Companies developing rare disease treatments need to make high-stakes decisions to bring treatments to patients. These decisions are often based on limited data, highly heterogeneous patient journeys, and diagnoses that come too late.

Patient experience data can help close these gaps and support the development of treatments that meet patient needs and timely reach patients. However, this is only possible if it is captured with rigor and used in ways that meaningfully influence strategy, development, and access decisions.

This fireside chat explored how to make patient experience data practical and decision-ready across the rare disease lifecycle.

The discussion covered what strong patient experience data looks like, where it adds the most value from early strategy through clinical development and launch planning, and how companies can navigate common challenges such as small populations, inconsistent methodologies, limited resources, and uncertainty around regulatory and health authority expectations.