Key Considerations for Market Access Success in the Increasingly Competitive Rare Disease Space

As the landscape for rare diseases evolves, orphan disease markets that once had few or no FDA-approved therapies are now becoming more competitive, with new, innovative treatments emerging to address symptoms, modify disease progression, and even offer cures. However, with this growing competition comes a critical need for strategic market access positioning to ensure commercial success.

This white paper explores four key considerations for market access success in the rare disease space: (1) evidence and value proposition—establishing unmet need and developing strong clinical endpoints, (2) pricing—impact of price on coverage and access decision-making, (3) contracting—when to contract, how, with whom, and how much, and (4) customer and stakeholder engagement—opportunities to engage early and often with key stakeholders.

As competition intensifies, the right market access strategy can make all the difference in delivering these life-changing treatments to the patients who need them most.

Download our white paper to explore actionable insights that can help you navigate this dynamic landscape. (February 2025)