• How do value assessment methodologies differ for ultra-rare vs. rare diseases?
• What pricing models balance R&D costs with patient access in small populations?
• How are ICER evaluations adapting for single-patient N-of-1 trials?
• What lessons come from the EU's cross-border procurement initiatives?
• How do manufacturing minimums impact COGS for micro-populations?
• What pediatric pricing incentives exist under the Creating Hope Act?
• How are global reference pricing systems affecting launch sequencing?
• What compassionate use data influences formal pricing decisions?
• How are payers structuring outcomes-based contracts for gene therapies?
• What HTA alignment strategies prevent reimbursement delays?
• How do revenue forecasts inform pipeline prioritization?
• What patient advocacy roles shape pricing transparency?

• Orphan Drugs
• Value Assessment
• Patient Access
• ICER Frameworks
• Cost-Effectiveness
• Pediatric Indications
• Global Reference Pricing
• Manufacturing Scale
• Patient Transparency
• HTA Alignment
• Revenue Forecasting
• Pipeline Prioritization

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