Navigating patient experience data in rare disease

Published 5 August 2026

Author(s): Vivienne Hanrahan, Sprout

Meaningful engagement is not defined solely by trial outcomes, but by whether patients feel heard, informed and involved throughout the development process

Patient experience data (PED) has become increasingly important in drug development, and especially so for rare disease, offering critical insight into how people live with their condition, what outcomes matter most to them and how they experience participation in research.

Regulators and HTA bodies expect to see PED alongside clinical evidence in submissions. Yet collecting patient insights is only part of the story. Across the pharmaceutical industry, dedicated teams work to bring patient perspectives into development for the right reasons, yet they frequently face the challenge of translating these efforts into a tangible reflection of the patients’ true priorities and realities.