Cello Health


In Clinical Development, Orphan Drug Congress, Orphan Drugs Live by Freya Smale

A Challenging Market Dynamic – provided by Cello Health

At best, the knowledge base about any individual rare disease is small, fragmented, and often the preserve of a relatively few thought leaders and experts in the field. The lack of knowledge about a rare disease combined with the sparseness of real-world experience in the field make identifying patients and providing a correct and timely diagnosis of the condition much more difficult. Just as importantly, delayed or repeated misdiagnoses create a separate set of challenges because this not only affects the individual patient in terms of delaying any potential benefits, but it also has a negative impact on those connected to the patient.

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Launching a rare disease asset necessitates the development of a successful clinical, commercial, and regulatory strategy that can effectively and efficiently find afflicted patients and communicate the evidence to the right stakeholders in a relevant, compelling, and timely way. In order to succeed, this requires a holistic, integrated approach.

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  • Clinical Trials: Big Bets for Uncertain Results
  • The Patient Journey: Uncharted Pathways
  • A New Planning Reality
  • The Power of Teamwork in Building Commercial Success

…and more!

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