AXLRx Book a callCommission

02 · Sizing & Access · Analysis type · 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.

Every Market Sizing Model analysis

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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

Market Sizing Model — frequently asked

What formats are included?

An editable 5-sheet Excel sizing 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.

How is the patient count verified?

Every input is triangulated across at least two independent sources (epidemiology-based and claims/registry-based) — no single-source number ships unverified.

Can I size a specific market or subpopulation?

Yes. You set the market, cohort definition, and comparators; the model is built to your scope and delivered editable.