Rare Disease · United Kingdom · In-Market

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.

5-sheet modelEpidemiology vs NICE-registry triangulationIn-MarketUpdated Q3 2026
Market United States GCC (Gulf) United Kingdom Stage
The Landscape

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 updated the original TA696 recommendation, 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. NHS England projects total ATTR-CM spend at £80-120 million a year once roughly 5,000 patients are on treatment, which sets a natural planning ceiling for how fast the diagnosed population, and therefore the addressable market, can grow. 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.

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
£80-120M
projected annual NHS ATTR-CM spend at peak, roughly 5,000 patients on treatment
TRIANGULATION

UK ATTR sizing — epidemiology-based prevalence versus NICE-registry treated population

Sizing MethodPopulation EstimateSource
Epidemiology-based (true ATTRwt-CM prevalence)20,000-40,000 patientsNational 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 share15,000-36,000 patientsDerived from registry-epidemiology gap
NHS peak spend scenario£80-120M/year at ~5,000 patientsNHS England ATTR-CM service specification 2023

Sources: National Amyloidosis Centre annual report; NHS England ATTR-CM service specification 2023; NICE TA984 decision document (updating TA696).

Commercial Questions

What this model answers

Every section answers a named commercial question your team is asking, scoped to your asset.

01
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?

Delivers

  • Epidemiology-based prevalence methodology
  • NICE TA984 registry-based treated-population count
  • the 15,000-36,000 undiagnosed gap this implies
02
What NHS diagnostic capacity constraint explains the registry-versus-epidemiology gap, and how fast can it close?

Delivers

  • NHS Tc-PYP nuclear cardiology capacity (around 50 centres)
  • National Amyloidosis Centre referral-pathway mechanics
  • growth-rate modelling for the diagnosed population
03
What NHS budget ceiling should a sizing model plan against as the diagnosed population grows toward the undiagnosed pool?

Delivers

  • NHS England's £80-120 million peak ATTR-CM spend projection at ~5,000 patients
  • scenario ranges tied to diagnostic-capacity expansion
  • sensitivity ranking of every input

Custom model delivered in 72 hours.

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Contents

What's inside

Rare Disease · 24–32 pp · In-Market · Analyst report + Excel model + PowerPoint readout

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
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
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
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
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
6 Editable Excel Model
  • The full triangulated model, re-runnable with your own assumptions
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
Formats

Included with every brief

PDF
PDF Brief
Market Sizing Brief — Complete Edition
PDF methodology brief accompanying the 5-sheet sizing model: epidemiology-based and NICE-registry-based triangulation for ATTR amyloidosis in the UK.
XLS
Excel Model
Market Sizing Model — Excel
5-sheet editable model: Cover, Model, Research Validation, QC, Sensitivity.
Methodology

How AXLRx builds this model

Prepared by MoatRx analysts.

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 service specification 2023, and the NICE TA984 decision document updating TA696.

  • Epidemiology-based UK ATTRwt-CM prevalence estimate verified against National Amyloidosis Centre prevalence modelling
  • NICE TA984-commissioned treated-population count and annual growth rate verified against NHS England ATTR-CM service specification 2023
  • NHS peak spend projection verified against NHS England ATTR-CM service specification 2023
FAQ

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.
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 request

Specify your indication, target market, and cohort definition.

2
Scoping call

AXLRx analyst confirms triangulation methods and comparator set before building.

3
Delivery

Research-verified sizing model in 72 hours with optional analyst readout.