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Big Pharma · Rare Disease

Novartis — Rare Disease

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.

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AXLRx reports featuring Novartis — Rare Disease

CI · Rare Disease · CI Team · Launch Lead US PNH Competitive Intelligence Iptacopan oral pivot versus the anti-C5 IV class. Orphan-drug exclusion from IRA negotiation (US), NICE HST (UK) and SFDA lag (GCC). US · In-Market · Read report → DL · Rare Disease · CI Team · Medical Affairs US PNH Disease Landscape PNH diagnosis pathway, FLAER flow-cytometry bottleneck and the treated-prevalent pool across US centres. US · In-Market · Read report → P&HTA · Rare Disease · Market Access · Medical Affairs US PNH Payer & HTA 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. US · In-Market · Read report → P&HTA · Rare Disease · Market Access · Medical Affairs US Sickle Cell Disease Payer & HTA Gene-therapy access at $2.2–3.1M, the CMS Cell & Gene Therapy Access Model, VOC-freedom endpoints, and the hydroxyurea step-edit. US · In-Market · Read report → CI · Rare Disease · CI Team · Launch Lead · Market Access US Sickle Cell Disease Competitive Intelligence Two Dec-2023 gene therapies (Casgevy, Lyfgenia) reset a ~100,000-patient market, while voxelotor's 2024 withdrawal thins the oral field. US · In-Market · Read report → DL · Rare Disease · CI Team · Launch Lead US Sickle Cell Disease Disease Landscape SCD epidemiology, genotype mix, VOC and organ-damage burden, and the 22-year life-expectancy gap across the US in-market population. US · In-Market · Read report →