Session (CTI)

Purpose: This breakout session will create a shared space for both US and global patients, caregivers, payers, regulators, clinicians, and industry perspectives to openly discuss when does investing in DSPROs Value Propositions actually change conversations, decisions, and experiences and when does it mostly add complexity and cost? By the end of the session, participants will be able to: • Compare the pros and cons of DSPROs vs generic measures for valuing orphan/innovative therapies. • Discuss ROI for companies and “return on burden/benefit” for patients and families. • Pinpoint when DSPROs are truly “must-have” versus “nice-to-have” in real-world decisions. Description: This session will begin with a brief framing presentation (10 minutes) to outline how orphan and innovative therapies are currently evaluated, highlight where generic measures seem to miss what matters most to patients and caregivers when developing a value proposition. Next, three brief talks (~10 minutes each) will examine DSPROs from different perspectives: • Steve brings 15+ years of experience in industry HEOR perspective currently representing Ionis Pharmaceutical talks on when DSPROs strengthen (or fail to strengthen) value and “return on investment” • Angie brings 30+ years of experience as global lead for patient-led musculoskeletal health advocacy patient advocacy and EUPATI Fellow, she talks about how DSPROs can amplify or miss lived experience, including “return on burden” • Nora brings 10+ years of conducting and disseminating research on child health outcomes at Queens University and gives an academic perspective on how DSPROs show up in real-world decision making, including what level of outcome evidence feels meaningful versus excessive in practice. The session will conclude with a moderated, interactive discussion and live polling (20 minutes), using case scenarios and a simple discussion guide to help attendees explore when investing in DSPROs adds meaningful value for stakeholders and when existing approaches may be sufficient in both US and global settings.