The Rare Disease eCOA Playbook

Why eCOA is Essential in Rare Disease Clinical Trials

With fewer than 200,000 patients affected per condition, rare disease trials must optimize every data point. Electronic Clinical Outcome Assessments (eCOAs) play a critical role in:

Reaching small, globally dispersed patient populations

Capturing nuanced symptom data in real time

Enabling decentralized and hybrid trial models

Supporting caregiver input via observer-reported outcomes

Ensuring audit-ready compliance across regulatory regions

Built for Sponsors and CROs Solving Rare Challenges

This playbook is ideal for a wide range of stakeholders involved in rare disease research, serving biotech and pharma clinical teams, as well as CROs who need scalable implementation frameworks.

It also provides guidance for digital health strategists deploying decentralized or hybrid models, patient advocacy and engagement teams collaborating on protocol design, and regulatory and quality teams responsible for overseeing eCOA compliance.