Line of therapy analysis

Case study

Supporting commercialisation efforts of rare disease, Pulmonary Arterial Hypertension (PAH)

Overview

Pulmonary arterial hypertension (PAH) often presents with nonspecific symptoms that complicate timely recognition. The challenges of differential diagnosis are significant, as PAH shares overlapping clinical features with other cardiopulmonary conditions, leading to frequent delays or misdiagnoses. Although current treatment options—including prostacyclin analogs, endothelin receptor antagonists, and phosphodiesterase-5 inhibitors (PDE5)—have improved patient outcomes, many populations, particularly those in underserved communities or with limited access to specialised care, remain under treated.

Challenges

The nonspecific symptoms and complicated differential diagnosis, only confirmed via invasive right heart catheterization, complicates study design within real-world data and leading to a paucity of reliable evidence to inform commercialisation strategy for new to market therapeutics.

Solution

A high confidence cohort was first established accounting for changes in ICD10 coding, patient activity in Symphony Health, an ICON plc company’s Integrated Dataverse (IDV®) and ongoing patient survival. Data source and capture was also considered to account for extensive product blocking within the market. Resulting patient cohort was benchmarked against epidemiological sources of data to ensure the sample accurately represented the true population. 

Symphony Health conducted a comprehensive line of therapy analysis to support ongoing commercialisation efforts of a new launch. Key metrics within the analysis included: 

  • Market shares by line
  • Time between lines
  • Time from diagnosis to treatment
  • Combination and monotherapy treatment, overall and by line
  • Examined differences between treatment preferences at academic versus community treatment locations

Insights

As therapeutic options emerge, overall disease state awareness has increased, and overall diagnosed patient volumes are rising. Healthcare professionals (HCPs) practicing in community settings are often less likely to prescribe combination therapy; this could be due to the patient presenting with less severe symptomology or a reluctance to increase overall pill burden. PDE5s remain the primary treatment choice, either alone or in combination, across all lines of therapy. Likely due to the availability of both brand and generic options and a better perceived safety profile when compared to endothelin receptor antagonists and prostacyclin.

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