Sustaining launch success as Orphan Medicines come of age

Launching Orphan Medicines excellently will become even more important over the next 5 years. More patients with rare diseases have pharmacotherapies available, and there are a growing number of disease-focussed registries, increasing public and policy maker awareness, and significant R&D investment in pharmacotherapies and in digital technologies to support trials and treatment. 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.