by pharmaceutical companies and public funding bodies. The most productive and cited journals are British Journal of Hematology and Blood, respectively. When author keywords were categorised into three clusters, i.e., cluster 1 (immune thrombocytopenic purpura (ITP)), cluster 2 (avatrombopag, lusutrombopag, and thrombocytopenia), and cluster 3 (TPO-RAs for ITP and off-label drug use), ITP was found to be the current research hotspot, while oral TPO-RAs and licensed or unlicensed drug indications of thrombocytopenic diseases require further investigation. Conclusion: This study has generated the knowledge map of TPO-RAs, which provides a dynamic roadmap for future research in this field.

Thrombopoietin receptor agonists (TPO-RAs) have been widely used to treat thrombocytopenia, however, a scientometric profile of TPO-RAs research is lacking. Methods : This study uses VOSviewer, CiteSpace, and R software to provide an overview of current research, highlight study hotspots, and predict future research directions of TPO-RAs. Results: One thousand seven hundred and nineteen relevant studies from 1993 to 2022 with 43962 citations were identified from the Web of Science Core Collection. Over three decades, the USA has been leading TPO-RAs publications. Industries and academic institutions have been actively involved in TPO-RAs research, with funding provided by pharmaceutical companies and public funding bodies. The most productive and cited journals are British Journal of Hematology and Blood, respectively. When author keywords were categorised into three clusters, i.e., cluster 1 (immune thrombocytopenic purpura (ITP)), cluster 2 (avatrombopag, lusutrombopag, and thrombocytopenia), and cluster 3 (TPO-RAs for ITP and off-label drug use), ITP was found to be the current research hotspot, while oral TPO-RAs and licensed or unlicensed drug indications of thrombocytopenic diseases require further investigation. Conclusion: This study has generated the knowledge map of TPO-RAs, which provides a dynamic roadmap for future research in this field., Competing Interests: The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Rong Hu reports statistical analysis, and writing assistance were provided by Guangdong Pharmaceutical Association. Rong Hu reports statistical analysis, and writing assistance were provided by Science and Technology Program of Guangzhou. Min Liu reports article publishing charges, statistical analysis, and writing assistance were provided by National Natural Science Foundation of China., (© 2024 The Authors. Published by Elsevier Ltd.)