Orphan medicines launch excellence: sustaining launch success

Presented by

Peter Meeus, European Orphan Medicines, Sarah Rickwood, IQVIA, Angela McFarlane, IQVIA, Paul Tunnah, pharmaphorum

About this talk

Launching orphan medicines excellently will become even more important over the next five years as more patients with rare diseases have pharmacotherapies available. There are also a growing number of disease-focussed registries, increasing public and policy maker awareness, and significant R&D investment in orphan drugs, as well as in digital technologies to support trials and treatment. But a new frontier of challenge faces orphan medicines companies as the gap between orphan medicines and mainstream specialty products narrows. To understand how to succeed in launching an orphan medicine in the coming years, companies must learn from past launches and apply an orphan medicines-focused launch excellence framework for success. This internationally relevant webinar will provide US and European examples to illustrate the ways in which the pharmaceutical market for orphan medicines is changing and how, consequently, launch strategies must evolve. This pharmaphorum debate, held in partnership with IQIVA, will also look at how pharmaceutical companies can optimise all the activities needed to take orphan medicines from clinical development to commercialisation, including: •Identifying the three fundamental challenges for orphan medicine launch excellence •Discussing the ways in which companies launching orphan medicines can learn from excellence in mainstream launches •Examining, in-depth, the specific areas of focus which are crucial for orphan medicines excellence.

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