Day 3 of ISPOR 2026 is live in Philadelphia — and today’s sessions are the ones rare disease evidence teams have been waiting for all week.
The pipeline is growing. The evidence bar is rising. And the frameworks the industry relies on still weren’t built for populations this small. The conversations happening on that floor today matter.
A look at what’s already been said — and what’s still ahead 👇
Yesterday (May 18) — Agentic AI in evidence submissions took center stage — drawing the line between AI tools that accelerate evidence workflows and ones that actually survive regulatory and HTA scrutiny. The bar has shifted from “can AI help?” to “can you prove it holds up?”
Today (May 19) — FDA’s evolving Rare Disease Evidence Principles are opening new doors — but only for teams with the right natural history data and patient-level evidence to walk through them. Rare disease innovation meets regulatory reality on the floor today.
Also today — Moderated by Shilpi Swami (ConnectHEOR), with Scott Ramsey, Sandipan Bhattacharjee (Bayer), and Diana Brixner (University of Utah) — rare oncology takes center stage. The patient populations are too small, the clinical complexity too high, and the payer scrutiny too intense for general RWE tools to cut it.
Tomorrow (May 20) — The week closes with the hardest question yet. How do you fit rare disease into an HTA paradigm that was never designed for it? Tomorrow’s discussion will shape how the field thinks about this gap for years to come.
At Clinakos, we built Clarion™ specifically for this. Our AI Research Agent navigates real, consented patient journeys across 1,500+ rare disease and oncology panels — turning complex evidence questions into high-fidelity insights in weeks, not years.
