Novartis across Rare Disease: Fabhalta, and the 6 AXLRx reports mapping the competitive, patient-flow and payer picture — each scoped to a single asset and cited 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.
SCD epidemiology, genotype mix, VOC and organ-damage burden, and the 22-year life-expectancy gap across the US in-market population.
Gene-therapy access at $2.2–3.1M, the CMS Cell & Gene Therapy Access Model, VOC-freedom endpoints, and the hydroxyurea step-edit.
Two Dec-2023 gene therapies (Casgevy, Lyfgenia) reset a ~100,000-patient market, while voxelotor's 2024 withdrawal thins the oral field.