Making it easier to ‘choose wisely’ (original) (raw)
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Genetics in Medicine, 2010
The Secretary's Advisory Committee on Heritable Disorders in Newborns and Children is charged with evaluating conditions nominated for addition to the uniform screening panel and consequently making recommendations to the secretary of the US Department of Health and Human Services. This report describes the framework by which the committee approaches its task. Key decision nodes include initial review of every nomination to determine whether conditions are amenable for systematic evidence review, review of systematic evidence reviews conducted by the committee's external review group, and deliberation and formal recommendation for addition or exclusion to the uniform panel. Data analyzed include the accuracy and specificity of screening and diagnostic tests for nominated disorders, the extent of predicted health benefits, harms impact on disease course, and cost from early diagnosis and treatment. The committee process is guided by approaches used by similar entities, but more flexible criteria are sometimes needed to accommodate data limitations stemming from the rarity of many of these conditions. Possible outcomes of committee review range from recommendation to add a nominated condition to the uniform panel; provide feedback on specific gaps in evidence that must be addressed before making a decision; or rejection of a nomination (e.g., because of identified harms). The committee's structured evidence-based assessment of nominated conditions supports a consistently rigorous, iterative and transparent approach to its making recommendations regarding broad population-based screening programs for rare conditions in infants and children. Genet Med 2010:12(3):153-159.
Innovative approaches to investigator-initiated, multicentre paediatric clinical trials in Canada
BMJ Open, 2019
Data from clinical trials are needed to guide the safe and effective use of medicines in children. Clinical trials are challenging to design and implement in all populations, and children present additional considerations. Several regions including the UK, USA and Europe have established clinical trial infrastructure to capitalise on expertise and promote clinical trials enrolling children. Our objective is to describe the partnerships and operational considerations for the development of paediatric clinical trials infrastructure in Canada. We describe the design and conduct of four emergency room paediatric trials, with four separate sponsors, across four provinces in parallel. Operations discussed include multisite contract development, centralised risk-based data monitoring, ethical review and patient engagement. We conclude with lessons learnt, additional challenges and potential solutions to facilitate drug development for children in Canada.
Journal of Hospital Medicine, 2019
BACKGROUND: The Choosing Wisely® Campaign (CWC) was launched in 2012. Five recommendations to reduce the use of “low-value” services in hospitalized children were published in 2013. OBJECTIVES: The aim of this study was to estimate the frequency and trends of utilization of these services in tertiary children’s hospitals five years before and after the publication of the recommendations. METHODS: We conducted a retrospective, longitudinal analysis of hospitalizations to 36 children’s hospitals from 2008 to 2017. The “low-value” services included (1) chest radiograph (CXR) for asthma, (2) CXR for bronchiolitis, (3) relievers for bronchiolitis, (4) systemic steroids for lower respiratory tract infection (LRTI), and (5) acid suppressor therapy for uncomplicated gastroesophageal reflux (GER). We estimated the annual percentages of the use of these services after risk adjustment, followed by an interrupted time series (ITS) analysis to compare trends before and after the publication of t...
Standard 5: Selection, Measurement, and Reporting of Outcomes in Clinical Trials in Children
PEDIATRICS, 2012
People making choices in health care should use the findings of clinical trials (ideally, incorporated in systematic reviews) to inform their decisions. If these findings are to be useful and reliable, researchers must select appropriate outcomes, measure them in a scientifically robust manner, and report results thoroughly. There are difficulties, however, relating to the selection and measurement of outcomes in clinical trials, and special considerations are needed when these studies are conducted in children. This article provides guidance for researchers working on clinical trials in children. Although this article is focused on trials of effectiveness, which are similar to therapeutic confirmatory trials, certain sections may also relate to efficacy trials, pharmacokinetic trials, therapeutic exploratory trials, and trials conducted earlier in drug development. SELECTION OF APPROPRIATE OUTCOMES To be useful, clinical trials that evaluate potential benefits and harms of health care interventions must measure outcomes of relevance to practitioners and patients who make shared decisions about treatment options; regulatory authorities who consider applications for marketing authorizations for medicines; organizations who decide whether to provide funding for an intervention (eg, health care commissioners, insurance companies); and policy makers interested in the impact of an intervention.
Archives of Disease in Childhood, 2021
Conduct of clinical trials in babies, children and young people is often hindered by issues that could have been foreseen before the trial opened; that is, some clinical trials are often underprepared. In order to identify a good approach to trial preparedness, the European Network of Paediatric Research at the European Medicines Agency formed a working group. The Working Group included representation from regulators, industry, academics, paediatric clinical research networks and parents. The Working Group consulted widely about how to prepare for paediatric clinical trials. The Group’s detailed recommendations have been published (https://www.ema.europa.eu/en/documents/other/preparedness-medicines-clinical-trials-paediatrics-recommendations-enpr-ema-working-group-trial\_en.pdf). This paper is a summary of the key recommendations including the following: start early, preferably in parallel to designing the medicine’s development plan and individual protocols; identify the rationale a...