The National Paediatric Medicines Forum: Who we are and what are we doing.

Abstract

An increasing understanding of the pathophysiology of complex diseases has contributed to the explosion in availability of newer medications in recent years. Newer medications frequently have more specific mechanisms of action and improved clinical outcomes for their approved indications compared to standard care. This translation of research into practice is exciting because it reflects precision medicine principles and offers renewed hope for patients. Unfortunately, clinical decision-making is not always straight forward. Data are extremely limited in some patient groups, including those with rare conditions, or others not routinely included in clinical trials; paediatrics commonly feature in both scenarios.1 Newer medications are usually markedly more expensive than comparators. These factors complicate clinical decision-making in an individual patient. The Drug and Therapeutics Committees (DTCs) in each hospital are responsible for achieving effective medicines governance, equity of medicines access and quality use of medicines to underpin patient's therapy. The assessment of medication treatments has become an increasingly onerous workload for DTCs, reflecting the increase in newer medications often used for novel indications. In the context of limited high-quality evidence, there is substantial duplication of effort across tertiary paediatric institutions, with multiple paediatric DTCs considering similar applications with variability in processes and outcomes between DTCs.2 Furthermore, resources and training may be insufficient given the technical complexity of some submissions.2 In 2016, the Sydney Children's Hospitals Network (SCHN) proposed at the Children's Healthcare Australasia (CHA) Children's Hospitals CEOs Forum that a national approach for paediatric medication assessment should be developed. The National Paediatric Medicines Forum (NPMF) was established to identify shared issues and to develop and disseminate recommendations, including guidance for evaluating the clinical- and cost-effectiveness of high-cost medications. Through sharing experiences and skills, NPMF aimed to optimise consistency and quality in decision-making and equity of access. Tertiary paediatric services nominated members, including medical specialists and hospital pharmacists with expertise in clinical pharmacology, health economics, quality use of medicines and/or clinical ethics. Medications for evaluation were chosen via horizon scanning issues identified by regional DTCs, including high cost and/or novel drugs with limited evidence in paediatric patients. Since June 2019, 11 meetings with 16 members have been held by videoconference. Amongst a range of topics discussed, 10 drugs/classes have been reviewed in detail including vedolizumab, ranibizumab, burosumab, trametinib, canakinumab, eltrombopag, romiplostim, palivizumab, Glucagon-like peptide 1 (GLP-1) analogues and melatonin. Here, ‘medication summaries’ were developed, following a survey of their usage in paediatrics across Australia (including indications and local restrictions), systematic review of the literature and economic analysis modelling for financial implications. Consensus recommendations for the indications, dosage and endpoints for monitoring the effect were proposed and discussed. Guests with subspecialist expertise contributed as required, including the fields of neonatology, ophthalmology, gastroenterology and oncology. Feedback was also sought from other subspecialists utilising professional networks. The interplay and challenges with Medicines Access Programs for innovative therapies in paediatrics were explored.3 The purpose of the medication summaries is to support both clinician and DTC decision-making in individual patients, contributing to the stated goals of the NPMF. Plain language summaries have been developed for some medications to engage patients and parents with the findings. Looking forward, the NPMF will update medication summaries as new information is available and develop summaries for other drugs. We will increase NPMF membership and clinician input. Medication summaries will be submitted for publication in peer-reviewed journals to invite further discussions, and other methods to broaden their dissemination will be explored. Research to measure the impact of the medication summaries on decision-making in practice is under development. A systematic approach to multisite data collection is anticipated to provide valuable clinical information on trends, usage and outcomes from the drugs, further contributing to the published literature; collaborations with clinical researchers will be invaluable. This collective clinical experience also offers an opportunity to be a voice of paediatric advocacy, including for submissions to the Pharmaceutical Benefits Advisory Committee (PBAC). We hope that these collective activities will make significant improvements in outcomes for paediatric patients.