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Tuesday, October 6
 

07:00 UTC

S204 - Creating Global Connections Through Early Statistical Leadership in Rare Disease Trials
Tuesday October 6, 2026 07:00 - 07:30 UTC
In complex clinical research, collaboration begins well before database lock or final analysis. It begins when statisticians are involved early enough in protocol development to shape what is measurable, sustainable, and meaningful for patients. This presentation uses a rare pediatric dermatology trial as a case study to show how early statistical input can prevent avoidable missing data before the first participant is enrolled.

The original design was scientifically ambitious but operationally burdensome, with frequent in-clinic visits, narrow assessment windows, and substantial caregiver burden. Statistically, this created a foreseeable risk of informative missingness, loss to follow-up, and reduced interpretability in a small and vulnerable population. Early statistical leadership helped reframe the design around feasibility as well as rigor.

The talk will illustrate how collaboration among statisticians, clinicians, programmers, operations teams, and digital partners can reduce this risk. Examples include identifying burden-sensitive endpoints, anticipating missing-data pathways, and considering remote ePRO, image capture, or AI-supported skin assessment to replace selected in-person visits without compromising data quality.

The broader message is that statistics is not only an analysis discipline; it is also a design and connecting discipline.
Speakers
avatar for Shrutimita Pokhariyal

Shrutimita Pokhariyal

PHASTAR
Shrutimita Pokhariyal is an experienced biostatistics professional with over 14 years in clinical research, with expertise spanning multiple phases of clinical trials. Her work includes statistical strategy, study design, protocol and SAP development, and collaboration across cross-functional... Read More →
Chairs/Hosts
avatar for Shrutimita Pokhariyal

Shrutimita Pokhariyal

PHASTAR
Shrutimita Pokhariyal is an experienced biostatistics professional with over 14 years in clinical research, with expertise spanning multiple phases of clinical trials. Her work includes statistical strategy, study design, protocol and SAP development, and collaboration across cross-functional... Read More →
Organizers
avatar for Shrutimita Pokhariyal

Shrutimita Pokhariyal

PHASTAR
Shrutimita Pokhariyal is an experienced biostatistics professional with over 14 years in clinical research, with expertise spanning multiple phases of clinical trials. Her work includes statistical strategy, study design, protocol and SAP development, and collaboration across cross-functional... Read More →
Tuesday October 6, 2026 07:00 - 07:30 UTC
Zoom Room #2
 
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