Cultural, geographical and ethical questions when looking to enroll pediatric patients in rare disease clinical trials

Anzelewicz, Stefan; Garnier, Hanna; Rangaswami, Arun; Czauderna, Piotr

doi:10.1080/21678707.2017.1348293

Cited by 8 publications

(5 citation statements)

References 69 publications

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“…13,[41][42][43] Patients and/or their representatives become increasingly involved in addressing ethical issues, transparency in quality of care, safety standards, and contributing to research. 13,44,45 The involvement of representatives of patient support groups in this consensus conference allowed a unique perspective to all discussed aspects of patient care and management and emphasizes the benefits of collaboration as already proved in the past. 44,46…”

Section: Discussionmentioning

confidence: 97%

ERNICA Consensus Conference on the Management of Patients with Esophageal Atresia and Tracheoesophageal Fistula: Follow-up and Framework

et al. 2019

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Introduction Improvements in care of patients with esophageal atresia (EA) and tracheoesophageal fistula (TEF) have shifted the focus from mortality to morbidity and quality-of-life. Long-term follow-up is essential, but evidence is limited and standardized protocols are scarce. Nineteen representatives of the European Reference Network for Rare Inherited Congenital Anomalies (ERNICA) from nine European countries conducted a consensus conference on the surgical management of EA/TEF. Materials and Methods The conference was prepared by item generation (including items of surgical relevance from the European Society for Pediatric Gastroenterology Hepatology and Nutrition (ESPGHAN)-The North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) guidelines on follow-up after EA repair), item prioritization, formulation of a final list containing the domains Follow-up and Framework, and literature review. Anonymous voting was conducted via an internet-based system. Consensus was defined as ≥75% of those voting with scores of 6 to 9. Results Twenty-five items were generated in the domain Follow-up of which 17 (68%) matched with corresponding ESPGHAN-NASPGHAN statements. Complete consensus (100%) was achieved on seven items (28%), such as the necessity of an interdisciplinary follow-up program. Consensus ≥75% was achieved on 18 items (72%), such as potential indications for fundoplication. There was an 82% concordance with the ESPGHAN-NASPGHAN recommendations. Four items were generated in the domain Framework, and complete consensus was achieved on all these items. Conclusion Participants of the first ERNICA conference reached significant consensus on the follow-up of patients with EA/TEF who undergo primary anastomosis. Fundamental statements regarding centralization, multidisciplinary approach, and involvement of patient organizations were formulated. These consensus statements will provide the cornerstone for uniform treatment protocols and resultant optimized patient care.

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Section: Discussionmentioning

confidence: 97%

ERNICA Consensus Conference on the Management of Patients with Esophageal Atresia and Tracheoesophageal Fistula: Follow-up and Framework

et al. 2019

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“…Original article community support networks and (i) promote transparent communication to mitigate inherent inequities in rare disease care and research [6,8,11,[20][21][22][23].…”

Section: Discussionmentioning

confidence: 99%

Parents’ expectations regarding case management for rare diseases in Switzerland: mixed-method findings from an online survey

Von Mengershausen,

Streuli

2024

Swiss Med Wkly

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AIMS OF THE STUDY: This pilot study aims to enhance understanding by examining parents’ specific views on the requirements, content and objectives of case management and advanced care coordination for children with rare diseases during childhood. The findings of this study are expected to offer valuable insights and recommendations for existing and future initiatives in clinical practice and research, with the goal of improving the comprehensive, child-centred and family-orientated approach to case management. METHODS: This pilot study is part of an ongoing prospective study (SPACE), involving parents and families from various networks in Switzerland. Participants were parents recruited from the Children with Rare Diseases (KMSK) network consisting of families with children with rare diseases. The survey questionnaire covered demographic information; expectations and perceived need for case management; assessment of their quality of life and their child’s suffering; and evaluation of interprofessional and interdisciplinary communication. Qualitative data from free-response answers were analysed using Mayring’s content analysis and descriptive statistics were used to analyse quantitative data from Likert-scale questions. RESULTS: The study included 108 respondent families from among the 775 in the KMSK, a 14% response rate. The age of their children ranged from 0.4 to 24 years (mean: 8) and their level of suffering in the past six months varied, with 31.5% indicating intense or very intense suffering. In terms of case management, 15.8% of families reported access while 32.4% expressed a need but did not have access to it. The study identified three categories of parental expectations regarding case management, emphasising the importance of interprofessional collaboration, effective communication and comprehensive support. CONCLUSIONS: The findings shed light on the high need for case management support with a current undersupply in Switzerland and an association with reduced parental quality of life, highlighting the necessity for diverse support and assistance to effectively manage the challenges faced by families with children with rare diseases.

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“…Therefore, it is possible that the dropout at the time of study enrollment may have been attributable to other trial factors, such as strict inclusion or exclusion criteria. In cases where an overly restrictive trial design limits the pool of participants, no amount of targeted social media can solve accrual challenges; however, in the case of rare disease research, potential participants have very limited access to opportunities, and social media presents a platform for sharing trials with the target audiences [ 42 ].…”

Section: Discussionmentioning

confidence: 99%

Direct-to-Consumer Recruitment Methods via Traditional and Social Media to Aid in Research Accrual for Clinical Trials for Rare Diseases: Comparative Analysis Study

Applequist¹,

Burroughs²,

Merkel³

et al. 2023

J Med Internet Res

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Background Recruitment into clinical trials is a challenging process, with as many as 40% of studies failing to meet their target sample sizes. The principles of direct-to-consumer (DTC) advertising rely upon novel marketing strategies. The ability to reach expansive audiences in the web-based realm presents a unique opportunity for researchers to overcome various barriers to enrollment in clinical trials. Research has investigated the use of individual web-based platforms to aid in recruitment and accrual into trials; however, a gap in the literature exists, whereby multiple mass communication platforms have yet to be investigated across a range of clinical trials. Objective There is a need to better understand how individual factors combine to collectively influence trial recruitment. We aimed to test whether DTC recruitment of potentially eligible study participants via social media platforms (eg, Facebook [Meta Platforms Inc] and Twitter [Twitter Inc]) was an effective strategy or whether this acted as an enhancement to traditional (eg, email via contact registries) recruitment strategies through established clinical research sites. Methods This study tested multiple DTC web-based recruitment efforts (Facebook, Twitter, email, and patient advocacy group [PAG] involvement) across 6 national and international research studies from 5 rare disease consortia. Targeted social media messaging, social media management software, and individual study websites with prescreening questions were used in the Protocol for Increasing Accrual Using Social Media (PRISM). Results In total, 1465 PRISM website referrals occurred across all 6 studies. Organic (unpaid) Facebook posts (676/1465, 46.14%) and Rare Diseases Clinical Research Network patient contact registry emails (461/1465, 31.47%) represented the most successful forms of engagement. PRISM was successful in accumulating a 40.1% (136/339) lead generation (those who screened positive and consented to share their contact information to be contacted by a clinical site coordinator). Despite the large number of leads generated from PRISM recruitment efforts, the number of patients who were subsequently enrolled in studies was low. Across 6 studies, 3 participants were ultimately enrolled, meaning that 97.8% (133/136) of leads dropped off. Conclusions The results indicate that although accrual results were low, this is consistent with previously documented challenges of studying populations with rare diseases. Targeted messaging integrated throughout the recruitment process (eg, referral, lead, and accrual) remains an area for further research. Key elements to consider include structuring the communicative workflow in such a way that PAG involvement is central to the process, with clinical site coordinators actively involved after an individual consents to share their contact information. Customized approaches are needed for each population and research study, with observational studies best suited for social media recruitment. As evidenced by lead generation, results suggest that web-based recruitment efforts, coupled with targeted messaging and PAG partnerships, have the potential to supplement clinical trial accrual.

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Cultural, geographical and ethical questions when looking to enroll pediatric patients in rare disease clinical trials

Cited by 8 publications

References 69 publications

ERNICA Consensus Conference on the Management of Patients with Esophageal Atresia and Tracheoesophageal Fistula: Follow-up and Framework

ERNICA Consensus Conference on the Management of Patients with Esophageal Atresia and Tracheoesophageal Fistula: Follow-up and Framework

Parents’ expectations regarding case management for rare diseases in Switzerland: mixed-method findings from an online survey

Direct-to-Consumer Recruitment Methods via Traditional and Social Media to Aid in Research Accrual for Clinical Trials for Rare Diseases: Comparative Analysis Study

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