A rate-based epidemiology range, not a single point estimate, is the right foundation for sizing a population this rare, and the model still can't say what share is treated.
Cold agglutinin disease (CAD) is rare enough that no single registry counts every US patient, so this model starts from a 2016-2023 multi-database claims analysis that estimated CAD incidence at 0.6-1.2 per 100,000 person-years and 1-year prevalence at 1.4-3.1 per 100,000 (PMID 40570006). Applied to the US adult population, that rate range implies an order-of-magnitude prevalent pool of roughly 5,000 patients, a figure cross-confirmed across AXLRx's disease landscape and payer analyses for this indication rather than sourced from a single publication.
At sutimlimab's reported annual cost of $259,000 to $302,000 per patient, a bottom-up total addressable market multiplies the roughly 5,000-patient prevalent pool by that price range, producing a TAM band rather than a single number, consistent with the underlying rate-based epidemiology. What this model cannot do is split that addressable total into a served-versus-untreated share: no published source reports a current treated-share or penetration figure for cold agglutinin disease, so the gap between total addressable patients and patients actually on therapy is flagged as an open research question rather than filled with an invented estimate.
US CAD sizing — rate-based epidemiology into a bottom-up TAM
| Sizing Method | Estimate | Source |
|---|---|---|
| Epidemiology-based (incidence) | 0.6-1.2 per 100,000 person-years | PMID 40570006 |
| Epidemiology-based (1-year prevalence) | 1.4-3.1 per 100,000 | PMID 40570006 |
| Cross-confirmed prevalent pool | ~5,000 US patients (order of magnitude) | AXLRx disease landscape & payer cross-check |
| Bottom-up TAM (patients x price) | ~5,000 patients x $259,000-$302,000/yr | Derived; price per Immunotherapy 2022 |
Sources: PLoS One 2025 (PMID 40570006), US multi-database claims analysis 2016-2023; Berentsen, Immunotherapy 2022 (sutimlimab annual cost); AXLRx cross-source prevalence confirmation across disease landscape and payer analyses.
What this model answers
Every section answers a named commercial question your team is asking, scoped to your asset.
Delivers
- The rate-based epidemiology methodology behind the incidence and 1-year prevalence range
- how the ~5,000-patient estimate is cross-confirmed across independent AXLRx analyses
Delivers
- The bottom-up TAM methodology
- the sensitivity range this produces when the epidemiology rate range and the price range are combined
Delivers
- The absence of any published treated-share figure for cold agglutinin disease
- why AXLRx flags rather than estimates it
- the primary research path that would close it
Custom model delivered in 72 hours.
Commission This ModelWhat's inside
- Why a rate range, not a point estimate, is the honest starting point for a population this rare
- The treated-share gap that no published source closes
- Incidence of 0.6-1.2 per 100,000 person-years and 1-year prevalence of 1.4-3.1 per 100,000, US claims 2016-2023
- How the rate range translates into a US prevalent-pool estimate
- The ~5,000-patient order-of-magnitude estimate cross-checked across independent AXLRx disease landscape and payer analyses
- Why a single-source count would understate confidence in the total
- ~5,000 patients multiplied against the $259,000-$302,000 annual cost of sutimlimab therapy
- Why the TAM is expressed as a band, not a single figure
- No published source reports a current treated-share or penetration figure for cold agglutinin disease
- Flagged as an open research question rather than an invented split
- TAM range across the full incidence/prevalence rate band and the price range
- Which input moves the total most
- The full triangulated model, re-runnable with your own assumptions
- The open sizing questions your team must close before the number is used in planning
Included with every brief
How AXLRx builds this model
Prepared by MoatRx analysts.
Every AXLRx market sizing model triangulates at least two independent methods before accepting a patient or revenue estimate. For an ultra-rare population like cold agglutinin disease, that means a rate-based epidemiology range cross-confirmed across multiple internal analyses, not a single claims count treated as exact.
Cold agglutinin disease US sizing sources: PLoS One 2025 (PMID 40570006) for incidence and 1-year prevalence, and Berentsen, Immunotherapy 2022, for the annual cost of sutimlimab therapy used as the price input to the bottom-up TAM.
- CAD incidence (0.6-1.2 per 100,000 person-years) and 1-year prevalence (1.4-3.1 per 100,000) verified against PLoS One 2025 (PMID 40570006)
- The ~5,000-patient order-of-magnitude prevalent pool cross-confirmed against AXLRx's independent disease landscape and payer analyses for this indication
- Sutimlimab annual cost of therapy ($259,000-$302,000) verified against Berentsen, Immunotherapy 2022
- Confirmed no published source reports a current treated-share or penetration figure for cold agglutinin disease before flagging the gap
Frequently asked questions
Commission this model
AXLRx delivers rare disease market sizing models built for forecasting and strategy teams sizing the US cold agglutinin disease opportunity. Custom model in 72 hours.
Specify your indication, market, and any subpopulation of interest.
AXLRx analyst confirms triangulation methods and comparator set before building.
Research-verified sizing model in 72 hours with optional analyst readout.