Model‐Based Assessment of Alternative Study Designs in Pediatric Trials. Part II: Bayesian Approaches

Smania, Giovanni; Baiardi, Paola; Ceci, Adriana; Cella, Massimo; Magni, Paolo

doi:10.1002/psp4.12092

Cited by 9 publications

(9 citation statements)

References 16 publications

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“…The merged distribution is called the posterior distribution and is used for data analysis. 36,37 In order to apply this approach, one has to assume that the historical data used to create the assumed distribution is applicable to the study population. One way of applying Bayesian statistics to pediatric trials is using data from uncontrolled pediatric studies or from adult studies as historical data.…”

Section: Discussionmentioning

confidence: 99%

Oral dexamethasone for the prevention of acute migraine recurrence in pediatric patients presenting to the emergency department with migraine

Orr

Richer

Barrowman

et al. 2018

Cephalalgia Reports

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Objective: To assess the feasibility of a randomized controlled trial protocol that aims to determine the efficacy and safety of oral dexamethasone compared to placebo for the prevention of migraine recurrence in children and adolescents visiting the pediatric emergency department (ED) with migraine. Methods: This study was a two-arm, parallel-group, randomized, placebo-controlled, double-blind pilot trial of patients presenting to the pediatric ED with migraine. Eligible participants were randomized at 1:1 ratio to receive either oral dexamethasone 0.6 mg/kg (maximum 15 mg) or matched placebo as a single dose. Efficacy and safety outcomes were assessed at discharge, 48 h and 7 days after discharge. The primary outcome of the trial was feasibility and was assessed through participant recruitment rate, follow-up completion rates, participant satisfaction ratings and comparison of enrolled versus non-enrolled participants. Efficacy and safety outcomes were not analyzed given that this was a pilot study. Results: Twelve participants were enrolled over the 6-month recruitment period. This represents 60% of the planned sample size and a 10.5% recruitment rate. No other feasibility issues were identified and patients expressed high satisfaction rates with their treatment: 90.9% were satisfied with their treatment at discharge and at 48-h follow-up and 81.8% were satisfied with their treatment at 7-day follow-up (81.8%). There were no significant differences observed when comparing enrolled participants to those not enrolled. Conclusion: This pilot randomized controlled trial is the first to assess dexamethasone in the pediatric ED for the prevention of migraine recurrence. The protocol is feasible but recruitment in a single center was lower than expected. Future pediatric ED migraine studies may use innovative or pragmatic trial designs to maximize feasibility from a recruitment standpoint.

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Section: Discussionmentioning

confidence: 99%

Oral dexamethasone for the prevention of acute migraine recurrence in pediatric patients presenting to the emergency department with migraine

Orr

Richer

Barrowman

et al. 2018

Cephalalgia Reports

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“…I-ACT and PTN share a goal of providing expert advice on CT design and conduct for drugs and medical devices in children. The Global Research in Pediatrics (GRiP) Network of Excellence, funded by the European Union has developed guidance and research tools for pediatric studies ( 24 , 25 , 26 , 27 , 28 ) that can be found in their publication repository ( http://www.grip-network.org/index.php/cms/en/publications ). The advantages of these international partnerships include harmonization, development of best research practice, and a global union of expertise and experience in pediatric clinical research.…”

Section: Current Regulatory Guidancementioning

confidence: 99%

Useful pharmacodynamic endpoints in children: selection, measurement, and next steps

Kelly

Sinha²,

Barker

et al. 2018

Pediatr Res

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Pharmacodynamic (PD) endpoints are essential for establishing the benefit-to-risk ratio for therapeutic interventions in children and neonates. This article discusses the selection of an appropriate measure of response, the PD endpoint, which is a critical methodological step in designing pediatric efficacy and safety studies. We provide an overview of existing guidance on the choice of PD endpoints in pediatric clinical research. We identified several considerations relevant to the selection and measurement of PD endpoints in pediatric clinical trials, including the use of biomarkers, modeling, compliance, scoring systems, and validated measurement tools. To be useful, PD endpoints in children need to be clinically relevant, responsive to both treatment and/or disease progression, reproducible, and reliable. In most pediatric disease areas, this requires significant validation efforts. We propose a minimal set of criteria for useful PD endpoint selection and measurement. We conclude that, given the current heterogeneity of pediatric PD endpoint definitions and measurements, both across and within defined disease areas, there is an acute need for internationally agreed, validated, and condition-specific pediatric PD endpoints that consider the needs of all stakeholders, including healthcare providers, policy makers, patients, and families.

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“…Some investigators have found that many factors such as protocol approval by sites, limited knowledge of methodology, lack of in-depth understanding of child physiology, psychology, the social embedding of children may hinder the use of innovative methods, 24 but experience is growing. [25][26][27][28] Networks can contribute to the design of trials in a number of ways, including registries 29 and modelling. 30,31 Trial design depends on understanding the clinical context of the trial: "trial designs, no matter how novel, will only be as good as the knowledge that underlies them."…”

Section: Optimal Design Of Studiesmentioning

confidence: 99%

Roles of Clinical Research Networks in Pediatric Drug Development

Turner

Attar

Wildt

et al. 2017

Clinical Therapeutics

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The evaluation of drugs that are used in children has been neglected historically but is now well established as an essential part of clinical drug development. The increase in pediatric activity among industry, and other sectors, has highlighted the importance of joint working. All participants in pediatric drug development need to be aware of the "big picture." An increasingly important part of this big picture in pediatrics, as in other populations, is the design and conduct of clinical trials in networks. This narrative review provides an overview of the roles of clinical research networks in pediatric drug development. Networks take many forms as specialty networks and geographic networks but work toward common principles, including sharing resources between trials, and using experience with trial conduct to improve trial design. Networks develop standardized processes for trial conduct (including performance management) that increase the speed and predictability of trial conduct while reducing burdens on sites, sponsors, and intermediaries. Networks can provide validated, real-world information about natural history, participant distribution, and standards of care to inform planning of development programs, including extrapolation and clinical trial simulation. Networks can work across geographic and jurisdictional barriers to promote global interoperability of drug development. Networks support participant centrality. Networks offer an opportunity to develop relationships with investigators, sites, and methodological experts that span pre-competitive foundations for drug development and specific products. Sustainable networks benefit all stakeholders by providing a multifunctional platform that promotes the quality and timeliness of clinical drug development.

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Model‐Based Assessment of Alternative Study Designs in Pediatric Trials. Part II: Bayesian Approaches

Cited by 9 publications

References 16 publications

Oral dexamethasone for the prevention of acute migraine recurrence in pediatric patients presenting to the emergency department with migraine

Oral dexamethasone for the prevention of acute migraine recurrence in pediatric patients presenting to the emergency department with migraine

Useful pharmacodynamic endpoints in children: selection, measurement, and next steps

Roles of Clinical Research Networks in Pediatric Drug Development

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