Clearing the way for patient access

Rare diseases affect more than 300 million people, about 4% of the world’s population, by conservative estimates. But the patient pool for each rare disease is tiny and geographically dispersed. New treatments must win reimbursement from diverse payers and health technology assessment agencies if patients are to benefit. Read on to learn how companies can negotiate with insurers to maximize patient access to treatments in the US market and navigate recent shifts in the EU.

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