2024-05-12-艾昆纬-从孤儿到机遇_掌握罕见病卓越上市（英）_25页_3mb

报告摘要

Rare Disease Launch Excellence Summary

Market Growth: Rare disease drugs represent a significant focus of innovation, with ~45% of EU and >50% of US new drug approvals being orphan-designated. 70% of clinical trial activity targets rare diseases.
Challenges: Healthcare systems face capacity gaps due to high-cost treatments (e.g., gene therapies), payer budget constraints, and complex HTA (Health Technology Assessment) processes evolving post-pandemic. Environmental factors like the COVID-19 pandemic exacerbated these issues.
Success Factors:
- Health System Readiness: Requires early infrastructure building, including diagnostic capacity and patient support programs. Example: The CF Foundation’s patient registry supports care pathways.
- Stakeholder Engagement: Early collaboration with patients, advocacy groups, and healthcare professionals builds trust and provides real-world insights. Horizon’s Tepezza launch used direct-to-consumer engagement during COVID, achieving rapid uptake.
- Value and Evidence: Developing integrated evidence strategies, including clinical and real-world data (e.g., quality of life metrics), is critical for payer acceptance.
Lessons Learned: Tailored approaches considering the "three populations" (label, market access, treatable) are essential. Companies must balance urgency with evidence generation and prepare well in advance for market access challenges.
Future Outlook: Resilience post-pandemic amid tightening budgets. Opportunities lie in unmet needs, evolving technologies, and partnerships (e.g., patient-centric development).