AXLRx Book a callCommission

Commercial stage 02

Sizing & Access

How large is the opportunity and who controls access?

Sizing & Access report types

34 published Patient Flow Model An incidence-to-eligible funnel built as a live, re-runnable Excel model: 8 sheets, 119 formulas, zero hardcoded cells. Every conversion step sourced, every assumption editable. PFM · Forecasting · Launch Lead 43 published Payer & HTA Coverage criteria and the evidence standard. How payers and HTA bodies evaluate access and step-edits. P&HTA · Market Access · HTA Lead 38 published Pricing Strategy Model 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. PSM · Market Access · Pricing Lead 37 published Market Sizing Model A static, triangulated patient-count model, not a forecast: 5-sheet workbook (Cover, Model, Research Validation, QC, Sensitivity), every input sourced and re-runnable. Sizing answers how many patients exist today; forecasting is a separate question. MSM · Forecasting · Strategy Lead 11 published HTA Strategy Model A 9-sheet HTA submission-strategy model: authority landscape, PICO framework, comparator defence, value-dossier self-assessment, HEOR gap register, and submission timeline — built before the dossier is written, not after. HTA · Market Access · HEOR Lead

Every Sizing & Access analysis

PFM · Oncology · Launch Lead · CI Team US NSCLC Patient Flow Model Incidence-to-eligible NSCLC funnel by cohort with sourced conversion assumptions and a share waterfall. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Oncology · Market Access · Medical Affairs US NSCLC Payer & HTA CMS routes all four approved 1L NSCLC IO agents through Part B buy-and-bill at ASP+6%. But PD-L1 assay requirements split coverage into three distinct biomarker-testing tiers. US · In-Market · 24–32 pp · PDF · Excel · PPT 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 · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs US IgA Nephropathy Payer & HTA US payers gate the five FDA-approved IgAN therapies on biopsy, proteinuria and RAS-blockade step-through. The Filspari REMS, Part D routing and the ICER 2026 assessment set the rest of the access path. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs US ATTR Amyloidosis Payer & HTA Tafamidis is shielded from IRA negotiation by the orphan-drug exclusion. ICER judged its ~$268K price ~85–95% too high, and acoramidis plus pending generics are the real net-price levers. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs US Hereditary Angioedema Payer & HTA Specialty-tier prior authorization, prophylaxis above $300K/patient/yr, ICER 2018/2021 value-based benchmarks and no-concurrent-acute-agent rules. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · CI Team · Launch Lead · Market Access US Myasthenia Gravis Payer & HTA ICER priced efgartigimod's value at $18,300–28,400 a year, under half its ~$418,400 launch cost. That gap is hardening into a three-tier FcRn-to-C5 step-edit across US commercial plans. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs US Gaucher Disease Payer & HTA IV enzyme replacement buy-and-bill under Part B vs oral SRT under Part D, the CYP2D6 PA gate, and generic miglustat. US · In-Market · 24–32 pp · PDF · Excel · PPT 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 · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs US Spinal Muscular Atrophy Payer & HTA Zolgensma's $2.125M one-time cost, outcomes-based Medicaid contracts, and Part B vs Part D routing across three SMA modalities. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs US Cold Agglutinin Disease Payer & HTA Sutimlimab (Enjaymo) is a ~$260K+/year Part B IV biologic in a few-thousand-patient population — and not cost-effective at the current price. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs US Dravet Syndrome Payer & HTA Fintepla's list price runs roughly 3x Epidiolex, and payer scrutiny turns on high WAC against a small, severe paediatric population. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs US Pompe Disease Payer & HTA Pompe ERT costs near $400,000 a year in Part B, and remains the only major rare-disease ERT category ICER has never reviewed. Pombiliti + Opfolda splits into two simultaneous prior authorisations across Part B and Part D. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs US Fabry Disease Payer & HTA A genetic test gates oral migalastat, and IV enzyme replacement sits in Part B. Fabrazyme has no US biosimilar, and the IRA's orphan-drug exclusion shields it from Medicare price negotiation. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Immunology · Market Access · Medical Affairs US Atopic Dermatitis Payer & HTA Topical-then-biologic step-therapy, JAK black-box PA gates, ICER's dupilumab-aligned and JAK-discount verdicts, and why Dupixent is not IRA-selected. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Metabolic · Market Access · Medical Affairs US MASH Payer & HTA Why Rezdiffra's $47,400 WAC lands inside ICER's value range, why the IRA reset hits semaglutide first, and how Part D routing shapes MASH access. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Metabolic · Market Access · Medical Affairs US Type 2 Diabetes Payer & HTA Type 2 Diabetes is IRA ground zero: three orals negotiated for 2026, semaglutide at $274 for 2027, and the class price anchor reset. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Neurology · Market Access · Medical Affairs US Alzheimer's Disease Payer & HTA CMS's coverage-with-evidence-development registry, not IRA negotiation, is the anti-amyloid access gate. ICER's below-value verdict and Part B routing set the rest. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Metabolic · Market Access · Medical Affairs US Obesity Payer & HTA Medicare covers Wegovy only for cardiovascular risk, not obesity alone. The IRA's IPAY 2027 semaglutide price applies across the franchise, and ICER returned a 2025 'high value' verdict. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Pulmonology · CI Team · Launch Lead US COPD Payer & HTA COPD access is a pharmacy-benefit story: inhalers and biologics run through Medicare Part D and commercial PBMs, not medical coverage. Step edits gate the base, an eosinophil threshold gates the biologic, and the IRA is reshaping both price exposure and negotiation risk. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Immunology · CI Team · Launch Lead US Plaque Psoriasis Payer & HTA US access to plaque psoriasis biologics is gated by step therapy and reshaped by two forces landing together. IRA price negotiation on Stelara and Enbrel, and biosimilar erosion of adalimumab and ustekinumab. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Oncology · CI Team · Launch Lead US Breast Cancer HR+/HER2- Payer & HTA The price ceiling for HR+/HER2- oral therapies is now set directly by the government. Palbociclib was selected for Medicare negotiation with a 50% cut, $15,741 to $7,871, effective 2027. Access runs through Part D and commercial prior authorization, and newer targeted agents face biomarker-gated coverage with cost-effectiveness ratios far above accepted thresholds. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Payer Hta FR PNH Payer & HTA France reimburses iptacopan second-line only, after at least six months on a C5 inhibitor, while ravulizumab holds the first-line position. The restriction, not the price, is what defines the addressable population. FR · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing UK Spinal Muscular Atrophy Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US Myasthenia Gravis Market Sizing Model US gMG prevalence runs 100,000-200,000, but only 4,000-6,000 patients are on FcRn therapy today. 1,200-2,100 of those remain inadequately controlled. The near-term opportunity is the funnel gap, not the epidemiology total. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Pricing · Medical Affairs UK Hereditary Angioedema Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC Myasthenia Gravis Payer & HTA Generalised myasthenia gravis has no formal NPHC programme. Efgartigimod access runs through private insurance, fastest at 1-4 weeks, or hospital pharmacy committees, with NPHC engagement targeted for 2025-2026. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC Sickle Cell Disease Market Sizing Model Carrier-rate-adjusted epidemiology implies 140,000-200,000 KSA sickle cell patients. NPHC's structured active-management programme reaches only 8,000-10,000 — a care-registration gap, not a measurement error. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC Pompe Disease Market Sizing Model Total GCC Pompe prevalence runs 400-600, consanguinity-elevated. 200-300 are actively managed on ERT, and NPHC's formulary budget centers on a narrower 80-120 long-term-stable core within that population. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US Spinal Muscular Atrophy Market Sizing Model US SMA sizing splits into two live populations, not one number. 8,000-10,000 prevalent patients across Types 1-4, and a separate 500-700-patient Zolgensma-treated cohort now aging into a monitoring window where 15-20% show early motor plateau. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US Hereditary Angioedema Market Sizing Model An estimated 8,000-9,000 Americans have HAE, and only 35-40% receive any prophylaxis. That leaves 2,500-4,000 attack-eligible patients never treated, a pool nearly as large as the entire treated population. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK Spinal Muscular Atrophy Market Sizing Model The UK SMA population is not one number. A ~1,000-patient actively-monitored NHS cohort and a 1,800-2,000-patient total prevalence estimate both appear across UK sources, and the gap between them is the pre-NBS legacy population outside the four specialist networks' active census. UK · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK Hereditary Angioedema Market Sizing Model A 37-centre national survey confirms 1,152 UK HAE type I/II patients. A top-down 1:32,000 prevalence rate implies roughly 2,000, and the UK HAE Alliance's broader planning estimate runs to 5,000-6,000, of which only 1,500-2,000 are on prophylaxis today. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC PNH Payer & HTA NPHC's KSA-first coverage model sets the de facto GCC access bar for anti-C5 agents. Iptacopan faces a 12-24 month SFDA registration queue before NPHC even evaluates it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC Hereditary Angioedema Payer & HTA GCC HAE access is structurally two-tier: broad acute coverage, but a prophylaxis bar few clear. Only 30-40% of applicants clear NPHC's individual-case prophylaxis review, and private insurance beats the NPHC pathway on speed. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead US Myasthenia Gravis Patient Flow Model 100,000-200,000 US gMG patients narrow to a 4,000-6,000 on-FcRn-therapy pool, and 1,200-2,100 of them remain inadequately controlled despite treatment. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK IgA Nephropathy Payer & HTA NICE's accepted cost-effectiveness case for budesonide (TA937, updated by TA1128) rests on a 5–8 year modelled ESRD delay. The same mandatory ACEi/ARB gate applied to sparsentan narrows the UK's eligible IgA nephropathy population from 10,000–15,000 to 3,000–5,000 patients. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead GCC Myasthenia Gravis Patient Flow Model 6,000-10,000 GCC gMG patients on the epidemiology estimate, of whom 200-300 are refractory and fewer than 50 are currently on biologic therapy. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead UK Sickle Cell Disease Patient Flow Model Newborn screening has been universal since 1999, so the UK's 15,000-17,000 diagnosed sickle cell patients are counted with confidence. They narrow to a 4,000-6,000-patient conventional-therapy gap and a separate 200-300-per-year gene-therapy-eligible pool. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead UK Fabry Disease Patient Flow Model 700-900 NHS-diagnosed Fabry patients split cleanly by therapy. Roughly 600 are on enzyme replacement and about 200, or 25%, on oral migalastat, with free NHS cascade testing adding 3-4 diagnosed relatives per index case. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing DE PNH Pricing Strategy 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. DE · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK PNH Payer & HTA Both ravulizumab and iptacopan cleared NICE's standard Technology Appraisal route (TA698 and TA1000). The real payer question is how fast the NHS converts patients from IV ravulizumab to oral iptacopan, not which drug got the easier appraisal at the ordinary £20,000–£30,000/QALY bar. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead US Spinal Muscular Atrophy Patient Flow Model 8,000-10,000 US SMA patients split roughly 60/27/13/under 5 percent across Types 1-4. The 500-700-patient Zolgensma cohort is what this funnel validates against. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC Sickle Cell Disease Payer & HTA The GCC's largest rare-disease programme by patient volume sits in a commercial vacuum. Crizanlizumab and voxelotor are both withdrawn, leaving 8,000-10,000 NPHC-managed SCD patients ahead of 2025-26 gene therapy registration. GCC · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Oncology · Medical Affairs · Heor UK Breast Cancer HR+/HER2- HTA Strategy Model NICE accepts palbociclib, ribociclib, and abemaciclib as comparators for each other. No trial has ever tested any of them head-to-head against exemestane plus everolimus, the older endocrine-based comparator regimen they effectively replaced. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC Fabry Disease Payer & HTA NPHC has a 72%-cost-reduction incentive to switch amenable-mutation Fabry patients from ERT to migalastat. A single HEK293 assay lab in the entire GCC is the only thing standing in the way. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Launch Lead · Forecasting US Hereditary Angioedema Patient Flow Model An estimated 8,000-9,000 Americans live with hereditary angioedema. Just 35-40% receive any prophylaxis, leaving 2,500-4,000 patients who meet treatment criteria untreated, the funnel this model sizes precisely. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK Pompe Disease Market Sizing Model The UK Pompe Consortium registry counts roughly 200 confirmed patients. Total estimated prevalence, including the undiagnosed pool, runs 350-450. Within the confirmed, treated population, 30-50 are ADA-positive inadequate responders and 80-120 are on home ventilation. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead GCC Dravet Syndrome Patient Flow Model 600-800 estimated GCC Dravet patients, fewer than 200 SCN1A-confirmed. A 200-400 near-term addressable cohort sits within that confirmed subset. GCC · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK Spinal Muscular Atrophy Payer & HTA All three funded SMA therapies in England reached the NHS through a conditional route. Zolgensma was recommended under HST15 and extended under HST24; nusinersen and risdiplam spent seven years in managed access before TA1162 moved them to routine commissioning. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead US PNH Patient Flow Model 15,000-20,000 Americans carry a PNH clone, but only about 3,500 reach complement-inhibitor therapy. Up to 1,200 of them stay anaemic on it. This model sizes every gap in between. US · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Launch Lead · Market Access GCC Hereditary Angioedema Patient Flow Model Epidemiology projects 1,200-1,500 GCC HAE patients; the GCC allergy society's own case registry counts only 400-600. The gap is not a contradiction, it is the diagnostic-capacity constraint of just 6-10 specialist physicians across all six states. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead UK PNH Patient Flow Model ~600-750 UK PNH patients are on active complement-inhibitor therapy against a reconciled total prevalence near 1,500. The other 750-900 are monitored-only or undiagnosed, and this model sizes the gap. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead UK Pompe Disease Patient Flow Model 350-450 UK Pompe disease patients, of whom roughly 200 form the registry-confirmed cohort. One in four long-term ERT patients is not holding stable, the segment this model is built to size. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead FR PNH Patient Flow Model France's PMSI hospital database counts 897 PNH patients over five years; Orphanet's older estimate puts national prevalence at 850-1,000. Only 270 of them, per the manufacturer's own estimate, are eligible for a second-line oral agent. FR · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead GCC Pompe Disease Patient Flow Model 400-600 GCC Pompe disease patients, of whom 200-300 are on enzyme replacement therapy. Just 40-60, patients with FVC decline despite alglucosidase already on home ventilation, are the segment this model is built to size. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing UK PNH Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK Pompe Disease Payer & HTA NHS England's Pompe commissioning policy defines a 45-50 patient switch-eligible cohort for avalglucosidase alfa (NICE TA821). That is a manageable NHS budget event; broader first-line uptake would be a materially larger one. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK Fabry Disease Payer & HTA Migalastat's HST4 recommendation in 2016 was the first oral mutation-specific rare disease therapy NICE approved. Agalsidase beta was never formally appraised at all. Together they underpin an estimated £144M NHS Fabry programme, with a £70-130K/patient/year switch incentive still unrealised at scale. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK ATTR Amyloidosis Payer & HTA NICE rejected tafamidis for ATTR-CM in May 2021 (TA696) and recommended it in June 2024 (TA984). Acoramidis (TA1121, January 2026) and vutrisiran (TA868 for ATTR-PN, TA1115 for ATTR-CM) complete the UK amyloidosis market. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing UK Fabry Disease Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing UK IgA Nephropathy Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing GCC PNH Pricing Strategy Model 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy FR PNH Market Sizing Model France's PMSI hospitalisation database identified 897 PNH patients between 2018 and 2022, putting prevalence near 1 in 94,000. That is below the 1-in-70,000-to-80,000 range Orphanet and France's national rare disease plan have long cited. FR · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing GCC Fabry Disease Pricing Strategy Model 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 · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US Fabry Disease Market Sizing Model Roughly 5,000-10,000 diagnosed US Fabry patients split first by GLA amenability, with 35-50% oral-eligible. ADA status narrows that to a precise 200-400 patient addressable niche, which a Fabrazyme-anchored $250-350K WAC makes commercially calculable. US · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK Myasthenia Gravis HTA Strategy Model NICE has never modelled a cost-per-QALY for a myasthenia gravis biologic. Eculizumab's appraisal (TA636) closed before a dossier was submitted; efgartigimod's (TA1069) closed on evidence gaps, not a quantified ICER breach. A new entrant inherits no reusable comparator or price benchmark from either. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US Sickle Cell Disease Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC Spinal Muscular Atrophy Payer & HTA SMA is the most mature rare-disease access model in the GCC. All three modalities are NPHC-covered, with Zolgensma's outcomes-based milestone rebate the GCC-first template other programmes follow. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US Dravet Syndrome Market Sizing Model US Dravet incidence of 1 in 15,700 births is consistent with the Dravet Syndrome Foundation's 6,000-8,000 prevalence estimate. Only 35-40% of that population, 1,400-2,000 patients, remains inadequately controlled on today's two branded agents. US · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK Dravet Syndrome HTA Strategy Model Both existing Dravet therapies cleared NICE's standard Technology Appraisal, not the ultra-rare Highly Specialised Technology route. A new entrant's WAC, PAS, and stakeholder-engagement calendar all need to be built against that lower cost-effectiveness bar, with soticlestat's 2026-27 appraisal setting the clock. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Pricing · Medical Affairs US Hereditary Angioedema Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing UK Sickle Cell Disease Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing GCC Spinal Muscular Atrophy Pricing Strategy Model 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Pricing · Medical Affairs GCC Hereditary Angioedema Pricing Strategy Model 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC Spinal Muscular Atrophy Market Sizing Model GCC SMA incidence runs 1:6,000-8,000 against a global 1:10,000. Newborn-screening coverage splitting 90%/85%/75% across KSA, UAE and Qatar means the addressable near-term population depends on which country's screening curve a launch model assumes. GCC · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK Myasthenia Gravis Payer & HTA NICE's June 2025 rejection of efgartigimod (TA1069) leaves UK myasthenia gravis with no NICE-recommended novel agent. Eculizumab's own appraisal (TA636) was withdrawn by the manufacturer in 2020 without a cost-effectiveness verdict, and the NHS IVIg cost-offset argument is now the strongest lever for a future resubmission. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead US Fabry Disease Patient Flow Model 5,000-10,000 diagnosed US classic Fabry patients face a 35-50% amenable-mutation gate to oral therapy. The treated population is still 60-65% on enzyme replacement. This funnel starts at diagnosis because no defensible undiagnosed estimate exists yet. US · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead UK Myasthenia Gravis Patient Flow Model The broader estimate puts 12,000-15,000 UK patients with generalised myasthenia gravis. About 4,000 have moderate-severe disease and 2,000-3,000 are refractory, with no NICE-recommended novel agent for any of them. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC ATTR Amyloidosis Payer & HTA GCC tafamidis costs roughly a tenth of its US price, yet uptake is not limited by affordability. Tc-PYP scintigraphy access at fewer than 8 GCC centres is the real constraint. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead UK Dravet Syndrome Patient Flow Model 2,000-2,500 UK Dravet patients, of whom 400-500 are SCN1A-confirmed in genetic registries. 600-900 remain inadequately controlled on cannabidiol plus fenfluramine. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead GCC Fabry Disease Patient Flow Model GCC male Fabry prevalence runs 1:20,000-30,000, elevated by founder mutations. Only 200-300 patients are diagnosed against a true burden estimated 3-5 times higher, and female diagnosis lags at under half the male rate. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead GCC Spinal Muscular Atrophy Patient Flow Model GCC SMA incidence runs 1:6,000-8,000 births, with newborn-screening coverage from 90% down to under 50%. An 800-1,200-patient legacy Type 2/3 pool and a 60-80/yr Zolgensma cohort anchor the on-therapy view. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead UK Spinal Muscular Atrophy Patient Flow Model A ~1,000-patient confirmed UK SMA cohort by type reconciles against a 1,800-2,000-patient total prevalence estimate. A 50-60/yr Zolgensma cohort is the on-therapy validation anchor. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead GCC ATTR Amyloidosis Patient Flow Model Two GCC ATTR-CM burden estimates both convert to fewer than 1,000 confirmed diagnoses. One puts the range at 15,000-25,000, the other at a narrower 2,000-5,000 ATTRwt-CM cohort. This model reconciles the gap and traces it to scintigraphy access. GCC · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK Hereditary Angioedema Payer & HTA NICE TA606 recommended lanadelumab in 2019 with a confidential simple-discount patient access scheme, and the first national UK audit found total attacks fell 87.7% at 6 months. Berotralstat's TA738 recommendation is measured against that benchmark. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC Pompe Disease Payer & HTA NPHC's well-established Pompe programme still gates avalglucosidase behind a 12-month failed-response switch criterion. Sanofi is pursuing NPHC first-line approval to bypass it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK ATTR Amyloidosis Market Sizing Model National Amyloidosis Centre-anchored epidemiology puts UK ATTRwt-CM prevalence at 20,000-40,000. Only 3,000-4,000 patients are on NICE-commissioned tafamidis today, leaving 15,000-36,000 undiagnosed. UK · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Oncology · Forecasting · Strategy US Breast Cancer HR+/HER2- Market Sizing Model 316,950 annual US invasive breast cancer diagnoses and a 6.0% metastatic-at-diagnosis rate size the incident flow. Layering CDK4/6-inhibitor and post-progression pricing onto that flow, and onto the separate, larger recurrence pool, is what turns a patient count into a market value. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Metabolic · Forecasting · Strategy US Type 2 Diabetes Market Sizing Model IQVIA puts the 2023 US T2D drug market at $22B top-down. Triangulated bottom-up against 29.7M diagnosed patients out of 38.4M with the disease, the two methods converge, but per-class revenue split remains an open gap this model flags rather than invents. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US Gaucher Disease Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead UK ATTR Amyloidosis Patient Flow Model True UK ATTRwt-CM prevalence runs 20,000-40,000, and 15,000-36,000 of those patients remain undiagnosed. Only 3,000-4,000 are on NHS-commissioned tafamidis today, adding 1,500-2,000 a year. UK · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy DE PNH Market Sizing Model Germany's DGHO Onkopedia guideline states plainly that Germany-specific PNH prevalence and incidence figures do not exist. It borrows 16 cases and 1.3 new diagnoses per million annually from British and French registries, and routes all case-finding through just two national centres, Ulm and Essen. DE · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead GCC IgA Nephropathy Patient Flow Model An estimated 8,000-12,000 GCC IgAN patients narrow to fewer than 30% ever biopsied, then to zero on novel therapy. No agent is SFDA-registered as of 2024. The funnel gap, not the prevalence estimate, is what a GCC patient flow model has to size. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead GCC PNH Patient Flow Model An estimated 2,000-3,000 Gulf patients carry a clinically significant PNH clone. Only about 400 are confirmed in national registries, and just 150-250 reach complement-inhibitor therapy. Diagnostic capacity, not drug access, is the constraint this model sizes. GCC · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK Pompe Disease HTA Strategy Model NICE accepts a £100,000-300,000 QALY threshold for ultra-rare Pompe disease, five to fifteen times the standard appraisal bar. That Highly Specialised Technologies ceiling is what avalglucosidase alfa's TA821 recommendation used, but only with a substantial confidential commercial arrangement. A new entrant without that leverage should build a standalone case for the antibody-positive inadequate-responder subgroup instead. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing GCC Dravet Syndrome Pricing Strategy Model 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC Dravet Syndrome Market Sizing Model GCC Dravet prevalence is estimated at 600-800 patients, but fewer than 200 are molecularly SCN1A-confirmed. The 200-400 figure used in launch planning is a distinct near-term actionable tier, not a fourth competing total. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC ATTR Amyloidosis Market Sizing Model GCC ATTR-CM burden runs 15,000-25,000 on a broad HFpEF-adjacent estimate, or 2,000-5,000 on the narrower ATTRwt-CM cohort. Fewer than 8 centres with scintigraphy capacity explain why so little of either total converts to a confirmed diagnosis. GCC · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK ATTR Amyloidosis HTA Strategy Model NICE recommends both ATTR-CM stabilisers, tafamidis (TA984) and acoramidis (TA1121), and directs clinicians to the cheaper one. Acoramidis cleared on indirect comparison alone. A third entrant must beat an invisible, PAS-discounted floor, with no published price target. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US IgA Nephropathy Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead GCC Sickle Cell Disease Patient Flow Model 200,000-250,000 GCC sickle cell patients, with 140,000-200,000 in Saudi Arabia alone. NPHC's actively-managed registry reaches only 8,000-10,000 — the addressable near-term funnel stage within a much larger under-managed population, not a contradiction. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead UK IgA Nephropathy Patient Flow Model 10,000-15,000 UK IgA nephropathy patients, of whom approximately 8,000 are tracked in the UK Renal Registry. Only 3,000-5,000 clear the mandatory ACEi/ARB and SGLT2i optimisation gate to become eligible for a novel agent, and fewer than 500 currently access one pre-NICE. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead US Dravet Syndrome Patient Flow Model 6,000-8,000 US Dravet patients, roughly three-quarters SCN1A-confirmed. 35-40% are still inadequately controlled on cannabidiol plus fenfluramine, the population any new agent must actually reach. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK Myasthenia Gravis Market Sizing Model Two UK gMG estimates disagree by 3-4x on purpose. A narrower moderate-severe subgroup of roughly 4,000 from the MGA UK survey sits inside a broader 12,000-15,000 total prevalence figure that also counts mild, well-controlled cases the narrower estimate excludes. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Metabolic · Market Access · Medical Affairs · Pricing US Obesity Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Metabolic · Market Access · Medical Affairs · Pricing US Type 2 Diabetes Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Immunology · Market Access · Medical Affairs · Pricing US Plaque Psoriasis Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US Cold Agglutinin Disease Market Sizing Model US cold agglutinin disease sizing starts from a rate range, not a point estimate. Incidence 0.6-1.2 and 1-year prevalence 1.4-3.1 per 100,000 imply ~5,000 prevalent patients. No published treated-share figure exists to split served from total addressable. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK Dravet Syndrome Market Sizing Model The UK's NICE-commissioned Dravet algorithm manages an estimated 2,000-2,500 patients on cannabidiol and fenfluramine. The NHS genetic testing registry logs only 400-500 molecularly SCN1A-confirmed cases — a registry-scope gap, not a population contradiction. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK Dravet Syndrome Payer & HTA NICE recommended both Dravet therapies through standard Technology Appraisal, not the ultra-rare HST route. This covers what the Fintepla Cardiac Monitoring Scheme costs the NHS, and why the UK treatment algorithm is now closed to new entrants without a significant clinical edge. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US PNH Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK PNH Market Sizing Model The Leeds National PNH Registry tracks about 600 UK patients on complement-inhibitor therapy. A broader estimate cited in AXLRx's own Launch Readiness research puts it near 1,500 total patients and 1,000 treated. Reconciling which count anchors a sizing model is the open question. UK · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK Spinal Muscular Atrophy HTA Strategy Model NICE built UK SMA access in sequence, not as an open field. Gene therapy took the pre-symptomatic subgroup first (HST15, HST24), then TA1162 moved chronic therapy to routine funding behind it. A new entrant inherits a fixed subgroup hierarchy and a comparator that shifts by population. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US Cold Agglutinin Disease Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US Fabry Disease Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US Gaucher Disease Market Sizing Model 6,000 US Type 1 Gaucher patients size a market near $1.8 billion at ERT pricing. That MedlinePlus-sourced population sits inside a genetic sub-segment where Ashkenazi carrier frequency runs 1 in 12-15, against a general-population disease frequency of just 0.70-1.75 per 100,000. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC Hereditary Angioedema Market Sizing Model Epidemiology implies 1,200-1,500 true GCC HAE patients, but the KFSH&RC registry confirms fewer than 200. A separate planning estimate used for launch work lands at 400-600 — three numbers, one diagnostic-capacity story. GCC · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK Sickle Cell Disease HTA Strategy Model NICE recommended crizanlizumab (TA743) via managed access in 2021, then withdrew it in 2023 after the confirmatory trial failed. Casgevy (TA1044) cleared only by restructuring its £1.65M price into managed access. A new entrant inherits no reusable ICER benchmark from either. UK · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK Hereditary Angioedema HTA Strategy Model Three completed NICE standard technology appraisals already fund HAE prophylaxis in England (TA606, TA738, TA1101). Every one cleared at the ordinary £20,000 to £30,000 per QALY bar and is held behind a confidential Patient Access Scheme, so a new entrant inherits a comparator-dense field in which garadacimab's £20,625 list pen is the only transparent price. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing GCC IgA Nephropathy Pricing Strategy Model 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Payer Hta DE PNH Payer & HTA Iptacopan's orphan-drug status let it clear Germany's AMNOG process with an established additional benefit and no comparator dossier at all. Ravulizumab's only PNH-specific G-BA review found no added benefit. DE · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US PNH Market Sizing Model US PNH prevalence spans 15,000 to 20,000 patients across three phenotypes, but only about 3,500 are on complement-inhibitor therapy. A 2.4-year average diagnostic delay, plus 200 to 400 patients a year undertreated at non-PNH centres, accounts for most of that gap. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US Dravet Syndrome Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC IgA Nephropathy Market Sizing Model A GCC nephrology network capacity survey estimates 8,000-12,000 IgA nephropathy patients across the region. The same survey finds kidney biopsy performed in fewer than 30% of eligible proteinuric patients, and zero patients on any SFDA-registered novel agent as of 2024. GCC · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK Sickle Cell Disease Market Sizing Model Near-universal newborn screening puts the UK's 15,000-17,000 SCD patients on the registry with confidence. But only the 4,000-6,000 hydroxycarbamide-inadequate subset is the addressable population for a new non-gene agent. UK · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK IgA Nephropathy HTA Strategy Model NICE accepted an eGFR-slope-to-ESRD-delay model, not headline proteinuria reduction, as the value basis in IgA nephropathy. That covers budesonide (TA937, expanded by TA1128) and sparsentan (TA1074). The mandatory ACEi/ARB and SGLT2 inhibitor optimisation gate narrows the UK's 10,000 to 15,000 patients to a 3,000 to 5,000 novel-agent-eligible pool before that pricing math applies. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead US ATTR Amyloidosis Patient Flow Model 500,000+ US patients aged 70+ with HFpEF carry undiagnosed ATTRwt-CM, against only 70,000-100,000 diagnosed and treated. Two further sub-populations, Val122Ile carriers and ATTR-PN, remain even less visible. US · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead US IgA Nephropathy Patient Flow Model 150,000 US IgA nephropathy patients, of whom 70,000-90,000 are biopsy-confirmed. Only 5,000-8,000 are on novel therapy today, while 15,000-25,000 patients with UPCR above 1g/g remain the addressable high-risk cohort this model sizes precisely. US · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs UK Sickle Cell Disease Payer & HTA NICE's SCD gene therapy appraisal may be the largest NHS rare disease budget event in history. It hinges on an annuity payment model that current NHS SCD management cost cannot yet clearly justify. UK · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US IgA Nephropathy Market Sizing Model An estimated 150,000 Americans have IgA nephropathy, but only 70,000-90,000 are biopsy-confirmed. Just 5,000-8,000 are on a disease-specific therapy today, within a 15,000-25,000-patient high-risk eligible cohort. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing UK Dravet Syndrome Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK IgA Nephropathy Market Sizing Model The UK Renal Registry tracks about 8,000 IgA nephropathy patients against an estimated 10,000-15,000 total prevalence. Only 3,000-5,000 are eligible for a novel agent, and fewer than 500 currently receive one. UK · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US Pompe Disease Market Sizing Model US Pompe prevalence runs 5,000 to 10,000 patients, 70-80% late-onset. Of the roughly 2,000 LOPD patients on enzyme replacement therapy, 375 to 600 are inadequate responders, the addressable population any new agent must reach. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing FR PNH Pricing Strategy Model 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. FR · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US ATTR Amyloidosis Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Metabolic · Forecasting · Strategy US MASH Market Sizing Model 6.7 million Americans have F2-F3 MASH, but Rezdiffra has already reported 42,250+ patients on therapy and $311.3M in Q1 2026 revenue. This model triangulates population against real uptake, not a modeled guess. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC PNH Market Sizing Model Saudi Arabia's rare disease registry counts about 400 confirmed PNH cases. Global prevalence rates, adjusted for the region's 25-50% consanguinity rate, imply a true GCC PNH population 20-30% higher, and fewer than 15 labs across six countries can even run the diagnostic test. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead US Sickle Cell Disease Patient Flow Model An estimated 100,000 US sickle cell patients narrow to a 55,000-65,000-patient pool with no adequate novel therapy. Only 50-100 of the gene-therapy-eligible minority were actually treated in year one. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy UK Fabry Disease Market Sizing Model 700-900 diagnosed UK Fabry patients split roughly 600 on ERT to 200 on oral therapy. Two niches sit inside that: 50-80 ADA-positive suboptimal responders and 80-120 undertreated symptomatic female heterozygotes, against a £144M NHS spend anchor. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing UK ATTR Amyloidosis Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Launch Lead · Forecasting UK Hereditary Angioedema Patient Flow Model The UK HAE Alliance counts 5,000-6,000 total patients, of whom 1,500-2,000 are on NICE-commissioned prophylaxis. A further 1,500-2,500 are attack-active but never treated, and Longhurst et al.'s 37-centre registry confirms 1,152 patients from the bottom up. UK · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC IgA Nephropathy Payer & HTA IgA nephropathy has no NPHC programme at all in the GCC. Access runs entirely through hospital pharmacy committees or private insurance, gated by a biopsy available at fewer than 20 GCC centres. GCC · In-Market · 24–32 pp · PDF · Excel · PPT P&HTA · Rare Disease · Market Access · Medical Affairs GCC Dravet Syndrome Payer & HTA The most effective Dravet agent is the least accessible in the GCC. Cannabidiol's Schedule-1-equivalent narcotics classification caps exceptional-import approval at 35-40%. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead US Pompe Disease Patient Flow Model 5,000-10,000 Americans live with Pompe disease. Roughly 2,000 late-onset patients are on enzyme replacement therapy, and 375-600 of them, one in four, are inadequate responders, the subtype this model is built to size. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US Pompe Disease Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC Fabry Disease Market Sizing Model 200-300 diagnosed GCC Fabry patients sit against a true prevalence estimated 3-5 times higher. A single regional laboratory gates the amenable-mutation test, and female heterozygotes are diagnosed at under half the male rate. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Rare Disease · Forecasting · Launch Lead DE PNH Patient Flow Model Germany has no domestic PNH prevalence count of its own. DGHO's Onkopedia guideline borrows a 16-per-million estimate from British and French registries, and only two national centres, Ulm and Essen, anchor referral. DE · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK Fabry Disease HTA Strategy Model UK Fabry disease is commissioned through three different NICE and NHS routes at once. No formal technology appraisal for either enzyme replacement therapy, a Highly Specialised Technologies recommendation (HST4, 2016) for migalastat, and a standard appraisal (TA915, 2023) for pegunigalsidase alfa. A new entrant inherits no single reusable comparator or price benchmark. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US Myasthenia Gravis Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Oncology · Medical Affairs · Pricing US Breast Cancer HR+/HER2- Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing GCC ATTR Amyloidosis Pricing Strategy Model 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT HTA · Rare Disease · Medical Affairs · Heor UK PNH HTA Strategy Model NICE has cleared all four modern PNH anti-complement therapies through its standard £20,000-30,000 Technology Appraisal route. Ravulizumab TA698, pegcetacoplan TA778, iptacopan TA1000 and crovalimab TA1019, each contingent on a confidential commercial arrangement, never the Highly Specialised Technologies threshold. A new submission inherits an unbroken standard-STA precedent it must be built to clear from the first dossier decision. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing UK Pompe Disease Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing GCC Sickle Cell Disease Pricing Strategy Model 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PFM · Oncology · Forecasting · Launch Lead US Breast Cancer HR+/HER2- Patient Flow Model 316,950 new US invasive breast cancer diagnoses in 2025. Only 6.0% present as metastatic at diagnosis, but 73.9% of the metastatic population is HR+/HER2-, the subtype this model is actually built to size. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing UK Myasthenia Gravis Pricing Strategy Model 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US Sickle Cell Disease Market Sizing Model A ~100,000-patient US SCD population narrows to a 55,000-65,000-patient addressable white space. Only 50-100 gene-therapy patients were actually treated in the first 12 months, versus 200-300 projected. US · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy GCC Myasthenia Gravis Market Sizing Model GCC gMG sizing treats the 6,000-10,000-patient disease-landscape prevalence estimate as the authoritative broad base. A 200-300-patient refractory subgroup, fewer than 50 currently on a biologic, is the narrower actionable segment inside it, not a competing total. GCC · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing US Spinal Muscular Atrophy Pricing Strategy Model 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. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing GCC Pompe Disease Pricing Strategy Model 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 · In-Market · 24–32 pp · PDF · Excel · PPT MSM · Rare Disease · Forecasting · Strategy US ATTR Amyloidosis Market Sizing Model An estimated 500,000+ US patients aged 70+ have undiagnosed ATTRwt-CM, against only 70,000-100,000 currently diagnosed and treated. A separate 100,000+ Val122Ile hereditary carrier pool sits alongside it. US · In-Market · 24–32 pp · PDF · Excel · PPT PSM · Rare Disease · Medical Affairs · Pricing GCC Myasthenia Gravis Pricing Strategy Model 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

Other stages

01 · Market Intelligence03 · Commercial Planning04 · Analytics & Operations