Exploring Global Market Access for Rare and Complex Conditions Part One: Overcoming challenges and seizing opportunities in global market access for rare and complex conditions

The outlook for drug development to treat rare diseases looks encouraging, offering hope to the 300 million people globally facing the significant challenges of these often-overlooked conditions.

However, for pharmaceutical manufacturers, navigating the path to effectively launch these transformative therapies in global markets is complex. Companies must address a multitude of evolving access challenges, including varying approaches to reimbursement across the globe, choosing the optimal pricing strategy that works globally and at the individual country level, and the increased demand for risk sharing outcomes-based access agreements that rely on the generation of real-world evidence.

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