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Advancing the Science of Study Endpoints


Session 9: Selecting COA Endpoints and Analyzing COA Data in Rare Disease - Dealing with Small Sample Sizes and Heterogeneity

Session Chair(s)

Emuella  Flood

Emuella Flood

  • Director, Patient-Reported Outcomes
  • AstraZeneca, United States
Rare disease populations are typically small and heterogeneous, and often involve different subtypes, such as specific gene mutation subgroups, further reducing sample sizes. Specific COA-related challenges related to small sample sizes and heterogeneity of rare diseases include the selection and validation of COA measures and analyzing and interpreting the COA data to support treatment benefit. This session will identify the challenges of selecting COA endpoints and analyzing COA data in rare disease populations, including in evaluating psychometric properties of COA instruments. Potential Solutions will be offered, illustrated though case examples. The regulatory perspective on addressing these challenges will also be presented.
Learning Objective : At the conclusion of this session, participants should be able to:
  • Identify the challenges of COA endpoint selection and analysis in rare disease populations and potential solutions, such as the use of RWE and composite endpoints
  • Employ strategies to perform psychometric validation in small, heterogeneous samples
  • Discuss the regulatory perspective on endpoint selection and analysis in rare disease

Speaker(s)

Laura Lee  Johnson, PhD

Selecting COA Endpoints and Analyzing COA Data in Rare Disease-Dealing with Small Sample Sizes and Heterogeneity

Laura Lee Johnson, PhD

  • Director, Division of Biometrics III, Office of Biostatistics, OTS, CDER
  • FDA, United States
Linda  Nelsen, MHS, MS

Selection and Development of Patient-focused COAs for Rare Disease Clinical Trials

Linda Nelsen, MHS, MS

  • Senior Director and Head, Value Evidence and Outcomes-Patient Centered Outcomes
  • GlaxoSmithKline, United States
Jeffrey  Palmer

Selecting COA Endpoints and Analyzing COA Data in Rare Disease-Dealing with Small Sample Sizes and Heterogeneity

Jeffrey Palmer

  • Senior Director, Biostatistics
  • Pfizer Inc, United States
R.J.  Wirth, PhD

Psychometric Evaluation with Small Samples

R.J. Wirth, PhD

  • CEO
  • Vector Psychometric Group, LLC, United States

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