Rare Disease Pricing & Access: Closing the Gap Between Innovation and Equity

Scientific breakthroughs have transformed rare disease treatment from aspiration to reality. But as innovation accelerates, patient access lags behind. Our latest white paper, The Rare Disease Access Gap: Navigating the Economics of Global Innovation, explores why pricing and reimbursement systems have not kept pace—and what must change to ensure that life-saving therapies reach those who need them most.

What’s Inside:

Section 1: The Economics of Rare Disease Drug Pricing

• Why orphan drug prices are rising—and what really drives them
• The innovation premium model and investor expectations
• The clash between pricing models and HTA cost-effectiveness frameworks

Section 2: The Global Market Access Landscape

• Regulatory harmonization vs. fragmented reimbursement systems
• Country-by-country analysis: U.S., EU4, Japan, and emerging markets
• How delays and inequities shape patient access worldwide

Section 3: The Evidence–Value Gap

• Why approval doesn’t guarantee coverage
• The mismatch between regulatory flexibility and payer rigor
• Strategies to bridge the gap with real-world evidence and adaptive trials

Section 4: Future Directions

• Integrated evidence planning from program inception
• Policy levers for affordability and innovation
• Collaborative models and cross-country agreements for sustainable access

Download the full white paper to learn how biopharma companies can navigate pricing and access challenges while sustaining innovation.