Key takeaways
- 20K-40K estimated true UK ATTRwt-CM prevalence
- 3K-4K UK patients currently on NICE-commissioned tafamidis (TA984), growing 1,500-2,000/year
- 15K-36K estimated undiagnosed UK ATTRwt-CM patients, the gap between epidemiology and registry counts
- ~50 NHS cardiac centres offering Tc-PYP scintigraphy — the diagnostic bottleneck capping how fast the addressable population grows
UK ATTRwt-CM prevalence is estimated at 20,000-40,000, but only 3,000-4,000 patients are on NICE-commissioned tafamidis today, leaving 15,000-36,000 undiagnosed.
Two independent methods size the UK ATTR cardiomyopathy population, and the National Amyloidosis Centre's data anchors both. The epidemiology method estimates true UK ATTRwt-CM prevalence at 20,000 to 40,000 patients, based on the same age-related wild-type mechanism driving the disease elsewhere, adjusted for the UK's demographic profile. The registry method counts confirmed, NHS-commissioned treatment: 3,000 to 4,000 patients are on tafamidis (Vyndaqel) under NICE TA984, the appraisal that reversed NICE's 2021 TA696 rejection, and that treated population is growing 1,500 to 2,000 patients a year as the NHS Tc-PYP scintigraphy referral pathway scales. Triangulating the two methods identifies a gap of 15,000 to 36,000 undiagnosed patients, not a rounding error between two versions of the same number.
That gap has a specific, tractable cause: NHS Tc-PYP nuclear cardiology capacity. Scintigraphy is available at only around 50 of the relevant NHS cardiac centres, and referral runs from HFpEF with increased echo wall thickness through the National Amyloidosis Centre's confirmation pathway before a patient reaches NICE-commissioned treatment. Our sensitivity analysis ranks diagnostic referral-pathway capacity above the underlying prevalence estimate as the assumption most likely to move the sized total over the next three years.
UK ATTR sizing — epidemiology-based prevalence versus NICE-registry treated population
| Sizing Method | Population Estimate | Source |
|---|---|---|
| Epidemiology-based (true ATTRwt-CM prevalence) | 20,000-40,000 patients | National Amyloidosis Centre prevalence modelling |
| Registry-based (NICE TA984-commissioned treatment) | 3,000-4,000 patients (+1,500-2,000/yr) | NHS England ATTR-CM commissioning documentation |
| Estimated undiagnosed share | 15,000-36,000 patients | Derived from registry-epidemiology gap |
Sources: National Amyloidosis Centre annual report; NHS England ATTR-CM commissioning documentation; NICE TA984 decision document (updating TA696).
What this model answers
Every section answers a named commercial question your team is asking, scoped to your asset.
- Why does epidemiology-based sizing put true UK ATTRwt-CM prevalence at 20,000-40,000 while only 3,000-4,000 patients are on NICE-commissioned treatment? • Epidemiology-based prevalence methodology • NICE TA984 registry-based treated-population count • the 15,000-36,000 undiagnosed gap this implies
- What NHS diagnostic capacity constraint explains the registry-versus-epidemiology gap, and how fast can it close? • NHS Tc-PYP nuclear cardiology capacity (around 50 centres) • National Amyloidosis Centre referral-pathway mechanics • growth-rate modelling for the diagnosed population
- How robust is the sized market to its key assumptions, and which input moves the total most? • A sensitivity ranking of every sizing input — placing Tc-PYP diagnostic referral-pathway capacity above the underlying prevalence estimate as the assumption most likely to move the three-year total • scenario ranges around the central 15,000-36,000 undiagnosed estimate
What's inside
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1 · The Binding Constraint 2 pp
- Why NHS diagnostic referral-pathway capacity, not the underlying prevalence estimate, is the assumption most likely to move the total
- Pressure-tested against the registry-versus-epidemiology gap before the rest of the model is built out
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2 · Epidemiology-Based Sizing 3 pp
- UK ATTRwt-CM prevalence methodology anchored in National Amyloidosis Centre data
- The 20,000-40,000 estimate this implies
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3 · Registry-Based Sizing 3 pp
- NICE TA984-commissioned tafamidis treated population (3,000-4,000, +1,500-2,000/yr)
- Cross-check against the epidemiology-based estimate
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4 · Triangulation & Confidence Range 3 pp
- Where the two methods agree and diverge
- The 15,000-36,000 undiagnosed gap and its diagnostic-capacity explanation
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5 · Sensitivity Analysis 3 pp
- Diagnostic referral-pathway capacity ranked above prevalence estimate as the binding assumption
- Scenario ranges tied to NHS Tc-PYP centre-capacity expansion
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6 · Editable Excel Model —
- The full triangulated model, re-runnable with your own assumptions
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7 · Client Alignment Questions 2 pp
- The open sizing questions your team must close before the number is used in NHS budget planning
Appendix and source ledger included · 45-minute analyst readout included with delivery
Custom model delivered in 72 hours.
Assess this reportIncluded with every brief
Get Started
Commission this model
AXLRx delivers rare disease market sizing models built for forecasting and strategy teams sizing the UK ATTR amyloidosis opportunity. Custom model in 72 hours.
- 1. Submit your requestSpecify your indication, target market, and cohort definition.
- 2. Scoping callAXLRx analyst confirms triangulation methods and comparator set before building.
- 3. DeliveryResearch-verified sizing model in 72 hours with optional analyst readout.
How AXLRx builds this model
Prepared by MoatRx analysts on the AXLRx commercial consulting engine.
Every AXLRx market sizing model triangulates at least two independent methods, epidemiology-based and registry-based, before accepting a patient count. This is a sizing model, a static patient count, distinct from a Patient Flow or forecasting model that models dynamic revenue and uptake.
UK ATTR sizing sources: the National Amyloidosis Centre annual report, NHS England's ATTR-CM commissioning documentation, and the NICE TA984 decision document updating TA696.
- Epidemiology-based UK ATTRwt-CM prevalence estimate verified against National Amyloidosis Centre prevalence modelling
Frequently asked questions
Deliverables What formats are included with every model?
Every commissioned Market Sizing Model includes an editable 5-sheet Excel model (Cover, Model, Research Validation, QC, Sensitivity) and a PDF methodology brief. There is no PowerPoint deck: a sizing model is built to be worked in directly, not presented from. An optional 45-minute analyst readout call is included.
Sources How is the patient count verified?
AXLRx triangulates every sizing estimate across at least two independent methods, epidemiology-based and registry-based. No single-source number ships unverified.
Customisation Can I size a specific market or subpopulation?
Yes. The intake form captures your indication, target market, and cohort definition. A scoping call confirms scope before research starts. Commission through the intake form to begin.