Session (CTI)

Advances in the development and real-world use of therapies for rare cancers are occurring rapidly. Real-world evidence (RWE) is increasingly important for market-access (MA) decisions because clinical development programs for rare cancers are often constrained by small sample sizes, single-arm trials, and immature endpoints. Despite greater availability of real-world data (RWD), payers and health-technology assessment bodies (HTAbs) remain cautious about the credibility, generalizability, and decision relevance of RWE generated in rare cancer settings. Equally important, yet seldom discussed, are the practical and methodological challenges of generating high-quality RWE in rare cancers with respect to MA decisions. The ISPOR RWE- and Oncology-SIGs jointly propose a multidisciplinary panel to close this gap by bringing together perspectives from academia, industry, HTAbs, and payers. The session will pinpoint common barriers, methodological challenges, and discuss potential evidence frameworks to enhance confidence in RWE for MA decisions. Moderator (Shilpi Swami) will set the stage with access challenges in rare cancer, why conventional evidence paradigms often fall short, and introduce the role of RWE across the evidence lifecycle. • The first panelist, Jagadeswara Rao Earla, will focus on practical realities and challenges of generating RWE in rare cancers (example: data sources, small sample size). • The second panelist, Sandipan Bhattacharjee (bringing perspectives of an academic rigor alongside industry applications), will focus on methods for how RWE advancements (example: external control arms, target trial emulation, artificial intelligence application) can improve the credibility of evidence generated in rare cancer settings. • The third panelist, Diana Brixner will discuss how RWE is interpreted and used by HTAbs and payers in MA decisions for rare cancers. The panel (40-45 minutes of total presentation and remaining time for Q&A) will provide MA, evidence generation, and policy stakeholders with a potential practical framework for evaluating the strengths and limitations of RWE in rare cancers.