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Vice President, EMEA Thought Leadership, IQVIA
Senior Consultant, EMEA Thought Leadership, IQVIA
Join IQVIA in its Launch Excellence web-series, where we build on our Launch Excellence framework to explore challenges and key drivers of commercial success for new launches in rare and orphan disease.
Medicines for rare diseases represent a significant share of all novel drug launches. In fact, 143 drugs launched with orphan drug designation in the US in the past five years i.e. 53% of the 268 US launches, and orphan approvals also represent a significant proportion of all European approvals. Rare diseases represent a significant focus of new innovation, a testament both to groundbreaking research and to the success of policies designed to encourage the development of orphan medicines. With high levels of remaining unmet need and almost a third of the clinical trial pipeline in the US/EU focused on rare diseases, there will be continued interest in this therapeutic area. An estimated 95% of ~7000 rare diseases still have no available treatment.
The post-pandemic launch environment has been challenging for innovative launch commercial success, but rare disease launches proved resilient. However, the ongoing challenges of healthcare system fragility and austerity mean that payers and policy makers increasingly do not see orphan medicines as ‘exceptions’, protected from budget scrutiny. From 2024, key policy changes/proposals on both sides of the Atlantic ultimately threaten to make the environment more challenging for orphan medicines, as some of the key enablers for orphan drugs are changed or at-risk. This makes the need for focus on the key pillars of rare disease launch excellence even more critical.
In this webinar, we will explore the three pillars of rare disease launch readiness, how companies have successfully addressed, Value and Evidence, Healthcare System Readiness, and Stakeholder Engagement to drive strong orphan medicine launches, and how these principles should be applied in the future.
3 key takeaways:
Take advantage of the latest tools, techniques, and deep healthcare expertise to create scalable resources, precision insights, and actionable ideas.
Combine data science, technology, and analytics driven by artificial intelligence to support new efficiencies and business insights -- without additional capital investment.
IQVIA is using vast quantities of data in powerful new ways. See how we can help you tap into information from past trials, patient reported outcomes and other sources to accelerate your research.
