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Actualités & Perspectives

Rare Diseases Treatments Launches

3/7/24

🚀 Rare Diseases Treatments Launches; Insights From IQVIA's Latest White Paper 🚀

Medicines for rare diseases are a significant contributor to novel drug approvals and launches, representing more than half of new active substance approvals in the U.S. and around 45% in Europe. Despite this, an estimated 95% of around 10,000 rare diseases lack treatments, highlighting the unmet need in this field. Understanding successful rare disease launches becomes paramount, here are 3 success points :

🏥 Health System Readiness:Despite progress, many health systems are not financially or operationally ready to adopt rare disease innovations. Companies must understand the patient journey within each country's healthcare system, focusing on early diagnosis support, patient programs, and health system partnerships.

😷 Stakeholder Engagement:Building strong relationships with patients, caregivers, patient organizations, and experts is essential from the earliest stages of rare disease medicine development. Early cooperation with Patient Advocacy Groups can lead to cross-stakeholder cooperation and the development of registries and trial designs.

🔍 Value and Evidence:An integrated evidence strategy, combining clinical and real-world evidence, is crucial in rare disease medicine development. Understanding disease epidemiology, accurate patient numbers, and generating real-world evidence are essential for successful market access. Particularly for curative therapies, evidence strategies are exceptionally complex, requiring ongoing adaptation to the latest developments.

🌍 The future of rare disease launches holds promise, yet challenges persist. Globally, rare disease launches are on the rise, with an increasing focus from both biotech and large pharma. Companies must invest early in meticulous planning, stakeholder engagement, and flexibility to navigate this complex landscape successfully.