Implications of Pediatric Initiatives on CNS Drug Development for All Ages-2020 and Beyond: Second of Three Sets of Expanded Proceedings from the 2020 ISCTM Autumn Conference on Pediatric Drug Development.

This article expands upon a session, titled "Implications of Pediatric Initiatives on CNS Drug Development for All Ages-2020 and Beyond," that was presented as part of a two-day meeting on pediatric drug development at the International Society for Central Nervous System (CNS) Clinical Trials and Methodology (ISCTM) Autumn Conference in Boston, Massachusetts, in October 2020. Speakers from various areas of pediatric drug development addressed a variety of implications of including children in drug development programs. The speakers wrote summaries of their talks, which are included here.

Toward patient-centered treatment goals for duchenne muscular dystrophy: insights from the "Your Voice" study.

Background: Patient-centered research has emerged as critically important for understanding the impact of treatments on key stakeholders. The subjective experience of quality of life (QOL) is increasingly recognized as fundamental to delineating treatment goals. The present study utilized content analysis of qualitative data and quantitative analysis to highlight important domains of disease burden and underlying reasons for their importance, and to characterize goals for new treatments for Duchenne Muscular Dystrophy (DMD).

Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy.

Background: Patients with Duchenne muscular dystrophy (DMD) adopt compensatory movement patterns as muscles weaken. The Duchenne Video Assessment (DVA) measures patient ease of movement through identification of compensatory movement patterns. The DVA directs caregivers to video record patients performing specific movement tasks at home using a secure mobile application, and DVA-certified physical therapists (PTs) score the videos using scorecards with prespecified compensatory movement criteria.

Evaluation of a General Practitioner with Special Interest model: lessons learned from staff experiences.

Background: The integration of general practitioners into specialist outpatient clinics is associated with improved access to care; however, little is understood about the organisation-level factors that affect successful implementation. We aimed to identify factors that were facilitators or barriers to the implementation of a General Practitioner with Special Interest (GPwSI) model of care across a range of specialties.

Methods: Semi-structured, in-depth interviews were conducted with 25 stakeholders at 13 GPwSI clinics in operation within a Queensland public health service.

Adapting traditional content validation methods to fit purpose: an example with a novel video assessment and training materials in Duchenne muscular dystrophy (DMD).

Purpose: The Duchenne muscular dystrophy (DMD) Video Assessment requires a caregiver to record six videos of their ambulatory child performing physical activities using a smartphone application (app). Innovative assessments that combine a performance measure, technology, and an observer require new approaches to content validation studies. This study presents a novel approach to establish the feasibility and content validity of the Video Assessment and usability of the app.