Archives of disease in childhood
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Comparative Study
Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations.
Market forces may not be sufficient to stimulate research and development of medicines for small patient populations, such as children and patients with rare diseases. Both the European Union Orphan and Paediatric Regulations were introduced to address the unmet public health needs of these smaller patient populations through the use of incentives, rewards and obligations. Developers for new medicines for rare diseases must agree a paediatric investigation plan (PIP) or waiver with the European Medicines Agency's (EMA) Paediatric Committee (PDCO), and can also apply for an orphan designation (OD) from the EMA's Committee of Orphan Medicinal Products (COMP). The scope of both the OD and the PIP (or waiver) is defined by the agreed condition. ⋯ These findings demonstrate that a collaborative approach allows both Regulations to work synergistically to foster pharmaceutical development for rare diseases in childhood.
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Observational Study
Inter-rater reliability in the Paediatric Observation Priority Score (POPS).
The primary objective of this study was to determine the level of inter-rater reliability between nursing staff for the Paediatric Observation Priority Score (POPS). ⋯ This study suggests there is good inter-rater reliability between different nurses' use of POPS in assessing sick children in the emergency department.
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Randomized Controlled Trial Multicenter Study Comparative Study Pragmatic Clinical Trial
Drooling Reduction Intervention randomised trial (DRI): comparing the efficacy and acceptability of hyoscine patches and glycopyrronium liquid on drooling in children with neurodisability.
Investigate whether hyoscine patch or glycopyrronium liquid is more effective and acceptable to treat drooling in children with neurodisability. ⋯ Hyoscine and glycopyrronium are clinically effective in treating drooling in children with neurodisability. Hyoscine produced more problematic side effects leading to a greater chance of treatment cessation.
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To develop and test patient-reported experience measures (PREMs) for children and young people in a specialist paediatric hospital setting. ⋯ The new PREMs will provide children and young people receiving care in specialist paediatric hospitals with the opportunity to provide feedback on their experience. Sustainability and ensuring that feedback results in improvements need to be addressed in future work.
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Randomized Controlled Trial
Neonatal resuscitation using a laryngeal mask airway: a randomised trial in Uganda.
Mortality rates from birth asphyxia in low-income countries remain high. Face mask ventilation (FMV) performed by midwives is the usual method of resuscitating neonates in such settings but may not always be effective. The i-gel is a cuffless laryngeal mask airway (LMA) that could enhance neonatal resuscitation performance. We aimed to compare LMA and face mask (FM) during neonatal resuscitation in a low-resource setting. ⋯ This trial was registered in https://clinicaltrials.gov, with registration number NCT02042118.