Role of Patients and Parents in Pediatric Drug Development

Tsang, Vivian; West, Leanne L.; Woods, Christine; Koh, Chester J.; McCune, Susan K.; Mullin, Theresa; Smith, Sharon R.; Gaillard, Ségolène; Claverol, Joana; Nafría, Begonya; Preston, Jennifer; Dicks, Pamela; Thompson, Charles A.

doi:10.1177/2168479018820875

Cited by 24 publications

(20 citation statements)

References 39 publications

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“…In contrast to common hospital-based administrative youth councils or teen advisory boards, the push towards youth participation in research specifically has resulted in both formal and informal partnerships between pharmaceutical industry and hospitals across the globe [ 22 ]. While there is need for public-private collaborations, and engagement of youth for national and international trials, there is a lack of standardized protocols for the development and training of YPAGs nor any quantifiable way to measure impact and adoption of youth opinion into upcoming or ongoing clinical trials and research.…”

Section: Discussionmentioning

confidence: 99%

Facilitators and barriers to the training and maintenance of young persons’ advisory groups (YPAGs)

Tsang

Chew

Junker

2020

International Journal of Pediatrics and Adolescent Medicine

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Introduction Increasing demands from public and private healthcare coupled with national initiatives in patient-oriented research has led to an increase in avenues to allow patients to be directly involved in research. In particular, the push towards participation of children and youth has resulted in the formation of pediatric patient advisory groups with broad partnerships and consultation requests across the globe. However, there is a lack of evidence to examine the challenges in formation and training of young persons’ advisory groups (YPAGs) and management processes required thereafter. Purpose and objectives This study’s purpose is to document YPAG formation and training protocols around the world, highlight common strengths, and evaluate pitfalls and challenges. The results from this study will subsequently inform the development of standardized training protocols for children and youth to be piloted globally. Methods In this study, 17 select YPAG team leaders from 7 countries were surveyed to determine current training techniques used within existing groups. 17 youth representatives and 16 team leaders were then interviewed to gather further qualitative data on facilitators and barriers that aid or prevent successful initiation and maintenance of these groups. Qualitative interview data was coded and analyzed using NVivo by two independent reviewers (SYC, VWLT). Any inconsistencies in thematic analysis was confirmed by a third reviewer (JB). Results The most common training topics include consent and assent (64.71%), clinical trials (64.71%), and patient safety (70.59%). There are significant discrepancies to the amount of training received by each team. Most YPAGs out of the 17 groups receive no formal training (58.82%) while training sessions in the remaining 7 groups vary in both duration and frequency. Collectively, meetings ranged from 15  minutes to 6  hours long, with the majority of team meetings being 2–3  hours long (58.82%). The most common training facilitators are a positive relationship with a local hospital (82.35%) and access to a dedicated team coordinator (64.71%). 70.59% of team leaders identified a lack of access to appropriate educational materials available as a drawback to the impact of their YPAG, making this the greatest common barrier. Conclusion Bringing children and youth to the forefront of paediatric trials and clinical research facilitates appropriate patient representation in subsequent research decision-making. There is an urgency to create and implement standardized protocols for the training of children and youth, especially in preparation for national and international research consultations. This low barrier framework may be of special interest to lower-middle-income countries who wish to encourage community participation in healthcare.

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Section: Discussionmentioning

confidence: 99%

Facilitators and barriers to the training and maintenance of young persons’ advisory groups (YPAGs)

Tsang

Chew

Junker

2020

International Journal of Pediatrics and Adolescent Medicine

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“…In 2017, I‐ACT for Children was initiated as a nonprofit using public‐private collaboration intended to optimize pediatric study designs, protocols, best practices, training, and engagement of patients and parents . Increasingly, patients and their families, as well as advocacy organizations, are engaging with physicians, sponsors, and regulators to advance quality and timeliness of pediatric clinical drug development …”

Section: Experience With Pk‐pd Modeling and Research Areas Going Forwardmentioning

confidence: 99%

“…52 Increasingly, patients and their families, as well as advocacy organizations, are engaging with physicians, sponsors, and regulators to advance quality and timeliness of pediatric clinical drug development. [53][54][55][56]…”

Section: Experience With Pk-pd Modeling and Research Areas Going Forwardmentioning

confidence: 99%

Developmental Pharmacodynamics and Modeling in Pediatric Drug Development

Conklin

Hoffman

Anker

2019

The Journal of Clinical Pharma

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Challenges in pediatric drug development include small patient numbers, limited outcomes research, ethical barriers, and sparse biosamples. Increasingly, pediatric drug development is focusing on extrapolation: leveraging knowledge about adult disease and drug responses to inform projections of drug and clinical trial performance in pediatric subpopulations. Pharmacokinetic‐pharmacodynamic (PK‐PD) modeling and extrapolation aim to reduce the numbers of patients and data points needed to establish efficacy. Planning for PK‐PD and biomarker studies should begin early in the adult drug development program. Extrapolation relies on the assumption that both the underlying disease and the mechanism of action of the drug used to treat that disease are similar in adults and pediatric subpopulations. Clearly, developmental changes in PK and PD need to be considered to enhance the quality of PK‐PD modeling and, therefore, increase the success of extrapolation. This article focuses on the influence of differences in PD between adults and pediatric subpopulations that are highly relevant for the use of extrapolation.

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“…There are many examples of involvement of children and parents in research . Patient engagement is, for example, arranged via members of the Children's Advisory Network and Young Persons Advisory Groups (YPAGs) with specific tools for involvement …”

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confidence: 99%

“…8 Patient engagement is, for example, arranged via members of the Children's Advisory Network and Young Persons Advisory Groups (YPAGs) 9 with specific tools for involvement. 10 In the United States, engagement (in general, not specific for pediatrics) is attained and promoted in (for example) Patient-Centered Outcomes Research Institute (PCORI) in Boston (https ://www.pcori.org/about-us/our-progr ams/engag ement/ public-and-patie nt-engag ement/ engag ement-resou rces#conte nt-4029). In Canada, patient engagement in research (in general) is promoted via Strategy for Patient-Oriented Research (SPOR; (http://www.cihr-irsc.gc.ca/e/41204.html)) and (http://www.…”

mentioning

confidence: 99%