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Case Study: Defining the Rare Disease Patient Journey to Support Commercialization of Therapy

The Case for Real-World Data (RWD) in Delivering Targeted Patient Care

the situation

The Situation

Rare diseases are complex, and with limited research available, therapeutic options are often limited. Gene therapy, a growing area of clinical research, is showing great promise in treatment that may be life-altering for patients with many rare diseases.

A client who specializes in gene therapy for a particular rare disease came to EVERSANA looking to better identify and target their therapy to patients and healthcare providers. They looked to gain a deep, data driven understanding of:

  • The patient journey across multiple age groups of patients.
  • Prescribing patterns of healthcare providers who specialize in this disease.
  • Geographical factors impacting patient care.
  • Cost of care.
  • Additional factors impacting patients and healthcare professionals in the treatment of this disease.
the solution

The Solution

Through a strategic, consultative approach, EVERSANA established a structured and holistic plan that leverage artificial intelligence, machine learning and robust claims data to define a richer patient journey profile and created an interactive dashboard of RWD and actionable insights.

Powered by ACTICS by EVERSANA, a premier, tech-enabled solutions that delivers commercial analytics, integrated patient analytics, pricing and market access analytics, along with RWD and Health Economics & Outcomes Research (HEOR), this dashboard was critical in informing the client’s clinical and go-to market strategy, which included:

  1. Successfully defining a patient study cohort with input from the client and EVERSANA’s clinical team for further analysis.
  2. Generating separate insights into the disease of patients in different age groups using machine-learning methods such as Mutual Information.
  3. Analyzing the existing payer landscape via cost-of-care analysis.
  4. Assessing a graph-based referral network to generate communities and identify key opinion leaders.
the results

The Results

Based on the data and analysis provided by EVERSANA, the client successfully:

  • Established guiding principles for therapy/indication by creating a patient definition.
  • Identified high-value targets.
  • Characterized the existing market landscape.
  • Addressed key business questions through a detailed, longitudinal cohort analysis.
  • Visualized the results through custom and interactive Tableau dashboards.