AXLRx Book a callCommission

02 · Sizing & Access · Analysis type · 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.

Every Patient Flow Model 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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

Patient Flow Model — frequently asked

What formats are included?

An editable Excel funnel model, a PDF write-up of the assumptions and result, and a PowerPoint readout, with a 45-minute analyst call included.

How are figures verified?

Every conversion is cited to a primary source and re-runnable; the model shows its working rather than a black-box number.

Can I tailor scope?

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