Palliative medicines for children – a new frontier in paediatric research

Abstract

Objectives This paper seeks to highlight from a UK perspective the current lack of a research evidence base in paediatric palliative care that has resulted in a paucity of available medicines with appropriate formulations (strength and dosage form) to provide symptom management for children with life-limiting illnesses and to raise awareness of this group of 'therapeutic orphans'. Currently, clinicians have limited, often unsuitable medication choices for their paediatric palliative care patients, with little hope of moving away from the status quo. Key Findings Most medicines used in children receiving palliative care are old and off-patent drugs, developed for and tested in an adult population. Many are not available in suitable formulations (dosage form and strength) for administration to children, and there are often no age-related profiles of adverse drug reactions or for safe dosing. Summary Existing regional paediatric palliative care networks and support organisations should lobby funding bodies and the academic community to support appropriate research for this group of therapeutic orphans. Support must also be provided to pharmaceutical companies in the development of suitable products with appropriate formulations.

Paediatric palliative care and intellectual disability—A unique context

“…There is a great need for randomized controlled trials and studies addressing the pharmacology of pain medications in children. Early progress is being made in this field; however, advocacy from funding bodies and the academic community is required to support appropriate research for this group of “therapeutic orphans” (Jamieson et al., ).…”

Healthcare professionals’ views of the use of oral morphine and transmucosal diamorphine in the management of paediatric breakthrough pain and the feasibility of a randomised controlled trial: A focus group study (DIPPER)

“…Conducting RCTs in this group of children is challenging, particularly in regard to maximising recruitment and minimising attrition. 12 The DIPPER study is a 4-phase investigation of the feasibility of a randomised controlled trial (RCT) of transmucosal diamorphine versus oral morphine for breakthrough pain in children and young people with life-limiting conditions. In focus groups as part of the DIPPER study, we explored healthcare professionals’ perspectives on the assessment and management of breakthrough pain in palliative care in children, the prescribing and administration of transmucosal diamorphine (buccal, sublingual and intranasal) and oral morphine, and views about patient/carer involvement in a future trial.…”

Transmucosal drug administration as an alternative route in palliative and end-of-life care during the COVID-19 pandemic

“…Some doctors, nurses and caregivers are not familiar with the principles of these delivery routes and are therefore uncertain about their efficacy [ 9 ]. Lack of clear guidance and local availability within hospital formularies are identified as barriers to their wider use [ 10 , 11 ]. Nevertheless, there is growing experience in paediatric palliative care practice, allowing the benefits of needle-free drug administration by family caregivers at home.…”