Apellis in United States — Rare Disease: the full competitive set, and the 3 AXLRx reports covering them with market-access, payer and competitive detail, every figure verified to a live source.
Iptacopan oral pivot versus the anti-C5 IV class. Orphan-drug exclusion from IRA negotiation (US), NICE HST (UK) and SFDA lag (GCC).
PNH diagnosis pathway, FLAER flow-cytometry bottleneck and the treated-prevalent pool across US centres.
Why the orphan-drug exclusion shields anti-C5 agents from IRA negotiation, ICER's 2024 value verdict on iptacopan, and Part B vs Part D routing.