Project Overview

A leading global biopharma company engaged us to gauge the therapeutic and commercial outlook for an investigational oral treatment (“Product X”) in China’s PNH arena. Twenty nationally recognized hematology key-opinion leaders (KOLs) participated in one-hour, web-assisted interviews to benchmark current injection-based regimens, identify unmet needs, and outline the market-access hurdles that must be overcome for an orally delivered alternative.

Key Learning Objectives

• Treatment Landscape – Map Chinese PNH diagnosis pathways, standard-of-care usage, and gaps in symptom control or quality-of-life support.

• Product X Potential – Assess clinical and practical advantages of an oral therapy versus existing injectable options, including fatigue reduction, adherence ease, and infusion-center avoidance.

• Access Barriers – Clarify reimbursement hurdles, prescription-switch triggers, and physician-education needs that could affect launch trajectory.

• Evidence Priorities – Gather KOL guidance on refining trial endpoints (e.g., patient-reported fatigue scales) and building real-world evidence to strengthen health-economics arguments.

Methodology

• Web-Assisted Depth Interviews

  • 60-minute, semi-structured calls with 20 leading hematologists across Tier-3 hospitals and national rare-disease centers.

  • Discussion guide covered patient journey, treatment decision drivers, perceived value of oral formulations, and access policy trends.

• Thematic Synthesis

  • Rapid content analysis to surface consensus themes and divergent viewpoints, feeding into opportunity and risk mapping.

Key Deliverables

• PNH Care Pathway Map visualizing diagnostic milestones, referral flows, and treatment drop-offs.

• Value Proposition Assessment comparing Product X to current injectables on efficacy, safety, convenience, and economic dimensions.

• Access Roadmap outlining strategies for Tier-3 hospital adoption, rare-disease-center partnerships, patient-assistance programs, and evidence packages for reimbursement negotiations in the next few years.

• Clinical-Trial Refinement Memo summarizing KOL-driven recommendations on endpoint selection and long-term data capture to align with payer expectations and patient priorities.