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Family Perspectives on Clinical Research for Pediatric Multiple Sclerosis: Enhancing Equity
Leslie A Mandel1, Ellen O'Donnell2, Katia Canenguez2
1Regis College, Weston, MA, USA.
Insights
Families impacted by pediatric multiple sclerosis (MS) desire greater involvement in research. They support future clinical studies but emphasize clear relevance, reduced burdens, and attention to medication side effects and patient comfort.
Area of Science:
- Pediatric Neurology
- Clinical Trial Design
- Patient-Centered Research
Background:
- Federally mandated pediatric new drug trials lack sufficient family perspectives.
- Multiple sclerosis (MS) is a chronic condition requiring long-term medical management.
- Limited understanding of family priorities hinders pediatric MS research optimization.
Purpose of the Study:
- To gather family perspectives on research priorities in pediatric-onset MS.
- To identify optimal methods for future pediatric MS research studies.
- To inform the design of patient-centered clinical trials.
Main Methods:
- Focus groups were conducted with families affected by pediatric-onset MS.
- Participants were recruited from geographically diverse Network of Pediatric MS Centers.
- Inquiries covered healthcare experiences, clinical trial views, and outcome measures.
Main Results:
- Families strongly supported future clinical studies.
- Patients expressed concerns about experimental medications and disease impact on activities.
- Parents prioritized medication delivery, side effects, and minimizing child discomfort.
Conclusions:
- Explicitly stating study relevance and rationale is crucial for participation.
- Future studies should incorporate compensation, reduce participation burdens, and consider psychological impacts.
- Engaging families is essential to bridge the pediatric research gap in rare diseases like MS.
Abstract:
Pediatric new drug trials are federally mandated, but family perspectives in multiple sclerosis (MS) research are limited. Due to MS chronicity and long-term medical system involvement, we obtained family views on research priorities and optimized methods for future studies. Focus groups were convened with families impacted by pediatric-onset MS. Recruitment included those followed by the Network of Pediatric MS Centers, geographically disparate locations, and centers' voluntary election. Study questions included: healthcare experiences, clinical trials perspectives, cognitive/psychosocial/educational outcomes, disease course and disability accrual. All subjects supported future clinical studies. Patients highlighted contribution to knowledge base but were wary of experimental medication and disease-course impeding activities. Parents underscored medication delivery modalities, side-effects, and limiting children's discomfort. All wanted study relevance made explicit. Suggested future study design elements included: providing compensation, limiting assumptions regarding outcome linkages, understanding study-related psychological impacts, and reducing participation burdens. Rare disease research can assist general medicine diagnosis and referral. Variable study designs and explicit rationale may augment participation. Closing the pediatric research gap requires family engagement in the research process.
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