Post by RTI Health Solutions

World Orphan Drug Congress in Boston didn't disappoint.    The rare disease community showed up with hard questions, and the conversations delivered.   A few themes that stood out for our team: - Global pricing dynamics are fundamentally reshaping launch strategy. The downstream effects of MFN pricing are real and require a more integrated approach to sequencing and early-access decisions. - Defining value in rare disease is still one of the field's hardest problems. Regulators, payers, and patients aren't always working from the same definition — and that gap has real consequences. - Early planning is critical to ensure you have decision-ready evidence when you need it. Our own Sandra Talbird and Dee Dee Mladsi took the stage to address this head-on with her keynote, Revealing the Value of Cell & Gene Therapies: The Power of Disease Modeling—exploring how disease modeling can help bridge evidence generation and stakeholder decision-making.   Always energizing to be in the room with this community. How is your organization bridging the gap between the evidence you generate and what payers and patients actually need to hear?   #RareDisease #MarketAccess #HEOR #PatientVoice #CellAndGeneTherapy #RTIHealthSolutions #MFN #WODC2026