High Prevalence of Off-Label Prescribing in Pediatric Palliative Care
Researchers explored the differences in off-label prescribing between pharmacists and physicians among a cohort of patients in pediatric palliative care.
Off-label prescriptions were significantly prevalent for pediatric patients undergoing palliative care, according to a study published in Children.1 Researchers’ finding underscores the need for greater physician-pharmacist collaboration to ensure safer and more effective prescription regimens.
“Children, defined as individuals aged 0 to 18 years, require targeted research to investigate the pharmacological and toxicological aspects of medications, ensuring the development of safe, effective, and high-quality drugs,” wrote the authors. “Although clinical trials involving pediatric populations are essential for the safe use of medications in this demographic, they often encounter various ethical challenges and concerns.”
The use of pharmaceuticals is highly cautioned in newborns and young patients at the early developmental stages of their lives. Despite the immense need for caution among this population, the challenges and regulations in clinical pediatric medication trials have led to off-label prescribing.
Despite children needing targeted research to investigate the pharmacological aspects of a drug, they are often prescribed medications that are off-label and not specified for clinical use. | image credit: Studio Romantic / stock.adobe.com
READ MORE: Palliative Care Pharmacist Working to Improve Transitions of Care for Patients
Off-label drug use is simply defined as the use of a medication outside of its authorized and approved utilization, or Summary of Product Characteristics (SmPC) as the researchers noted.1 According to the FDA, the sole regulator of authorized medications in the US, off-label drug use is the unapproved use of an approved medication. FDA provided the example of using chemotherapy approved for 1 type of cancer to treat a different type of cancer.2
“Clinically, the absence of robust scientific evidence and an increased risk of adverse drug events are major concerns, as many off-label indications lack support from high-quality clinical trials,” they wrote.1 “Despite regulatory efforts to incentivize pediatric drug research, off-label prescribing remains widespread due to the limited availability of pediatric-specific formulations and the paucity of age-appropriate clinical trials.”
While issues remain in the field of pediatric research, the investigators in the current study looked to providers instead of the patients they serve. “This study aims to explore the perspectives of physicians and clinical pharmacists on off-label prescribing within [pediatric palliative care (PPC)], focusing on whether their views diverge and identifying potential gaps in knowledge regarding pediatric treatments,” they continued.
The study was conducted from August through October 2021 at a PPC center in Italy. All included patients were prescribed at least 1 medication and were 22 years old or younger. A total of 169 patients (51% women; median age, 12.5 years) were included in the researchers’ analysis.1 “The age distribution revealed that 5.9% were infants (1 month to 2 years), 19.5% were preschool-aged children (3–5 years), 22.5% were school-aged children (6–11 years), and 38.5% were adolescents (12–18 years),” added the authors.
Including clinical indication, patient age, route of administration, dosage regimen, and other relevant factors, researchers recorded the dimensions of providers’ off-label prescribing.
The main differences were that more physicians (57.1%) used prescriptions off label for different indications when compared with pharmacists (25.9%). More pharmacists (9.9%), however, prescribed off label based on route administration than physicians (4.6%), while pharmacists also prescribed more off label due to dosage when compared with physicians (44.1% for pharmacists vs. 18.3% for physicians).1
“Our analysis highlights the percentage (26.5%) of off-label prescriptions in this PPC cohort and the difference between physicians and pharmacists regarding its perception. This divergence underscores the complexity inherent in off-label prescribing practices and the need for a collaborative framework,” continued authors of the study.
While researchers highlighted the prevalence in off-label prescribing amongst patients undergoing PPC, they too identified disparities between physicians and pharmacists.
“This result can highlight both accuracy and strict adherence to regulation and guidelines by the pharmacists on one side and underestimation by the clinical physicians on the other,” they wrote.1 “This is probably because the common and routine use of these drugs in such a subspecialized context, despite their original indication, can lead physicians to become used to their prescription in this way and then to underestimate their off-label prescribing rate.”
Because of physicians’ increased attention on patients’ symptoms and clinical needs, researchers believe they were more inclined to prescribe off label because of their focus on safety and effectiveness, as opposed to drug-drug interactions in polypharmacy—defined as a patient regularly using 5 or more medications at the same time.3 As it’s in the name, researchers of the current study believe pharmacists are much better positioned to facilitate patients’ polypharmacy when compared with physicians.
From both providers’ patient-facing positioning in health care to their respective expertise in patient care and drug interactions, pharmacists and physicians should be working together to alleviate issues within PPC and off-label prescribing.
“In conclusion, fostering increased collaboration between health care providers, alongside the adoption of adaptable regulatory measures, will ensure the safe and effective use of off-label medications, ultimately benefiting pediatric patients. This integration might range from investigating the effects of regular medication reviews, which could reveal significant insights for optimizing drug therapy, to supporting and delivering effective patient education on drug adherence to empower patients and their caregivers, and ultimately improving care for vulnerable patients,” concluded the authors.1
READ MORE: Pediatrics Resource Center
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