Dataset Information

Brief Report: Assessment Experiences of Children with Neurogenetic Syndromes: Caregivers' Perceptions and Suggestions for Improvement.

ABSTRACT: It is well-recognized that measurement options for diagnosing and monitoring children with neurogenetic syndromes (NGS) associated with moderate to severe intellectual impairment are limited (Berry-Kravis, Dev Med Child Neurol https://doi.org/10.1111/dmcn.13018, 2016), and caregivers experience significant concerns regarding the assessment process. However to date, these concerns have not been summarized into actionable steps for clinicians and test-makers. As such, we used a mixed methods approach to assess caregiver-derived perceptions and suggestions for improving assessments in NGS. Results indicated many shared challenges and suggestions for improvement, particularly in the domains of testing procedures and examiner communication. Integrating these suggestions into future protocols is an important next step toward improving the quality of assessment procedures for children with NGS and their families across both clinical and research contexts.

SUBMITTER: Kelleher B

PROVIDER: S-EPMC8136762 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature

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Brief Report: Assessment Experiences of Children with Neurogenetic Syndromes: Caregivers' Perceptions and Suggestions for Improvement.

Kelleher Bridgette B Halligan Taylor T Garwood Tessa T Howell Samantha S Martin-O'Dell Breanna B Swint Amber A Shelton Liberty-Ann LA Shin Joey J

Journal of autism and developmental disorders 20200401 4

It is well-recognized that measurement options for diagnosing and monitoring children with neurogenetic syndromes (NGS) associated with moderate to severe intellectual impairment are limited (Berry-Kravis, Dev Med Child Neurol https://doi.org/10.1111/dmcn.13018, 2016), and caregivers experience significant concerns regarding the assessment process. However to date, these concerns have not been summarized into actionable steps for clinicians and test-makers. As such, we used a mixed methods appro ...[more]

PMID: 31955311

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Project description:ImportanceThe manufacturing and marketing of medical devices is regulated by the US Food and Drug Administration (FDA), and the FDA premarket approval (PMA) process evaluates the safety and effectiveness of medical devices. The PMA process includes a detailed scientific, regulatory and quality system review and is critical to ensure that novel devices are safe, effective, and meet the needs of patients.ObjectiveTo survey current voting members serving on panels of the FDA's Medical Devices Advisory Committee to better characterize panel decision-making and identify steps for improvement.Design, setting, and participantsThis qualitative survey study included 36 questions that were mailed to FDA device panelists regarding their opinions on the influence of sources of information, pivotal trial design, quality of evidence, panel composition and internal deliberative process, time allocation, and impartiality of the FDA. The survey was mailed to the members of all 18 FDA device panels in January and February 2017. Data were collected from January to May 2017 and analyzed from 2018 to 2019.ExposuresRespondents read and returned the aforementioned paper survey, while nonrespondents did not.Main outcomes and measuresThe main outcomes included panel members' perceptions, and their implications for process improvement. χ2 or Fisher exact tests were used to test differences between subgroups.ResultsOf 64 of 92 panel members who responded (69.6%), 38 of 64 (59.4%) were male, 3 of 63 (4.8%) were Black respondents, 46 of 63 (73.0%) were White respondents, and 36 of 60 (60.0%) were in academic practice. The mean (range) panel service was 6.8 (1-22) years with 3.9 (1-19) meetings attended. Overall, respondents considered information presented by the FDA unbiased, and 28 of 61 (45.9%) believed that pivotal trials were frequently well-designed, 55 of 62 respondents (88.7%) suggested FDA consult panel members preemptively regarding trial design and 54 of 64 (84.4%) regarding the device label. Most indicated that prior FDA approval of another device serving the same medical purpose (43 of 62 [69.4%]) or approval in other countries with comparable regulatory regimes, such as Canada and Europe (39 of 62 [62.9%]), would make them more likely to recommend approval. Respondents rated written information (50 of 60 [83.3%]), live presentations (43 of 58 [74.1%]), and prior professional knowledge (41 of 60 [68.3%]) as the most important sources of information in deciding whether to recommend approval. Additionally, 52 of 58 respondents (89.7%) recommended that a panel member-only executive session would allow more clarity and honesty in deliberations, and 33 of 59 (55.9%) believed a three-fourths majority appropriate for recommending approval, which would be a deviation from the current system in which an overall vote is reported without designation of a vote threshold.Conclusions and relevanceIn this survey study of FDA device panel members, respondents wanted improved study designs, more relevant clinical data, including from other countries, involvement of panelists in study design and device label development, and inclusion of an executive session. Demographically, panels could be made more diverse.

Dataset Information

Brief Report: Assessment Experiences of Children with Neurogenetic Syndromes: Caregivers' Perceptions and Suggestions for Improvement.

Publications

Brief Report: Assessment Experiences of Children with Neurogenetic Syndromes: Caregivers' Perceptions and Suggestions for Improvement.

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