Shaping the launch strategy for a novel hereditary angioedema (HAE) therapy across European markets

A global biopharmaceutical company developing a novel hereditary angioedema (HAE) therapy sought to understand baseline awareness, diagnostic pathways, and treatment practices across key European markets.

The objective was to inform launch strategy, refine stakeholder targeting, and identify medical education priorities for this ultra-rare condition (prevalence ~1 in 50,000) with limited physician familiarity.

Phronesis delivered actionable insights into diagnostic challenges, referral pathways, and stakeholder engagement, ensuring the launch strategy was grounded in real-world clinical behavior and unmet needs.

Challenge:
Ultra-rare disease dynamics created significant complexity in research design and execution:

• Low physician exposure: Limited patient volumes meant many clinicians had minimal direct experience with the condition
• Delayed and fragmented diagnosis: Non-specific symptoms led to prolonged diagnostic journeys (median 12.6 years from symptom onset to diagnosis) involving multiple specialties
• Multidisciplinary pathways: Patient management extended beyond core specialists to include general physicians involved in early identification and referral

• Recruitment feasibility constraints: Traditional screening criteria such as "treated at least five patients in the past year" were not viable, requiring more flexible inclusion approaches

Approach:

Phronesis designed a multi-phase research program to capture insights across the full patient journey:

• Multi-specialty coverage: Included core specialists (allergists and immunologists), adjacent specialties (gastroenterologists and dermatologists), and PCPs involved in diagnosis and referral pathways
• Targeted recruitment strategy: Combined proprietary panels, expert outreach, and engagement with specialist networks and treatment centers
• Two-wave quantitative design: 35- minute. baseline study with 100 respondents to establish awareness, diagnostic, and treatment benchmarks. 30-minute. follow-up wave after six months with same respondents to track evolution in perceptions and behaviors
• Integrated qualitative inputs: Embedded open-ended questions to capture decision drivers, referral patterns, and treatment considerations

Business Impact:

The study generated actionable insights that directly informed launch and engagement strategy:

• Clear mapping of diagnostic pathways enabled identification of key delays and intervention points
• Broader stakeholder targeting strategy highlighted the importance of engaging beyond core specialists
• Refined medical education priorities focused on addressing awareness gaps and misdiagnosis patterns
• Informed a phased launch sequence that prioritized core specialists before expanding engagement to adjacent and primary care stakeholders
• Improved understanding of diagnostic challenges provided a foundation for targeted awareness and earlier patient identification