AXLRx Book a callCommission

Commercial stage 01

Market Intelligence

What does the competitive and scientific landscape look like?

Market Intelligence report types

43 published Competitive Intelligence Who controls which cohort, and on what evidence. Drug-by-drug commercial posture, prescribing share and payer positioning. CI · CI Team · Launch Lead 43 published Disease Landscape Disease burden, diagnosis rates and the treatment pathway. The market map you build commercial strategy on. DL · CI Team · Medical Affairs 23 published KOL Mapping A six-sheet KOL universe and engagement workbook, built gate by gate: sizing, tier classification, scientific influence, engagement readiness, MSL territory coverage, and cross-border network. Every KOL entry carries a verification tag. KOL · Medical Affairs · Commercial Lead

Every Market Intelligence analysis

CI · Neurology · CI Team · Launch Lead US Alzheimer's Disease Competitive Intelligence Lecanemab versus donanemab in early AD. CMS amyloid-confirmation coverage and ARIA monitoring as the access gate. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Immunology · CI Team · Launch Lead US Atopic Dermatitis Competitive Intelligence Dupilumab's 70% biologic share against JAK step-edits. A two-tier US payer landscape for IL-4Ra biologics and oral JAKs. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Metabolic · CI Team · Launch Lead US MASH Competitive Intelligence Rezdiffra and Wegovy now split the noncirrhotic MASH label. This is a Year 0 to Year 1 access and retention fight, not a pre-launch window. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Oncology · CI Team · Launch Lead US NSCLC Competitive Intelligence Four approved IO agents split 1L NSCLC into three PD-L1 cohorts. Pembrolizumab holds an estimated 52% share ahead of 2028 patent expiry. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Oncology · CI Team · Medical Affairs US NSCLC Disease Landscape 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. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Metabolic · CI Team · Launch Lead US Obesity Competitive Intelligence Tirzepatide 20.9% versus semaglutide 15.3% weight loss. The Medicare coverage gap and commercial step-edit reality. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead US PNH Competitive Intelligence Iptacopan oral pivot versus the anti-C5 IV class. Orphan-drug exclusion from IRA negotiation (US), NICE HST (UK) and SFDA lag (GCC). US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs US PNH Disease Landscape PNH diagnosis pathway, FLAER flow-cytometry bottleneck and the treated-prevalent pool across US centres. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Metabolic · CI Team · Launch Lead US Type 2 Diabetes Competitive Intelligence Tirzepatide takes 41% of new GLP-1 starts; SELECT CV indication and IRA negotiation reshape US formulary access. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US IgA Nephropathy Competitive Intelligence From zero disease-specific drugs to five in three years: how endothelin, complement and APRIL inhibitors are redrawing the IgAN market. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US Hereditary Angioedema Competitive Intelligence The prophylaxis class is fracturing along route: oral berotralstat and the first oral on-demand agent against a still-injectable antibody field. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US ATTR Amyloidosis Competitive Intelligence Tafamidis's ATTR-CM monopoly meets acoramidis — while the orphan-drug exclusion keeps the stabilizer class out of IRA price negotiation. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US ATTR Amyloidosis Disease Landscape 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 · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US Hereditary Angioedema Disease Landscape HAE pathophysiology, the Type I/II split, attack burden and the diagnostic-delay problem that defines the US in-market landscape. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US IgA Nephropathy Disease Landscape 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. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead · Market Access US Myasthenia Gravis Disease Landscape Neuromuscular-junction autoantibody biology, the AChR/MuSK/seronegative split, and the crisis burden that defines US gMG. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US Myasthenia Gravis Competitive Intelligence Three novel mechanisms in 24 months — FcRn antagonists vs C5 inhibitors, and the AChR+ vs MuSK+ line that segments the market. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US Dravet Syndrome Competitive Intelligence Fenfluramine leads on efficacy, cannabidiol anchors the lower-cost branded option, and stiripentol holds the adjunct niche. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US Gaucher Disease Disease Landscape Gaucher type 1 epidemiology, the Ashkenazi Jewish founder burden, GBA1 genotype–phenotype, and the 20-fold Parkinson's risk. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US Cold Agglutinin Disease Disease Landscape 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. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US Gaucher Disease Competitive Intelligence Five FDA-approved Gaucher type 1 therapies (three IV enzyme replacement vs two oral substrate reduction) and eliglustat's oral first-line pivot. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US Pompe Disease Competitive Intelligence Two next-generation ERTs are moving to displace alglucosidase alfa in US late-onset Pompe disease. Avalglucosidase alfa and cipaglucosidase alfa plus miglustat now define the competitive set. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US Spinal Muscular Atrophy Disease Landscape SMN1/SMN2 biology, the Type 1–4 severity spectrum, and how newborn screening splits SMA into pre-symptomatic and symptomatic populations. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US Sickle Cell Disease Competitive Intelligence Two Dec-2023 gene therapies (Casgevy, Lyfgenia) reset a ~100,000-patient market, while voxelotor's 2024 withdrawal thins the oral field. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US Fabry Disease Disease Landscape 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. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US Pompe Disease Disease Landscape GAA-deficiency biology, the LOPD-versus-IOPD split, the years-long diagnostic delay, and newborn screening across the US Pompe population. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US Dravet Syndrome Disease Landscape US incidence 1 in 15,700, de novo SCN1A genetics, and one of the highest SUDEP rates documented in epilepsy. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead US Sickle Cell Disease Disease Landscape SCD epidemiology, genotype mix, VOC and organ-damage burden, and the 22-year life-expectancy gap across the US in-market population. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US Cold Agglutinin Disease Competitive Intelligence Sutimlimab (Enjaymo) is the only FDA-approved CAD therapy — competing against off-label rituximab-based standard of care, not a branded rival. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US Fabry Disease Competitive Intelligence Pegunigalsidase now challenges Fabrazyme's two-decade lead in US Fabry disease. Two IV enzyme replacement therapies meet an oral chaperone only ~35–50% of patients can take. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access US Spinal Muscular Atrophy Competitive Intelligence Three mechanisms chase one SMN target: a one-time $2.125M gene therapy, chronic intrathecal ASO, and daily oral. Newborn screening resets the battlefield. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Metabolic · CI Team · Launch Lead US MASH Disease Landscape 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 · In-Market · 24–32 pp · PDF · Excel · PPT DL · Immunology · CI Team · Launch Lead US Atopic Dermatitis Disease Landscape US atopic dermatitis epidemiology — 16.5M adults, the moderate-to-severe pool, and the atopic march that frames the biologic-eligible segment. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Metabolic · CI Team · Launch Lead US Type 2 Diabetes Disease Landscape 38.4M US adults, an 8.7M undiagnosed pool, and complication burden driving GLP-1 and SGLT2 organ-protection strategy. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Neurology · CI Team · Launch Lead US Alzheimer's Disease Disease Landscape US Alzheimer's staging, the amyloid-confirmation diagnostic pathway, and the FDA-cleared plasma pTau-217 blood test resizing the addressable pool. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Metabolic · CI Team · Launch Lead US Obesity Disease Landscape US adult obesity at 41.9% (CDC NHANES). BMI-class distribution, severe-obesity burden, and the comorbidity segments that drive coverage. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Pulmonology · CI Team · Launch Lead US COPD Competitive Intelligence Two 2024 approvals moved the COPD maintenance fight past the inhaler for the first time in a decade. Ensifentrine and dupilumab split the market into an inhaler base and a biology-defined add-on tier. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Pulmonology · CI Team · Launch Lead US COPD Disease Landscape 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. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Immunology · CI Team · Launch Lead US Plaque Psoriasis Competitive Intelligence Six mechanisms now compete for moderate-to-severe plaque psoriasis in the US, and the efficacy bar has moved from PASI 75 to PASI 90. IL-17A, dual IL-17A/F, IL-23p19, IL-12/23, TNF and oral TYK2 all hold ground. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Immunology · CI Team · Launch Lead US Plaque Psoriasis Disease Landscape 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. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Oncology · CI Team · Launch Lead US Breast Cancer HR+/HER2- Competitive Intelligence Only ribociclib has posted consistent overall-survival wins in the three-way first-line CDK4/6 contest. MONALEESA-2 showed 63.9 versus 51.4 months (HR 0.76). The real competition has moved downstream, where 2023 approvals of an oral SERD and an AKT inhibitor carve the post-CDK4/6 line by biomarker. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Oncology · CI Team · Launch Lead US Breast Cancer HR+/HER2- Disease Landscape 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. US · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead · Market Access UK ATTR Amyloidosis Competitive Intelligence Tafamidis's ATTR-CM franchise meets acoramidis under NICE — which now tells clinicians to pick the least-expensive stabiliser. UK · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC PNH Competitive Intelligence Anti-C5 IV therapy holds the GCC PNH market under NPHC and MOH specialist-centre gating. Iptacopan's 82.3% haemoglobin-response rate has not reached a single GCC formulary. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC IgA Nephropathy Competitive Intelligence Novel IgAN agents are registered across the GCC but not yet formulary-listed. Nephrology specialist centres and the private hospital market are the fastest access channel, while biopsy capacity limits diagnosis. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC Hereditary Angioedema Competitive Intelligence GCC HAE management is acute-only, with prophylaxis penetration near zero. More than 85% of patients are undiagnosed, and NPHC coverage for lanadelumab would be the access trigger for the region's largest market. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC ATTR Amyloidosis Competitive Intelligence ATTR-CM in the GCC is a pre-commercial opportunity gated by diagnosis, not by drug access. Tafamidis is SFDA-registered and tender-priced 80-90% below US list, but Tc-PYP scintigraphy runs at fewer than 8 centres. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC Dravet Syndrome Competitive Intelligence GCC Dravet prescribing runs opposite the US and EU hierarchy. Cannabidiol is de-facto inaccessible under narcotics law, so stiripentol is the specialist standard of care. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead UK Dravet Syndrome Competitive Intelligence Cannabidiol and fenfluramine anchor the NHS-commissioned NICE algorithm; stiripentol still holds a backbone role. New entrants must beat an entrenched three-drug sequence, not just show efficacy. UK · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC Myasthenia Gravis Competitive Intelligence NPHC formulary criteria for the FcRn class don't yet exist, so pyridostigmine and steroids still treat 85%+ of GCC patients. Efgartigimod reached GCC neurology centres via SFDA and MOH UAE registration in 2023. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead UK IgA Nephropathy Competitive Intelligence IgAN doesn't qualify for the ultra-rare HST track, so both novel agents must clear the standard £20,000-30,000/QALY bar. Both sit in Named Patient access ahead of their NICE decisions. UK · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead UK Myasthenia Gravis Competitive Intelligence No novel biologic is yet NHS-commissioned for generalised MG. Eculizumab's manufacturer withdrew its 2020 NICE appraisal before a verdict, and NICE rejected efgartigimod outright in 2025 — leaving rozanolixizumab as the FcRn class's last untested NICE bid. UK · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC Sickle Cell Disease Competitive Intelligence GCC carries one of the highest per-capita SCD burdens globally: ~140,000 patients in Saudi Arabia alone. Both novel disease-modifiers hit regulatory trouble in 2023-2024, leaving a 25-year-old generic as the only agent with a stable market position. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC Fabry Disease Competitive Intelligence GCC is a two-ERT Fabry market, with agalsidase alfa via the EMA pathway alongside agalsidase beta. Migalastat's oral advantage, covering 35-50% of patients, is bottlenecked by the single GCC lab that can run the amenable-mutation assay. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC Spinal Muscular Atrophy Competitive Intelligence All three SMA mechanisms are formulary-listed across the GCC. Outcomes-based rebate contracts now anchor Zolgensma's ~$1.5-1.8M price to a 24-month motor milestone, after an NBS expansion generating 60-80 new gene-therapy candidates a year. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Market Access GCC Pompe Disease Competitive Intelligence Nexviazyme beat Lumizyme on 6-minute-walk distance in COMET, but NPHC has set no switch criteria. So 80-120 GCC ERT patients mostly stay on the 2006-era standard. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead UK Fabry Disease Competitive Intelligence Pegunigalsidase's suboptimal-responder recommendation (TA915) reshapes NHS-commissioned Fabry therapy. It sets oral migalastat against IV enzyme replacement across the UK treated population. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead UK Spinal Muscular Atrophy Disease Landscape 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 · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs UK Hereditary Angioedema Disease Landscape UK HAE Alliance genetic testing, an 87% attack-rate reduction on lanadelumab, and the NICE TA606 prophylaxis standard defining NHS management. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs UK PNH Disease Landscape Leeds National Registry data, FLAER access without referral, and the 30% PNH-aplasia overlap defining the NHS commercial picture. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead UK Pompe Disease Disease Landscape 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team UK Hereditary Angioedema Competitive Intelligence England's NICE has issued three positive technology appraisals funding HAE prophylaxis since 2019 (TA606, TA738, TA1101). The two newest MHRA-licensed agents, donidalorsen and sebetralstat, remain in NICE appraisal with no confirmed final NHS funding decision. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC Dravet Syndrome Disease Landscape SCN1A molecular confirmation gap, the cannabidiol regulatory restriction, and the stiripentol-backbone standard of care across GCC paediatric neurology. GCC · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs UK Myasthenia Gravis Disease Landscape 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC Hereditary Angioedema KOL Mapping KFSH&RC, AUH, and Hamad Medical Corporation anchor a GCC HAE specialist community that SACIA sizes at just 6-10 physicians regionwide. That concentration is exactly what this workbook sizes before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK ATTR Amyloidosis KOL Mapping The National Amyloidosis Centre holds the clinical-expert seat at every NICE ATTR appraisal to date. This workbook sizes the UK ATTR KOL network before a name enters it: NAC's 400-500-patient ATTRv surveillance registry, the separate UCL and Queen Elizabeth Hospital Birmingham National ATTRv Registry tracking approximately 800 patients, and the roughly 30 NHS centres running Tc-PYP scintigraphy nationally. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC Dravet Syndrome KOL Mapping A five-institution Saudi consortium across Riyadh, Jeddah and Dammam tracked 44 Dravet patients on stiripentol combination therapy. That kind of coordinated, cross-city publication is exactly the institutional signal this workbook sizes before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK Sickle Cell Disease KOL Mapping Four London teaching hospitals anchor roughly 6,000 of the UK's 15,000-17,000 sickle cell patients. Barts, King's, Imperial and Homerton, with Birmingham Heartlands, Manchester, Bristol and Nottingham forming the next ring. That eight-centre structure is exactly the institutional signal this workbook sizes before any individual name enters it. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK Spinal Muscular Atrophy KOL Mapping Four NHS Highly Specialised Services networks and six designated gene-therapy centres carry the UK SMA treatment pathway. This workbook sizes that institutional structure, plus the Bristol Institute of Child Health attenuation-cohort follow-up, before any physician name enters it. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · Disease Landscape FR PNH Disease Landscape 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. FR · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team FR PNH Competitive Intelligence HAS reimburses iptacopan second-line only, after at least six months on a C5 inhibitor, while ravulizumab holds first-line. France split the anti-complement class by line of therapy, not by price. FR · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs UK ATTR Amyloidosis Disease Landscape Vutrisiran's TA868 PAS-backed approval is reshaping UK ATTR identification and treatment. It joins NICE-recommended tafamidis (TA984, June 2024, after the 2021 TA696 rejection) and a 30-centre Tc-PYP diagnostic pathway. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC Pompe Disease Disease Landscape 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 · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC Spinal Muscular Atrophy Disease Landscape 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs UK IgA Nephropathy Disease Landscape 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead UK Spinal Muscular Atrophy Competitive Intelligence All three SMA therapies cleared NICE with confidential PAS. The UK's 2021 newborn screening programme is now the real access lever, shifting competition to physician and family preference. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC Hereditary Angioedema Disease Landscape HAE family cascade screening opportunity, laryngeal attack burden, and the prophylactic therapy access gap across GCC specialist centres. GCC · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead UK Sickle Cell Disease Disease Landscape 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC Sickle Cell Disease Disease Landscape 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. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC Fabry Disease KOL Mapping KFSH&RC runs the only HEK293 amenable-mutation assay in the GCC. Its formulary committee recommendation drives NPHC coverage decisions for every Fabry disease treatment. That kind of single-institution gatekeeping is exactly the concentration this workbook sizes before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC Myasthenia Gravis KOL Mapping Just 10-15 neuromuscular neurologists across six named GCC referral centres manage 70-80% of confirmed generalised myasthenia gravis. That concentration is exactly what this workbook sizes before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead UK PNH Competitive Intelligence All three UK PNH agents cleared NICE via the standard Technology Appraisal route, not the ultra-rare one. Ravulizumab (TA698), iptacopan (TA1000) and crovalimab all took it. Convenience and switch dynamics, not pathway-driven affordability, now determine NHS share. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC Fabry Disease Disease Landscape Arabian Peninsula founder mutations, the female diagnosis gap, and the HEK assay bottleneck limiting oral chaperone therapy access across the GCC. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC Pompe Disease KOL Mapping Six to eight named metabolic centres across the GCC hold ERT infusion capacity for Pompe disease. KFSH&RC leads with roughly 120 patients over 15 years. That kind of institutional concentration is exactly what this workbook sizes before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK PNH KOL Mapping Fewer than 25 consultants manage over 90% of the UK's PNH caseload. They sit across five NHS Highly Specialised Services centres anchored by the Leeds National PNH Service. That kind of institutional concentration is exactly the signal this workbook sizes before any individual name enters it. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Oncology · Medical Affairs · Medical Affairs UK Breast Cancer HR+/HER2- KOL Mapping A single five-centre South West England consortium tracks 666 UK patients across all three approved CDK4/6 inhibitors. That kind of coordinated, multi-centre evidence generation is exactly the institutional signal this workbook sizes before any individual name enters it. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK Dravet Syndrome KOL Mapping Six NHS centres concentrate the UK's refractory Dravet syndrome cohort. Great Ormond Street, Bristol, Alder Hey, Birmingham Children's, Leeds and Newcastle. The British Paediatric Neurology Association's Dravet working group shapes NICE evidence review 18-24 months ahead of submission, and that institutional network is what this workbook sizes first, not a roster of named physicians. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead UK Dravet Syndrome Disease Landscape 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Launch Lead UK Fabry Disease Disease Landscape 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. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC PNH Disease Landscape PNH diagnostic pathway, FLAER flow-cytometry bottleneck and the undiagnosed clonal pool across GCC specialist centres. GCC · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · Disease Landscape DE PNH Disease Landscape 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. DE · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead UK Sickle Cell Disease Competitive Intelligence Crizanlizumab's EMA/MHRA withdrawal leaves a VOC-prevention gap. Casgevy's NICE recommendation is the watershed NHS gene-therapy access event — Lyfgenia has no UK regulatory status. UK · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC IgA Nephropathy Disease Landscape IgAN diabetes-masking effect, the kidney biopsy bottleneck, and the ESRD progression gap across GCC nephrology practice. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team DE PNH Competitive Intelligence Iptacopan's orphan-drug status let it clear AMNOG with an established additional benefit and a substantial quality-of-life finding. Ravulizumab, tested on Germany's only PNH-specific G-BA review to date, found no added benefit at all. DE · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK Pompe Disease KOL Mapping A six-physician BIMDG subcommittee advises NICE directly on Pompe disease. It sits at the centre of a five-centre UK Pompe Consortium and an eight-centre NHS Highly Specialised Service network. That kind of institutional concentration is exactly what this workbook sizes before any individual name enters it. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC Spinal Muscular Atrophy KOL Mapping Four designated GCC centres carry nearly all regional SMA gene-therapy and specialist referral activity. KAMC and KFSH&RC in Riyadh, Sidra Medicine in Doha, and SKMC in Abu Dhabi. This workbook sizes that institutional concentration before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK IgA Nephropathy KOL Mapping The Renal Association's IgAN guideline committee, not any single physician, sets the reference point NICE checks against. This workbook sizes that committee and the network behind it before individual outreach begins. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC Sickle Cell Disease KOL Mapping Only three GCC institutions run structured adult sickle cell programmes for 200,000-250,000 patients. KFSH&RC, KAMC and AUH. That three-centre concentration, anchored by the KFSH&RC Dammam flagship and 12+ MOH regional centres in Eastern Province, is exactly the institutional signal this workbook sizes before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs FR PNH KOL Mapping Saint-Louis Hospital anchors France's MaRIH-coordinated PNH reference network of 14 competence centres. Only 24 of the 50-plus centres in the national clone-observatory network actively report into it. That kind of institutional structure is exactly what this workbook sizes before any individual name enters it. FR · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Oncology · Medical Affairs · Medical Affairs US Breast Cancer HR+/HER2- KOL Mapping A 9,146-patient US real-world study found no significant survival difference between palbociclib, ribociclib, and abemaciclib. When the drugs perform equivalently, KOL guidance decides which one gets prescribed, not clinical data. US · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC Myasthenia Gravis Disease Landscape The thyroid-disease diagnostic confounder, specialist neurologist concentration, and the FcRn antagonist access pathway across GCC neurology practice. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK Hereditary Angioedema KOL Mapping Five NHS specialist centres manage more than 90% of UK HAE patients. Sheffield, Cambridge, Birmingham, Guy's and St Thomas', and Manchester. That concentration is what this workbook sizes before any physician enters the K1-K6 gate structure. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK Fabry Disease KOL Mapping Royal Free London's National Fabry Service was NICE's appointed clinical expert twice over. It advised on both migalastat's HST4 and pegunigalsidase alfa's TA915, across a network of five named NHS lysosomal storage disorder centres. That repeated appointment is exactly the institutional signal this workbook sizes before any individual name enters it. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs UK Myasthenia Gravis KOL Mapping A 30-centre NHS neuromuscular network coordinated through Muscular Dystrophy UK carries UK myasthenia care. That network, plus a 3,000-member Myasthenia Gravis Association UK patient registry, is the institutional signal this workbook sizes before any individual name enters it. UK · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC ATTR Amyloidosis KOL Mapping KFSH&RC maintains the only GCC-wide ATTRv genetic registry and houses the region's nuclear cardiology programme. Fewer than 8 named cardiology and scintigraphy centres region-wide, spanning KAMC, AUH, HMC Doha, and OCCI Muscat, define a small institutional field this workbook sizes before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC IgA Nephropathy KOL Mapping GCC IgA nephropathy expertise concentrates in five named tertiary centres. Roughly 300 practising nephrologists work the region, of whom 30 to 50 carry glomerular-disease subspecialty interest. This workbook sizes that concentrated institutional base before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT DL · Rare Disease · CI Team · Medical Affairs GCC ATTR Amyloidosis Disease Landscape ATTR-CM diagnostic abyss, Arabian Peninsula TTR variant registry, and the referral-pathway delay across GCC cardiology centres. GCC · In-Market · 24–32 pp · PDF · Excel · PPT KOL · Rare Disease · Medical Affairs · Medical Affairs GCC PNH KOL Mapping Seven institutions across Riyadh, Jeddah, Dubai and Abu Dhabi hold the GCC's confirmed PNH caseload. Just 5 to 8 physicians function as genuine adoption gatekeepers. That kind of concentration is exactly what this workbook sizes before any individual name enters it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT CI · Rare Disease · CI Team · Launch Lead UK Pompe Disease Competitive Intelligence Avalglucosidase alfa's NICE recommendation (TA821) puts it against entrenched alglucosidase alfa. NHS switch criteria and the Pombiliti queue position define the next 18 months of UK access. UK · In-Market · 24–32 pp · PDF · Excel · PPT

Other stages

02 · Sizing & Access03 · Commercial Planning04 · Analytics & Operations