A 12-sheet pricing architecture built market by market: price ladder, international reference-price basket, value-based ICER, gross-to-net waterfall, launch sequencing, and revenue scenarios — not a single number, a re-runnable model.
Iptacopan's substantial-benefit finding gave Germany real negotiating leverage over Novartis. Six months on, no negotiated net price has surfaced in the Lauer-Taxe — the pricing signal this model is built to catch before a launch-sequencing decision locks in the wrong assumption.
NICE has cleared every modern PNH anti-complement therapy through the standard £20,000-30,000 Technology Appraisal route. From ravulizumab to crovalimab, none used the more generous Highly Specialised Technologies threshold, and each went via a confidential patient access scheme.
GCC anti-C5 tender pricing already runs 40-60% of US WAC, anchored to whichever EU comparator prices lowest. A further 10-20% negotiation discount compounds it. Iptacopan has to clear the same cascade, still 12-24 months from SFDA registration.
Agalsidase beta runs an estimated £150-250K per patient per year against migalastat's £80-120K. NICE has quantified that £70-130K annual switch saving but the NHS has not captured it at scale, and pegunigalsidase's TA915 commercial arrangement now sets a third price point.
NICE's accepted 20-35% PAS discount off budesonide's WAC sets the pricing floor sparsentan already clears. The £140-180M NHS budget ceiling applies once the ACEi/ARB gate narrows eligibility to 3,000-5,000 patients.
NPHC pays roughly SAR 1.2-2.4M per patient per year for enzyme replacement against SAR 400-600K for migalastat. That 72% differential gives NPHC a direct incentive to switch eligible patients, capped almost entirely by a single-laboratory diagnostic bottleneck rather than by price.
GCC Dravet pricing is an import-cost problem, not a rebate negotiation. Cannabidiol's Schedule-1-equivalent narcotics classification adds USD 10-15K in compassionate-programme cost plus SAR 5-8K in import logistics, while stiripentol's non-narcotic status keeps it at SAR 30-50K through standard hospital import.
Casgevy and Lyfgenia list at $2.2M and $3.1M, but the sticker price is not what gets paid. CMS's Cell and Gene Therapy Access Model, Medicaid concentration, and a $1.5-1.9M ICER ceiling decide the realised net.
NICE recommended crizanlizumab in 2021, then withdrew the guidance in 2023 when the confirmatory trial failed. The licence was revoked. In UK sickle cell, price is not the binding constraint. Confirmatory evidence is.
Lanadelumab lists near $450,000 a year against berotralstat's roughly $95,000. ICER's 2021 fair-value benchmark for berotralstat lands almost exactly on that list price, and the three 2025 entrants carry no ICER anchor of their own.
NPHC's negotiated Zolgensma price runs $1.5-1.8M against $2.125M US list. But the real mechanism is a 24-month motor-milestone rebate, the same structure now anchoring risdiplam and nusinersen pricing across the Gulf.
Lanadelumab tenders at SAR 300,000-400,000 a year, but NPHC has no routine formulary price at all. Access runs through an individual-case bar only 30-40% of submissions clear, while private VHI approves at a materially lower documentation threshold.
Three IV enzyme replacement brands run ~$300,000/year with no generic rival, so preferred-ERT designation is the real pricing lever. Eliglustat's CYP2D6 gate and generic miglustat's trial-first rule complete the picture.
All five FDA-approved IgAN therapies clear the same prior-authorisation gate, not a negotiated rebate table. That gate is biopsy-confirmed diagnosis, UPCR 0.8-1.5 g/g, eGFR 30 or higher, and RAS-blockade step-through. No formal net-price data exists because access here runs on step-therapy criteria, not payer negotiation.
Fabrazyme's orphan-drug exclusion under the IRA shields it from Medicare price negotiation entirely. No rebate or net-price figure is disclosed anywhere in the primary record for any Fabry therapy. The orphan exclusion, not a discount ladder, is what a Fabry pricing strategy has to be built around.
Wegovy lists at roughly $1,349 a month, but CMS pays $274 for a 30-day semaglutide supply from 2027. That 71% cut applies to Ozempic, Rybelsus and Wegovy alike. Tirzepatide sits outside the negotiation entirely, for now.
Two IRA negotiation cycles have now cut across the T2D formulary. Januvia down 79% to $113, Jardiance down 66% to $197, Farxiga down 68% to $178 from January 2026, and semaglutide down 71% to $274, with Janumet and Tradjenta added for 2027.
Stelara's negotiated price falls to $4,695 from a $13,836 list, a 66% cut, effective January 2026. That is the same month ustekinumab biosimilars begin launching. Two separate pricing shocks land on one legacy biologic at once.
Iptacopan's ~$550,000 annual WAC sits roughly 71% above ICER's $156,000-157,000 value-based benchmark. The orphan-drug exclusion keeps anti-C5 incumbents outside IRA's reach entirely. Those are the two forces any new US PNH entrant must price against.
Sutimlimab costs $259,000-$302,000 per patient per year. A peer-reviewed analysis puts its ICER at $2.34M/QALY, with standard of care favoured in all 10,000 probabilistic-sensitivity iterations. The value gap, not a rival drug, sets the pricing discipline.
IgA nephropathy sits outside NPHC's genetic/orphan disease scope entirely, so there is no GCC formulary price to model. Budesonide clears case-by-case at SAR 80,000-120,000/year through hospital committees or private insurance; sparsentan is not yet tender-priced at all.
Fintepla's weight-based list price runs roughly 3x Epidiolex. Payers work that gap through step-edit design layered on Part D pharmacy-benefit routing, and no generic cannabidiol reaches the US market before the late 2030s.
Both NHS-commissioned Dravet therapies cleared NICE's standard £20,000-30,000/QALY bar, not the ultra-rare HST threshold. A new entrant is held to the same bar the incumbents already cleared, and total NHS Dravet spend still runs a modest £9-16M.
France prices PNH on two tracks. Ravulizumab holds first-line; iptacopan is reimbursed second-line only, and reached patients through early access two weeks before its EU marketing authorisation took effect.
Tafamidis prices 12-17x above ICER's fair-value benchmark, yet the orphan-drug exclusion keeps it out of IRA negotiation. Acoramidis and pending generics, not Medicare, now set net price.
NICE's TA1121 cost-minimisation rule directs clinicians to whichever ATTR-CM stabiliser costs less. That rule, not the QALY threshold alone, now sets a new entrant's UK price ceiling.
Pompe ERT WAC runs near $400,000 a year, and the Pombiliti + Opfolda regimen splits across Medicare Part B and Part D. Zero ICER reviews exist today, with one expected in 2025 and a 12-month J-code lead time for any new entrant.
Ibrance's Medicare-negotiated price takes effect a full year before Kisqali's and Verzenio's. Three clinically equivalent drugs, staggered IRA negotiation cycles, means Pfizer sets the reference point Novartis and Lilly then have to negotiate against.
ICER priced efgartigimod's value at $18,300-28,400 a year, under half its ~$418,400 assumed launch price. That gap is hardening into a three-tier step-edit staircase across US commercial and Part B plans.
GCC tafamidis pricing is not one number. Private-import pricing near SAR 820,000-850,000/year describes the pre-registration state; post-registration tender pricing near SAR 70,000-90,000 describes what follows.
NHS-commissioned alglucosidase alfa runs £200,000-350,000 per patient a year post-PAS. Avalglucosidase alfa's NICE TA821 recommendation carries a 20-30% WAC premium and a switch-population budget impact of just £2-4M a year.
At 200,000-250,000 GCC patients, US or UK list pricing is commercially impossible. NPHC's own exceptional-access threshold caps a novel agent near SAR 8,000-20,000/year, a fraction of a $2.2M gene-therapy WAC.
No UK gMG biologic has ever cleared NICE at any price. Eculizumab's manufacturer withdrew before submitting an ICER, and efgartigimod's June 2025 rejection means a future entrant's price ladder starts from zero precedent, not a benchmark.
Zolgensma's $2.125M sticker price obscures the real US SMA pricing lever. A 10-state Medicaid outcomes-based annuity pays $212,500 a year for ten years, set against chronic Spinraza and Evrysdi costs and a Part B/Part D routing split that changes patient cost by drug.
NPHC's annual Pompe ERT budget runs SAR 100-160M across 80-120 patients. That is SAR 800K-1.2M for alglucosidase versus SAR 1.2-1.8M for avalglucosidase. A new entrant should target SAR 2.0-2.5M a year, with switch approvals clearing at only 40-60%.
GCC gMG has no single price. Efgartigimod costs SAR 300,000-600,000/yr through private VHI, a different figure through hospital pharmacy committees, and a third through NPHC exceptional access, with a unified formulary price still 12-18 months out.
Every SMA therapy the NHS funds entered through a conditional route. Nusinersen and risdiplam sat in time-limited managed access from 2019 until TA1162 moved them to routine commissioning. UK SMA pricing is a question of how long an asset stays conditional.
Three NICE technology appraisals (TA606, TA738, TA1101) each carry a confidential Patient Access Scheme. Garadacimab's published £20,625 per-pen price is the only fully transparent figure in the class, and two MHRA-licensed agents still have no NICE-confirmed net price.
An editable 12-sheet Excel pricing model and a PDF methodology brief, with a 45-minute analyst call included. No PowerPoint deck — this deliverable is built to be worked in directly, not presented from.
Every reference price, rebate mechanism, and ICER input is cited to a primary regulatory or HTA source — no price point is carried from model memory.
Yes. You set the market basket, comparator class, and WTP threshold; the model is built to your scope and delivered editable.