Madlen Gazarian

The status of paediatric medicines initiatives around the world—what has happened and what has not?

PurposeThis review was conducted to examine the current status of paediatric medicines initiatives across the globe.MethodsThe authors made a non-systematic descriptive review of current world situation.ResultsTwo regions, the United States (US) and the European Union (EU), and the World Health Organization (WHO) have introduced strong paediatric initiatives to improve children’s health through improving access to better paediatric medicines. The experience from the US initiative indicates that it is possible to stimulate development and study of paediatric medicines and provide important new information for improvement of paediatric therapy. The early results from the EU initiative are similarly encouraging. In Canada, Japan, Australia and other developed countries, specific paediatric medicines initiatives have been less extensive and weaker, with modest results. Disappointingly, current evidence suggests that results from clinical trials outside the US often do not benefit children in the country in which the trials were largely conducted. Pharmaceutical companies that have derived a financial benefit commensurate with the cost of doing the paediatric trials in one country do not seem to be making the results of these trials available to all countries if there is no financial incentive to the company. The WHO campaign ‘make medicines child size’ has produced substantive accomplishments in building improved foundations to improve mechanisms that will enhance children’s access to critical medicines in resource-limited settings. However, practically all of this work has been performed using an amalgamation of short-term funding from a variety of sources as opposed to a sustained, programmatic commitment.ConclusionsAlthough much still needs to be done, it’s clear that with concerted efforts and appropriate resources, change is possible but slow. Retaining and fostering public and political interest in paediatric medicines is challenging, but pivotal for success.

Child and parent satisfaction with the use of spacer devices in acute asthma

Objective:  To evaluate child and parent satisfaction with the use of spacers in acute asthma.

Long-term Reduction in Adverse Drug Events: An Evidence-Based Improvement Model

OBJECTIVES: To develop and test an evidence-based model for reducing medication errors and harm in hospitalized children. METHODS: Prospective interrupted time series study evaluating the effectiveness of a multifaceted, staged intervention over 4 years in a major urban pediatric referral hospital. Guidelines for safe pediatric prescribing were implemented by using an evidence-based model. Key components included early clinician engagement and improved multidisciplinary communication, consensus development, interactive education, and timely data feedback by using iterative Plan-Do-Study-Act cycles. Impact on medication error and harm (adverse drug events, [ADEs]) was measured by using standard definitions and a multimethod approach. Prospective data from voluntary reports by nursing, medical, and pharmacy staff and intensive chart review were combined. All data were reviewed by a multidisciplinary panel, including causality assessments for ADEs. RESULTS: Reviewed over 3 time periods were 1011 patients with 6651 medication orders. Total ADEs decreased by > 50% in the first year and this was maintained at 4 years. Greatest improvements were in potential ADEs, which decreased from 12.26 per 100 patients at baseline to 4.60 per 100 patients at 4 years (P < .05). Total medication errors decreased from 4.51 per 100 orders at baseline to 2.78 per 100 orders at 4 years (P < .05). Prescribing errors decreased by 65%, from 4.07 per 100 orders at baseline to 2.05 orders at 4 years (P < .05). CONCLUSIONS: A multifaceted, evidence-based model for safe prescribing guideline implementation, engaging multidisciplinary clinicians, was effective in reducing medication error and harm in hospitalized children, resulting in sustained long-term improvement.

Delivering Better Medicines to Children

We are experiencing an exciting and unprecedented period in the history of children’s medicines globally. Milestone developments unfolding in recent years include the formation of the International Alliance for Better Medicines for Children in 2006 and landmark initiatives by the EU, the World Health Assembly (WHA), and the WHO in 2007. However, the challenges of optimizing the development, wider availability, and routine use of effective, safe, and affordable medicines addressing important child health needs are considerable. Each aspect of this continuum has so far received differential attention. Major initiatives in the US and EU have focused on stimulating research into children’s medicines, largely driven by drug regulatory reforms, but with important gaps remaining. Many countries are lacking similar reforms, so the benefits of these initiatives are currently not well reflected in the rest of the world. A systematic approach to knowledge translation to improve use of best evidence and deliver quality use of medicines (QUM) to children routinely has also been largely a ‘missing link’ so far. The WHO’s Essential Medicines List for Children and related initiatives are addressing improving children’s access to needed medicines. Priority research gaps, especially in the developing world, are also being pursued by the WHO. However, in many countries, including developed nations such as Australia, the policy response to the WHA resolution on ‘Better Medicines for Children’ has been inconsistent or fragmented. A better integrated overall approach, linking global medicines research efforts to child health needs and actual medicines use is needed. International networking to support the conduct, synthesis, and rapid dissemination of pediatric medicines research will help close knowledge gaps at a global level. Harmonization of pediatric drug regulation will support this goal and facilitate improving access to needed medicines. Increasing research into and dissemination of effective strategies to promote QUM is an essential component to maximize return (in health benefits) on increased investment in medicines research. A greater focus on QUM should also help create demand for better evidence from clinicians. Delivering on the promise of better medicines for children, wherever in the world they may be, depends on achieving successful integration between the science, the policy, and the practice of pediatric medicines.

Changes in anticonvulsant prescribing for Australian children: implications for Quality Use of Medicines.

Aims:  The evidence‐base guiding choices between newer versus established anticonvulsants in children is limited. Inappropriate use exposes children to potentially ineffective and/or harmful medicines. Our objective is to describe recent anticonvulsant prescribing patterns in the Australian paediatric population, evaluating overall trends and extent of off‐label prescribing of newer agents.

Pediatric Drug Safety Signal Detection: A New Drug–Event Reference Set for Performance Testing of Data-Mining Methods and Systems

BackgroundBetter evidence regarding drug safety in the pediatric population might be generated from existing data sources such as spontaneous reporting systems and electronic healthcare records. The Global Research in Paediatrics (GRiP)–Network of Excellence aims to develop pediatric-specific methods that can be applied to these data sources. A reference set of positive and negative drug–event associations is required.ObjectiveThe aim of this study was to develop a pediatric-specific reference set of positive and negative drug–event associations.MethodsConsidering user patterns and expert opinion, 16 drugs that are used in individuals aged 0–18 years were selected and evaluated against 16 events, regarded as important safety outcomes. A cross-table of unique drug–event pairs was created. Each pair was classified as potential positive or negative control based on information from the drug’s Summary of Product Characteristics and Micromedex. If both information sources consistently listed the event as an adverse event, the combination was reviewed as potential positive control. If both did not, the combination was evaluated as potential negative control. Further evaluation was based on published literature.ResultsSelected drugs include ibuprofen, flucloxacillin, domperidone, methylphenidate, montelukast, quinine, and cyproterone/ethinylestradiol. Selected events include bullous eruption, aplastic anemia, ventricular arrhythmia, sudden death, acute kidney injury, psychosis, and seizure. Altogether, 256 unique combinations were reviewed, yielding 37 positive (17 with evidence from the pediatric population and 20 with evidence from adults only) and 90 negative control pairs, with the remainder being unclassifiable.ConclusionWe propose a drug–event reference set that can be used to compare different signal detection methods in the pediatric population.

Celecoxib use and overuse - too much 'celebration'?

Aim: To characterise indications for celecoxib use in a hospital population and to evaluate whether hospital prescribing of celecoxib was consistent with agreed hospital guidelines.

Drug development: The use of unlicensed/off-label medicines in pediatrics.

Received August 30, 201 From the Probiotics Re School of Medicine, J Medicine, Universit zUniversity of New S of Paediatrics and C Stellenbosch Univers Centre for Child Heal ment of Pediatrics, Pe University of Rome, R and Inborn Errors Pro MD. Address correspondence PhD, Juntendo Univer (e-mail: yamasiro@ju The authors report no co Copyright # 2012 by E Hepatology, and Nut Gastroenterology, He DOI: 10.1097/MPG.0b01

Training Pediatric Clinical Pharmacology and Therapeutics Specialists of the Future

In recent years there has been a rapid and marked increase in global recognition of the need for better medicines for children, with various initiatives being implemented at global and regional levels. These exciting developments are matched by recognition of the need to build greater capacity in the field of pediatric clinical pharmacology and therapeutics to help deliver on the promise of better medicines for children. A range of pediatric medicines researchers, educators, clinical therapeutics practitioners, and experts in drug evaluation, regulation, and broader medicines policy are needed on a larger scale, in both developed and developing world settings. The current and likely future training needs to meet these diverse challenges, the current realities of trying to meet such needs, and the opportunities for international networking to help meet future training needs are discussed from a global perspective.

Rational Use of Medicines (RUM) for Children in the Developing World: Current Status, Key Challenges and Potential Solutions

With Rational and Responsible Use of Medicines (RUM) patients receive medicines appropriate to their needs at lowest cost to them and their community. Activities, capabilities and existing health resources are aligned to these goals. Achieving RUM in the paediatric population is associated with special challenges, many of which are amplified in LMICs. This chapter provides examples of multiple contributing factors and outlines key challenges for delivering RUM. Many countries lack national medicines policies and the absence of child-specific policies in particular is a fundamental problem. Key obstacles include the lack of paediatric-specific information to inform decisions and to guide evaluation of medicines use and outcomes in this population; lack of paediatric-specific skills and knowledge amongst health care workers; lack of practical tools; and the presence of perverse financial incentives. The authors discuss potential solutions, with national policy recommendations specific to child healthcare, consistent with WHO’s general framework of core interventions.