02 · Sizing & Access · Report type

Patient Flow Model reports, across indications

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.

Patient Flow Model reports across indications

NSCLC
PFM
PFM Oncology Launch LeadCI 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 Read report →
Myasthenia Gravis
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
Myasthenia Gravis
PFM
PFM Rare Disease ForecastingLaunch Lead

UK Myasthenia Gravis Patient Flow Model

12,000-15,000 UK gMG patients on the broader estimate, of whom ~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 Read report →
Sickle Cell Disease
PFM
PFM Rare Disease ForecastingLaunch Lead

US Sickle Cell Disease Patient Flow Model

An estimated 100,000 US sickle cell disease patients narrow to a 55,000-65,000-patient pool with no adequate novel therapy, while only 50-100 of the gene-therapy-eligible minority were actually treated in year one.

US In-Market 24–32 pp PDF · Excel · PPT Read report →
Fabry Disease
PFM
PFM Rare Disease ForecastingLaunch Lead

US Fabry Disease Patient Flow Model

5,000-10,000 diagnosed US classic Fabry patients, a 35-50% amenable-mutation gate to oral therapy, and a treated population 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 Read report →
Myasthenia Gravis
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
Sickle Cell Disease
PFM
PFM Rare Disease ForecastingLaunch Lead

UK Sickle Cell Disease Patient Flow Model

15,000-17,000 diagnosed UK sickle cell disease patients, universal since 1999 newborn screening, 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 Read report →
Fabry Disease
PFM
PFM Rare Disease ForecastingLaunch Lead

UK Fabry Disease Patient Flow Model

700-900 NHS-diagnosed Fabry patients split cleanly: roughly 600 on enzyme replacement, about 200 (25%) on oral migalastat, and free NHS cascade testing that adds 3-4 diagnosed relatives per index case.

UK In-Market 24–32 pp PDF · Excel · PPT Read report →
Dravet Syndrome
PFM
PFM Rare Disease ForecastingLaunch Lead

US Dravet Syndrome Patient Flow Model

6,000-8,000 US Dravet patients, roughly three-quarters SCN1A-confirmed, and 35-40% still inadequately controlled on cannabidiol plus fenfluramine, the population any new agent must actually reach.

US In-Market 24–32 pp PDF · Excel · PPT Read report →
Spinal Muscular Atrophy
PFM
PFM Rare Disease ForecastingLaunch Lead

US Spinal Muscular Atrophy Patient Flow Model

8,000-10,000 US SMA patients, a Type 1-4 severity split running roughly 60/27/13/under 5 percent, and the 500-700-patient Zolgensma cohort this funnel validates against.

US In-Market 24–32 pp PDF · Excel · PPT Read report →
Fabry Disease
PFM
PFM Rare Disease ForecastingLaunch Lead

GCC Fabry Disease Patient Flow Model

GCC male Fabry prevalence runs 1:20,000-30,000, elevated by founder mutations, yet only 200-300 patients are diagnosed against a true burden estimated 3-5 times higher, and female diagnosis lags under half the male rate.

GCC In-Market 24–32 pp PDF · Excel · PPT Read report →
ATTR Amyloidosis
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
Dravet Syndrome
PFM
PFM Rare Disease ForecastingLaunch Lead

UK Dravet Syndrome Patient Flow Model

2,000-2,500 UK Dravet patients, of whom 400-500 are SCN1A-molecularly-confirmed in genetic registries, and 600-900 still inadequately controlled on cannabidiol plus fenfluramine.

UK In-Market 24–32 pp PDF · Excel · PPT Read report →
Hereditary Angioedema
PFM
PFM Rare Disease Launch LeadForecasting

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 Read report →
ATTR Amyloidosis
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
Spinal Muscular Atrophy
PFM
PFM Rare Disease ForecastingLaunch Lead

UK Spinal Muscular Atrophy Patient Flow Model

A ~1,000-patient confirmed UK SMA cohort by type, reconciled against a 1,800-2,000-patient total prevalence estimate, and a 50-60/yr Zolgensma cohort as the on-therapy validation anchor.

UK In-Market 24–32 pp PDF · Excel · PPT Read report →
Dravet Syndrome
PFM
PFM Rare Disease ForecastingLaunch Lead

GCC Dravet Syndrome Patient Flow Model

600-800 estimated GCC Dravet patients, fewer than 200 SCN1A-molecularly-confirmed, and a 200-400 near-term addressable cohort within that confirmed subset.

GCC In-Market 24–32 pp PDF · Excel · PPT Read report →
Hereditary Angioedema
PFM
PFM Rare Disease Launch LeadForecasting

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 Read report →
PNH
PFM
PFM Rare Disease ForecastingLaunch Lead

US PNH Patient Flow Model

15,000-20,000 Americans carry a PNH clone, but only about 3,500 reach complement-inhibitor therapy, and up to 1,200 of them stay anemic on it. This model sizes every gap in between.

US In-Market 24–32 pp PDF · Excel · PPT Read report →
Spinal Muscular Atrophy
PFM
PFM Rare Disease ForecastingLaunch Lead

GCC Spinal Muscular Atrophy Patient Flow Model

GCC SMA incidence of 1:6,000-8,000 births, country-level 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 as the on-therapy anchor.

GCC In-Market 24–32 pp PDF · Excel · PPT Read report →
Hereditary Angioedema
PFM
PFM Rare Disease Launch LeadMarket 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 Read report →
ATTR Amyloidosis
PFM
PFM Rare Disease ForecastingLaunch Lead

GCC ATTR Amyloidosis Patient Flow Model

Two GCC ATTR-CM burden estimates, 15,000-25,000 and a narrower 2,000-5,000 ATTRwt-CM cohort, both convert to fewer than 1,000 confirmed diagnoses. This model reconciles the gap and traces it to scintigraphy access.

GCC In-Market 24–32 pp PDF · Excel · PPT Read report →
PNH
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
Pompe Disease
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
PNH
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
Pompe Disease
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
Pompe Disease
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
Sickle Cell Disease
PFM
PFM Rare Disease ForecastingLaunch Lead

GCC Sickle Cell Disease Patient Flow Model

200,000-250,000 GCC-wide sickle cell disease patients (140,000-200,000 in Saudi Arabia alone), but 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 Read report →
PNH
PFM
PFM Rare Disease ForecastingLaunch Lead

GCC PNH Patient Flow Model

An estimated 2,000-3,000 Gulf patients carry a clinically significant PNH clone, but 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 Read report →
PNH
PFM
PFM Rare Disease ForecastingLaunch 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. Only two national centres, Ulm and Essen, anchor referral.

DE In-Market 24–32 pp PDF · Excel · PPT Read report →
IgA Nephropathy
PFM
PFM Rare Disease ForecastingLaunch 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, since 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 Read report →
IgA Nephropathy
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
IgA Nephropathy
PFM
PFM Rare Disease ForecastingLaunch 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 Read report →
Breast Cancer HR+/HER2-
PFM
PFM Oncology ForecastingLaunch 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 Read report →

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.