Disease burden, diagnosis rates and the treatment pathway. The market map you build commercial strategy on.
How the US NSCLC population segments by histology and biomarker, where it is actually diagnosed and tested, and where the gap between diagnosis and treatment costs patients.
PNH diagnosis pathway, FLAER flow-cytometry bottleneck and the treated-prevalent pool across US centres.
The ATTR-CM vs ATTR-PN split, the wild-type/hereditary divide, and why Tc-PYP scintigraphy, not biopsy, is now the diagnostic gate for a largely undiagnosed population.
HAE pathophysiology, the Type I/II split, attack burden and the diagnostic-delay problem that defines the US in-market landscape.
US IgA nephropathy epidemiology, the biopsy-and-proteinuria diagnostic gate, and the shift from RAS-blockade supportive care to five disease-modifying therapies.
Neuromuscular-junction autoantibody biology, the AChR/MuSK/seronegative split, and the crisis burden that defines US gMG.
SMN1/SMN2 biology, the Type 1–4 severity spectrum, and how newborn screening splits SMA into pre-symptomatic and symptomatic populations.
US incidence 1 in 15,700, de novo SCN1A genetics, and one of the highest SUDEP rates documented in epilepsy.
GAA-deficiency biology, the LOPD-versus-IOPD split, the years-long diagnostic delay, and newborn screening across the US Pompe population.
SCD epidemiology, genotype mix, VOC and organ-damage burden, and the 22-year life-expectancy gap across the US in-market population.
A rare classical-complement autoimmune hemolytic anemia driven by cold-reactive IgM — primary CAD vs cold agglutinin syndrome, and the US hemolysis burden.
Gaucher type 1 epidemiology, the Ashkenazi Jewish founder burden, GBA1 genotype–phenotype, and the 20-fold Parkinson's risk.
Classic childhood-onset Fabry versus a largely undiagnosed later-onset cardiac form, an X-linked organ timeline, and the ~35–50% amenable-mutation treatment gate.
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, but 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 Pompe Consortium's shared-care network, a 3–8 year late-onset diagnostic delay, and the ~25% inadequate-ERT-responder subset defining the next-generation enzyme-replacement opportunity.
A ~4,000-patient UK gMG treatment gap, NHS neuromuscular network diagnostics, and efgartigimod's June 2025 NICE rejection (TA1069) that leaves the access gap unresolved.
NICE-commissioned tafamidis access (TA696, updated by TA984), a 30-centre Tc-PYP diagnostic pathway, and vutrisiran's TA868 PAS-backed approval reshaping UK ATTR identification and treatment.
SCN1A molecular confirmation gap, the cannabidiol regulatory restriction, and the stiripentol-backbone standard of care across GCC paediatric neurology.
The newborn screening expansion for infantile-onset Pompe, the late-onset limb-girdle diagnostic detour, and the ERT infusion access gap across GCC metabolic centres.
GCC SMA incidence running 1:6,000–8,000 births, newborn screening now covering up to 90% in leading states, and an 800–1,200-patient pre-NBS-era Type 2/3 cohort defining 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.
UK HAE Alliance genetic testing, an 87% attack-rate reduction on lanadelumab, and the NICE TA606 prophylaxis standard defining NHS management.
Europe's largest SCD population at 15,000–17,000 patients, near-universal newborn-screening diagnosis since 1999, and NICE's recommendation of Casgevy (TA1044) as the gene therapy that 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.
NHS GMS free SCN1A testing, a NICE-defined CBD-then-fenfluramine algorithm, and 25 paediatric epilepsy HSS centres running the most treatment-advanced Dravet pathway in Europe.
The NHS lysosomal-storage-disorder specialist network, UK Fabry Outcome Survey longitudinal data, and both NHS-commissioned ERT and NICE HST4-recommended oral chaperone therapy across an estimated 800-patient UK cohort.
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 own national hospitalization database puts PNH prevalence near 1 in 94,000, 897 patients from 2018 to 2022, lower than the 16-per-million figure Germany's DGHO Onkopedia guideline borrows from French and UK registries.
Leeds National Registry data, FLAER access without referral, and the 30% PNH-aplasia overlap defining the NHS commercial picture.
Europe's first national SMA newborn-screening programme, all three therapies NICE-recommended with commercial arrangements, and a living UK cohort of ~1,000 patients across four disease types.
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.