Case Study

Build a real-world dataset for rare disease evidence generation

Project overview

Inspire partnered with a client to provide data collection, integration, and analysis for end-to-end evidence generation for two rare neuromuscular disorders.

Impact

By capturing both retrospective and prospective data for each patient from all of their providers, Inspire can:

Capture novel insights about the patient journey prior to disease onset
Study treatment patterns in the rare neuromuscular disorders community
Better understand the impact on clinical and economic outcomes.

Patient recruitment

Prior to engaging with the client, Inspire had existing access to over 9,000 patients with rare neuromuscular disorders. These patients have organically joined Inspire from both urban and rural areas across 50 U.S. states.

In addition, Inspire was able to capture over seven years of retrospective health data.

Custom data modeling

Based on client objectives, Inspire:

Created a fit-for-purpose dataset by abstracting both structured and unstructured (e.g., physican notes) data elements.
Prospectively collected patient-reported outcome measures (PROM) and patient experience data (PED) directly from patients.
Designed custom abstraction methods in order to extract condition-specific variables, including disability measurements, neurologic signs related to progression, and disease subtype.

Unique integrated data

Inspire links medical and pharmacy claims data with PROM and PED to enable additional analyses around healthcare resource utilization by leveraging tokenization, complementing the clinical data available through EHR.