Disease burden, diagnosis rates and the treatment pathway. The market map you build commercial strategy on.
US NSCLC segments by histology and biomarker before it segments by drug. This maps where patients are actually diagnosed and tested, and where the gap between diagnosis and treatment costs them.
PNH diagnosis pathway, FLAER flow-cytometry bottleneck and the treated-prevalent pool across US centres.
Tc-PYP scintigraphy, not biopsy, is now the diagnostic gate in ATTR amyloidosis. This maps the ATTR-CM versus ATTR-PN split and the wild-type/hereditary divide across a largely undiagnosed population.
US IgA nephropathy has shifted from RAS-blockade supportive care to five disease-modifying therapies. This maps the epidemiology and the biopsy-and-proteinuria diagnostic gate that still controls who reaches them.
HAE pathophysiology, the Type I/II split, attack burden and the diagnostic-delay problem that defines the US in-market landscape.
Neuromuscular-junction autoantibody biology, the AChR/MuSK/seronegative split, and the crisis burden that defines US gMG.
SCD epidemiology, genotype mix, VOC and organ-damage burden, and the 22-year life-expectancy gap across the US in-market population.
Gaucher type 1 epidemiology, the Ashkenazi Jewish founder burden, GBA1 genotype–phenotype, and the 20-fold Parkinson's risk.
US incidence 1 in 15,700, de novo SCN1A genetics, and one of the highest SUDEP rates documented in epilepsy.
Fabry disease splits into classic childhood-onset and a largely undiagnosed later-onset cardiac form. An X-linked organ timeline and the ~35–50% amenable-mutation gate decide who is treatable.
SMN1/SMN2 biology, the Type 1–4 severity spectrum, and how newborn screening splits SMA into pre-symptomatic and symptomatic populations.
Cold agglutinin disease is a rare classical-complement autoimmune haemolytic anaemia driven by cold-reactive IgM. This separates primary CAD from cold agglutinin syndrome and sizes the US haemolysis burden.
GAA-deficiency biology, the LOPD-versus-IOPD split, the years-long diagnostic delay, and newborn screening across the US Pompe population.
The MASLD-to-MASH-to-F2-F3 funnel, NASH-CRN fibrosis staging, and the FIB-4-to-elastography diagnostic gap behind the 6.7M label-eligible pool.
US atopic dermatitis epidemiology — 16.5M adults, the moderate-to-severe pool, and the atopic march that frames the biologic-eligible segment.
38.4M US adults, an 8.7M undiagnosed pool, and complication burden driving GLP-1 and SGLT2 organ-protection strategy.
US Alzheimer's staging, the amyloid-confirmation diagnostic pathway, and the FDA-cleared plasma pTau-217 blood test resizing the addressable pool.
US adult obesity at 41.9% (CDC NHANES). BMI-class distribution, severe-obesity burden, and the comorbidity segments that drive coverage.
COPD is a large, under-diagnosed, exacerbation-driven US disease that has just become biomarker-stratified. The blood eosinophil count now decides which of ~14 million diagnosed adults can reach a biologic.
Psoriasis affects about 3.0% of US adults, roughly 7.55 million people. Only the moderate-to-severe minority reaches the systemic and biologic therapies that define the commercial market.
HR+/HER2- is the largest breast cancer subtype, roughly 68% of the estimated 317,000 new US invasive female cases in 2025. Most present early and are curable, but 20-30% recur to metastatic disease where five-year survival falls to about a third, making biomarker testing (ESR1, PIK3CA) the fork that determines the treatment path.
The UK ran Europe's first national SMA newborn-screening programme. All three therapies are NICE-recommended with commercial arrangements, across a living UK cohort of ~1,000 patients in four disease types.
UK HAE Alliance genetic testing, an 87% attack-rate reduction on lanadelumab, and the NICE TA606 prophylaxis standard defining NHS management.
Leeds National Registry data, FLAER access without referral, and the 30% PNH-aplasia overlap defining the NHS commercial picture.
A ~25% inadequate-ERT-responder subset defines the next-generation enzyme-replacement opportunity in UK Pompe. The UK Pompe Consortium's shared-care network sits against a 3–8 year late-onset diagnostic delay.
SCN1A molecular confirmation gap, the cannabidiol regulatory restriction, and the stiripentol-backbone standard of care across GCC paediatric neurology.
A ~4,000-patient UK gMG treatment gap remains unresolved. Efgartigimod's June 2025 NICE rejection (TA1069) left it open, against NHS neuromuscular network diagnostics.
Vutrisiran's TA868 PAS-backed approval is reshaping UK ATTR identification and treatment. It joins NICE-commissioned tafamidis access (TA696, updated by TA984) and a 30-centre Tc-PYP diagnostic pathway.
The ERT infusion access gap across GCC metabolic centres defines the Pompe opportunity. Newborn screening is expanding for infantile-onset disease, while late-onset still takes a limb-girdle diagnostic detour.
GCC SMA incidence runs 1:6,000–8,000 births, with newborn screening now covering up to 90% in leading states. An 800–1,200-patient pre-NBS-era Type 2/3 cohort defines the chronic-therapy opportunity.
The thyroid-disease diagnostic confounder, specialist neurologist concentration, and the FcRn antagonist access pathway across GCC neurology practice.
UK Renal Registry data, the ACEi/ARB-first Renal Association pathway, and the NHS economic case for novel agents built on £40–50M annual ESRD cost.
HAE family cascade screening opportunity, laryngeal attack burden, and the prophylactic therapy access gap across GCC specialist centres.
The UK has Europe's largest SCD population at 15,000–17,000 patients. Newborn screening has made diagnosis near-universal since 1999, and NICE's recommendation of Casgevy (TA1044) will define UK access to a functional cure.
Premarital screening impact, the adult transition care gap, and the gene therapy access horizon across one of the world's highest per-capita SCD burdens.
Arabian Peninsula founder mutations, the female diagnosis gap, and the HEK assay bottleneck limiting oral chaperone therapy access across the GCC.
IgAN diabetes-masking effect, the kidney biopsy bottleneck, and the ESRD progression gap across GCC nephrology practice.
The NHS runs the most treatment-advanced Dravet pathway in Europe. Free SCN1A testing on GMS, a NICE-defined CBD-then-fenfluramine algorithm, and 25 paediatric epilepsy HSS centres underpin it.
An estimated 800-patient UK Fabry cohort has both NHS-commissioned ERT and NICE HST4-recommended oral chaperone therapy. The NHS lysosomal-storage-disorder specialist network and UK Fabry Outcome Survey longitudinal data track it.
PNH diagnostic pathway, FLAER flow-cytometry bottleneck and the undiagnosed clonal pool across GCC specialist centres.
Germany has no confirmed PNH prevalence data of its own. The DGHO's Onkopedia guideline borrows an estimate from British and French registries, and diagnosis funnels through just two national referral centres.
France's national hospitalisation database puts PNH prevalence near 1 in 94,000, or 897 patients from 2018 to 2022. That is lower than the 16-per-million figure Germany's DGHO Onkopedia guideline borrows from French and UK registries.
ATTR-CM diagnostic abyss, Arabian Peninsula TTR variant registry, and the referral-pathway delay across GCC cardiology centres.
A PDF analyst assessment, an editable Excel sizing model, and a PowerPoint readout, with a 45-minute analyst call included.
Every epidemiological figure is cited to its primary source at the point of writing and cross-checked against that source.
Yes. You set the indication, market and the segmentation that matters to your team; scope is confirmed on a call before research begins.