Regulating pediatric off-label uses of medicines in the EU and USA: challenges and potential solutions

Guidi, Benedetta; Parziale, Andrea; Nocco, Luca; Maiese, Aniello; Russa, Raffaele La; Paolo, Marco Di; Turillazzi, Emanuela

doi:10.1007/s11096-021-01303-5

Cited by 20 publications

(19 citation statements)

References 15 publications

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“…Studies showed that there were deficiencies in the current drug instructions, including the absence of necessary content, unclear and too many nomenclatures, slow updates, and deviations from the current clinical guidelines ( Chinese Clinical Medication Safety Group of International Network for the Rational Use of Drugs, 2019 ). More importantly, the off-label use of drugs is common in pediatrics around the world, whether in hospitals or outpatient clinics ( Cui et al, 2021 ; Guidi et al, 2021 ). Therefore, drug instructions are not an ideal basis for pediatric nurses to make decisions for HAMs, let alone drug handbooks, which are potentially insufficient in terms of accuracy and timeliness.…”

Section: Discussionmentioning

confidence: 99%

Call for Decision Support for High-Alert Medication Administration Among Pediatric Nurses: Findings From a Large, Multicenter, Cross-Sectional Survey in China

Huang²,

Lü³

et al. 2022

Front. Pharmacol.

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Background: Children have a higher risk of medication errors (MEs) than adults. The Institute for Safe Medication Practice (ISMP) defined high-alert medications (HAMs) as a group of medications that could cause significant patient harm or even death when they are used in error. Nurses are actively involved in and responsible for patient care, especially in medication administration. This study aimed to estimate the knowledge, decision-making basis and confidence and decision support needs related to HAMs among pediatric nurses in China.Methods: A web-based, cross-sectional survey was conducted among pediatric nurses who were recruited from 14 member hospitals of the Pediatric Nursing Alliance of National Children’s Medical Center in China using a convenient sampling technique. Data were collected using a self-administered instrument composed of four parts: the demographic characteristics of participants, participants’ knowledge about HAMs, participants’ self-evaluation of the basis of and confidence in decision-making, and decision support needs regarding HAMs. Among the participants, the maximum score for HAM knowledge was 100. All data were entered and analyzed using SPSS 20.0.Results: A total of 966 nurses participated in this study. Nurses were found to have insufficient knowledge about HAMs, with a median (IQR) of 75.0 (70.0, 80.0), out of a maximum score of 100. Knowledge about HAM administration was significantly higher than that about HAM regulation, with a p value < 0.001. The three lowest-scoring items concerned HAM regulation, and the “Treat fentanyl skin patches as a regulated narcotic” item obtained the lowest score, with only 1/5 of respondents answering it correctly. Most participants reported that their basis for decision-making about HAMs was drug instructions (90.0%) or drug handbooks (81.9%) and evaluated their confidence in decision-making about HAMs as high or relatively high (84.6%). The decision-making difficulties when encountering HAMs focused on most stages of HAM administration, especially the appropriateness of prescriptions, checks, preparation and administration. The vast majority of participants assessed decision support as necessary or very necessary (92.0%), and the most popular options for decision support were computerized clinical decision support systems (46.4%) and real-time online communication with pharmacists (23.9%).Conclusion: Our study demonstrated the inadequacies in HAM knowledge, the basis and difficulty of decision-making, and decision support needs regarding HAMs in Chinese pediatric nurses. Nurses need greater support in HAM administration, including not only training but also adequate technology, mutually beneficial interprofessional collaboration, and a positive institutional culture.

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Section: Discussionmentioning

confidence: 99%

Call for Decision Support for High-Alert Medication Administration Among Pediatric Nurses: Findings From a Large, Multicenter, Cross-Sectional Survey in China

Huang²,

Lü³

et al. 2022

Front. Pharmacol.

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“…Cromoglicic acid (S01GX01) 0 (0) <1 (3) <1 (3) <1 (6) Saccharomyces boulardii (A07FA02) <1 ( 2) <1 (3) 0 (0) <1 ( 5)…”

Section: Otc Medicineunclassified

“…Furthermore, paediatric medicines are often used off label and children have different responses to pharmacotherapy than adults. [5][6][7][8] In order to keep track of children's medicine use and the various potential challenges associated with it, comprehensive population studies on medicine use and related factors are needed. Research on medicine use aims to promote safe and effective use of medicines by identifying the factors that lead to appropriate and inappropriate use.…”

Section: Introductionmentioning

confidence: 99%

Medicine use in children aged under 12 years—Population survey in Finland

Kesti

Saastamoinen

Lavikainen

et al. 2022

Pharmacoepidemiology and Drug

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Purpose: To study the prevalence of prescribed and over-the-counter (OTC) medicine use in children under 12 years of age in Finland and to examine factors associated with the medicine use. Methods: A nationwide population survey was carried out in Finland in spring 2018 with a random sample of children under 12 years (n = 8000). A response rate of 24% (n = 1921) was attained. The questionnaire was sent to the child's primary guardian. Current use of prescribed medicines and the use of OTC medicines in the preceding 2 days were the main outcome measures. Results: Of all the children 19% had used prescribed medicines, 15% OTC medicines, and 31% either prescription or OTC medicines during the preceding 22 days. Children's health related factors were positively associated with prescribed medicine use including fairly good to poor health status, symptoms experienced, and illnesses diagnosed by a physician. The guardian's healthcare education and use of prescribed medicines were also associated with children's prescribed medicine use. Symptoms of illnesses and fairly good health status were found to be associated with childrens' use of OTC medicines. Conclusions: The use of medicines in children under 12 years of age is quite common in Finland. As expected, children's health-related factors were associated with medicine use. Also, guardians' healthcare education and the use of prescribed medicines were associated with children's prescription medicine use. Children's medicine use should be continuously monitored and investigated further to identify the reasons leading to appropriate and inappropriate medicine use in children. K E Y W O R D S child, medicine use, over-the-counter medicines, prescribed medicines, survey Key Points • One in five children under 12 years in Finland used prescribed medicines and one in seven over-the-counter (OTC) medicines.

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“…This is particularly true for pediatric patients due to ethical and methodological obstacles that often arise when trying to prepare and conduct randomized controlled trials. Indeed, in pediatrics, off-label treatment represents roughly half of the total number of treatment prescriptions ( 1 ), reaching more than 90% in certain areas such as neonate intensive care units ( 2 ).…”

Section: Introductionmentioning

confidence: 99%

Off-label use of canakinumab in pediatric rheumatology and rare diseases

Giudice

Sota²,

Orlando³

et al. 2022

Front. Med.

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Since the first success of interleukin-1 blockade in cryopyrin-associated periodic syndrome, the use of interleukin-1 inhibitors has expanded to other disorders, including off-label indications. In particular, canakinumab has been employed in an off-label fashion in several diseases such as rare monogenic autoinflammatory diseases and multifactorial autoinflammatory diseases, disclosing an excellent efficacy and good safety profile in pediatric patients unresponsive to standards of care. In addition, hyperferritinemic syndromes and complex disorders, as well as Kawasaki disease, uveitis, and other pediatric rare disorders, represent additional areas where canakinumab efficacy is worth exploring. Altogether, the results summarized below are of paramount importance in pediatric patients where a considerable proportion of treatments are prescribed off-label. This review focuses on the off-label use of canakinumab in pediatric patients affected by systemic immune-mediated diseases.

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Regulating pediatric off-label uses of medicines in the EU and USA: challenges and potential solutions

Cited by 20 publications

References 15 publications

Call for Decision Support for High-Alert Medication Administration Among Pediatric Nurses: Findings From a Large, Multicenter, Cross-Sectional Survey in China

Call for Decision Support for High-Alert Medication Administration Among Pediatric Nurses: Findings From a Large, Multicenter, Cross-Sectional Survey in China

Medicine use in children aged under 12 years—Population survey in Finland

Off-label use of canakinumab in pediatric rheumatology and rare diseases

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