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Background and ObjectivesFacioscapulohumeral muscular dystrophy (FSHD) is a rare, debilitating disease characterized by progressive muscle weakness. MRI is a sensitive assessment of disease severity and progression. We developed a quantitative whole-body (WB) musculoskeletal MRI (WB-MSK-MRI) protocol analyzing muscles in their entirety. This study aimed to assess WB-MSK-MRI as a potential imaging biomarker providing reliable measurements of muscle health that capture disease heterogeneity and clinically meaningful composite assessments correlating with severity and more responsive to change in clinical trials.MethodsParticipants aged 18–65 years, with genetically confirmed FSHD1, clinical severity 2 to 4 (Ricci scale, range 0–5), and ≥1 short tau inversion recovery–positive lower extremity muscle eligible for needle biopsy, enrolled at 6 sites and were imaged twice 4–12 weeks apart. Volumetric analysis of muscle fat infiltration (MFI), muscle fat fraction (MFF), and lean muscle volume (LMV) in 18 (36 total) muscles from bilateral shoulder, proximal arm, trunk, and legs was performed after automated atlas-based segmentation, followed by manual verification. A WB composite score, including muscles at highest risk for progression, and functional cross-sectional composites for correlation with relevant functional outcomes including timed up and go (TUG), FSHD-TUG, and reachable workspace (RWS), were developed.ResultsSeventeen participants enrolled in this study; 16 follow-up MRIs were performed at 52 days (range 36–85 days). Functional cross-sectional composites (MFF and MFI) showed moderate to strong correlations: TUG (ρ = 0.71, ρ = 0.83), FSHD-TUG (ρ = 0.73, ρ = 0.73), and RWS (left arm: ρ = −0.71, ρ = −0.53; right arm: ρ = −0.61, ρ = −0.65). WB composite variability: LMVtot, coefficient of variation (CV) 1.9% and 3.4%; MFFtot, within-subject SD (Sw) 0.5% and 1.5%; and MFItot (Sw), 0.3% and 0.4% for normal and intermediate muscles, respectively. CV and Sw were higher in intermediate (MFI ≥0.10; MFF DiscussionWe developed a WB-MSK-MRI protocol and composite measures that capture disease heterogeneity and assess muscle involvement as it correlates with FSHD-relevant clinical endpoints. Functional composites robustly correlate with functional assessments. Stability of the WB composite shows that it could be an assessment of change in therapeutic clinical trials.Classification of EvidenceThis study provides Class II evidence that quantitative WB-MSK-MRI findings associate with FSHD1 severity measured using established functional assessments.

Demand for qualified medical writers is high and expected to continue to grow. With competition for experienced writers getting fierce, the need for alternative pools of talent has become evident. This article describes a pilot internship program developed at an independent clinical and regulatory consulting company designed to allow an alternate path for entry into the field of medical writing and to foster and retain talent. The internship will provide novice writers with relevant training, connections, and job experience to aid in attaining a position in regulatory writing. We discuss the challenges and opportunities discovered during the inaugural internship year and provide lessons for developing an internship program.

This article will discuss the real-world application of agile project strategies to expedite the development of high-quality deliverables that satisfy the structure and rigor of the regulatory environment. The coronavirus disease 2019 (COVID-19) pandemic challenged regulatory writers not just to rethink the structure of their workdays and the nature of their interactions with their colleagues but to leverage technology and adopt strategic project thinking to help their teams meet aggressive timelines while working in the virtual workspace. Scenarios will include the application of agile project strategies to working on COVID-19 programs or programs heavily impacted by COVID-19, from expedited submission processes to rapid responses to regulatory authority requests for information. This assessment will include what strategies worked well, what strategies did not, and what an agile project should look like within the regulatory writing space.

The coronavirus disease 2019 (COVID-19) pandemic has necessitated that medical writers adapt their work practices quickly to assist with the preparation of documentation related to COVID-19 research and clinical trials, as well as to assess the impact of the pandemic on other clinical trials. Complexities and challenges medical writers have faced include the growing scientific knowledge of the virus, the burgeoning global footprint of the pandemic, and the evolving regulatory landscape on both COVID-19 and non-COVID-19 clinical trials.