Webinar Overview

Stakeholder groups, including regulators, payers, policy makers, and patients, have demanded patient-centeredness in rare disease drug development programs. Thus, patient-centered outcomes are critical for inclusion as endpoints in rare disease drug development programs. A key aspect of ensuring optimal patient-centered endpoint strategy involves patient and/or caregiver engagement to inform endpoint selection. Yet traditional methods for gathering early patient input, such as focus groups, are very often impossible in rare disease populations due to the nature of rare diseases. This webinar will cover innovative methods for gathering patient input regarding meaningful endpoint concepts in these difficult to reach populations.  Specific examples will be provided regarding:

  • Successful engagement with patient advocacy groups
  • Utilizing existing data sources such as patient blogs or registries to understand the patient experience
  • Innovative techniques for identifying and gathering information from rare disease patients or their caregivers
  • Technology solutions to enable patient engagement and reduce patient burden

Who Should Attend

  • Those involved in planning or implementing drug development programs in rare diseases.

Key Learning Objectives

  1. Understand the need for inclusion of patient-centered outcomes in rare disease drug development programs
  2. Recognize the key challenges of engaging patients in rare disease research
  3. Identify innovative solutions to engage patients in endpoint strategy and selection/inclusion of patient-centered outcomes in drug development programs


Kathleen W. Wyrwich, PhD, Senior Research Leader, Outcomes Research, Evidera

Margaret Vernon, Senior Research Scientist, Outcomes Research, Evidera