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.


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.

Infographic illustrating that Inspire is the central point where electronic medical records, healthcare claims data, patient-reported outcomes, and authentic patient and caregiver conversations come together

Benefit to the client

Together with Inspire, the client was able to:


Find and collect data from rare-disease patients from within existing Inspire patient communities.


Integrate PED and PROM data with patients’ longitudinal medical data. Because Inspire patients are both fully-consented and recontactable, the data asset was curated both retrospectively and prospectively. Patients can be easily recruited for future work.


Analyze this integrated data to drive evidence generation and dissemination efforts, shaping value discussions and optimizing marketing access across clinical development and commercialization phases.


Have on-demand, ongoing access to patients across a variety of diseases to meet their evolving evidence needs.

Illustration of a group of qualified individuals displaying and analyzing a collection of health data

Contact Inspire to learn more about our real-world data and evidence generation capabilities

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