Symposium: Real-World Data: Driving Innovation and Improved Outcomes in Rare Diseases

Rare diseases are often debilitating, with life-threatening manifestations that constitute some of the biggest unmet needs in healthcare, namely, clinicians with specialized training and availability of treatments. Patients often experience disease progression due to lack of diagnosis or misdiagnosis, resulting in years of high healthcare resource use and poor outcomes. The definition of rare disease varies by market, which makes quantifying the true volume of rare diseases challenging. Limited real-world data impact our ability to observe or forecast the full patient journey and generate sufficient evidence to meet requirements for orphan drug regulatory submissions and stakeholder engagement needs. Accelerated processes are required for disease awareness, diagnosis, treatment access, digital health monitoring, and telehealth delivery by primary care providers and specialists. Although orphan drug costs are presently relatively unchallenged by payers, an increase in expensive drugs has drawn scrutiny, as most payers believe current prices aren’t sustainable. Thus, the need for innovative solutions that illuminate the evidence pathway for economic sustainability and improvement of rare disease outcomes is imperative. This session will examine disparities in healthcare access and patient outcomes, evaluate analytical challenges faced in these areas, and explore technological, analytical, and engagement strategies to mitigate these challenges in rare diseases.
March 21 @ 15:30
15:30 — 17:00 (1h 30′)

Hyatt Regency New Orleans

Aaron Kamauu, Canter Martin, Johnathan Wert, Kathy Belk, Laura Clark, Neeraj Iyer