What formats are included?

A PDF access assessment, an Excel coverage and GTN grid, and a PowerPoint readout, with a 45-minute analyst call included.

How are figures verified?

Coverage and HTA claims are cited to the live policy or assessment document at the point of writing and re-checked before delivery.

Yes. You set the market, payer set and comparators; scope is confirmed on a call before research begins.

Payer & HTA Reports, All Indications

P&HTA · Oncology · Market Access · Medical Affairs US NSCLC Payer & HTA CMS routes all four approved 1L NSCLC IO agents through Part B buy-and-bill at ASP+6%. But PD-L1 assay requirements split coverage into three distinct biomarker-testing tiers. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US PNH Payer & HTA Why the orphan-drug exclusion shields anti-C5 agents from IRA negotiation, ICER's 2024 value verdict on iptacopan, and Part B vs Part D routing. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US IgA Nephropathy Payer & HTA US payers gate the five FDA-approved IgAN therapies on biopsy, proteinuria and RAS-blockade step-through. The Filspari REMS, Part D routing and the ICER 2026 assessment set the rest of the access path. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US ATTR Amyloidosis Payer & HTA Tafamidis is shielded from IRA negotiation by the orphan-drug exclusion. ICER judged its ~$268K price ~85–95% too high, and acoramidis plus pending generics are the real net-price levers. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US Hereditary Angioedema Payer & HTA Specialty-tier prior authorization, prophylaxis above $300K/patient/yr, ICER 2018/2021 value-based benchmarks and no-concurrent-acute-agent rules. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · CI Team · Launch Lead · Market Access US Myasthenia Gravis Payer & HTA ICER priced efgartigimod's value at $18,300–28,400 a year, under half its ~$418,400 launch cost. That gap is hardening into a three-tier FcRn-to-C5 step-edit across US commercial plans. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US Gaucher Disease Payer & HTA IV enzyme replacement buy-and-bill under Part B vs oral SRT under Part D, the CYP2D6 PA gate, and generic miglustat. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US Sickle Cell Disease Payer & HTA Gene-therapy access at $2.2–3.1M, the CMS Cell & Gene Therapy Access Model, VOC-freedom endpoints, and the hydroxyurea step-edit. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US Spinal Muscular Atrophy Payer & HTA Zolgensma's $2.125M one-time cost, outcomes-based Medicaid contracts, and Part B vs Part D routing across three SMA modalities. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US Cold Agglutinin Disease Payer & HTA Sutimlimab (Enjaymo) is a ~$260K+/year Part B IV biologic in a few-thousand-patient population — and not cost-effective at the current price. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US Dravet Syndrome Payer & HTA Fintepla's list price runs roughly 3x Epidiolex, and payer scrutiny turns on high WAC against a small, severe paediatric population. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US Pompe Disease Payer & HTA Pompe ERT costs near $400,000 a year in Part B, and remains the only major rare-disease ERT category ICER has never reviewed. Pombiliti + Opfolda splits into two simultaneous prior authorisations across Part B and Part D. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs US Fabry Disease Payer & HTA A genetic test gates oral migalastat, and IV enzyme replacement sits in Part B. Fabrazyme has no US biosimilar, and the IRA's orphan-drug exclusion shields it from Medicare price negotiation. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Immunology · Market Access · Medical Affairs US Atopic Dermatitis Payer & HTA Topical-then-biologic step-therapy, JAK black-box PA gates, ICER's dupilumab-aligned and JAK-discount verdicts, and why Dupixent is not IRA-selected. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Metabolic · Market Access · Medical Affairs US MASH Payer & HTA Why Rezdiffra's $47,400 WAC lands inside ICER's value range, why the IRA reset hits semaglutide first, and how Part D routing shapes MASH access. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Metabolic · Market Access · Medical Affairs US Type 2 Diabetes Payer & HTA Type 2 Diabetes is IRA ground zero: three orals negotiated for 2026, semaglutide at $274 for 2027, and the class price anchor reset. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Neurology · Market Access · Medical Affairs US Alzheimer's Disease Payer & HTA CMS's coverage-with-evidence-development registry, not IRA negotiation, is the anti-amyloid access gate. ICER's below-value verdict and Part B routing set the rest. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Metabolic · Market Access · Medical Affairs US Obesity Payer & HTA Medicare covers Wegovy only for cardiovascular risk, not obesity alone. The IRA's IPAY 2027 semaglutide price applies across the franchise, and ICER returned a 2025 'high value' verdict. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Pulmonology · CI Team · Launch Lead US COPD Payer & HTA COPD access is a pharmacy-benefit story: inhalers and biologics run through Medicare Part D and commercial PBMs, not medical coverage. Step edits gate the base, an eosinophil threshold gates the biologic, and the IRA is reshaping both price exposure and negotiation risk. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Immunology · CI Team · Launch Lead US Plaque Psoriasis Payer & HTA US access to plaque psoriasis biologics is gated by step therapy and reshaped by two forces landing together. IRA price negotiation on Stelara and Enbrel, and biosimilar erosion of adalimumab and ustekinumab. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Oncology · CI Team · Launch Lead US Breast Cancer HR+/HER2- Payer & HTA The price ceiling for HR+/HER2- oral therapies is now set directly by the government. Palbociclib was selected for Medicare negotiation with a 50% cut, $15,741 to $7,871, effective 2027. Access runs through Part D and commercial prior authorization, and newer targeted agents face biomarker-gated coverage with cost-effectiveness ratios far above accepted thresholds. US · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Payer Hta FR PNH Payer & HTA France reimburses iptacopan second-line only, after at least six months on a C5 inhibitor, while ravulizumab holds the first-line position. The restriction, not the price, is what defines the addressable population. FR · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC Myasthenia Gravis Payer & HTA Generalised myasthenia gravis has no formal NPHC programme. Efgartigimod access runs through private insurance, fastest at 1-4 weeks, or hospital pharmacy committees, with NPHC engagement targeted for 2025-2026. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC PNH Payer & HTA NPHC's KSA-first coverage model sets the de facto GCC access bar for anti-C5 agents. Iptacopan faces a 12-24 month SFDA registration queue before NPHC even evaluates it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC Hereditary Angioedema Payer & HTA GCC HAE access is structurally two-tier: broad acute coverage, but a prophylaxis bar few clear. Only 30-40% of applicants clear NPHC's individual-case prophylaxis review, and private insurance beats the NPHC pathway on speed. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK IgA Nephropathy Payer & HTA NICE's accepted cost-effectiveness case for budesonide (TA937, updated by TA1128) rests on a 5–8 year modelled ESRD delay. The same mandatory ACEi/ARB gate applied to sparsentan narrows the UK's eligible IgA nephropathy population from 10,000–15,000 to 3,000–5,000 patients. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK PNH Payer & HTA Both ravulizumab and iptacopan cleared NICE's standard Technology Appraisal route (TA698 and TA1000). The real payer question is how fast the NHS converts patients from IV ravulizumab to oral iptacopan, not which drug got the easier appraisal at the ordinary £20,000–£30,000/QALY bar. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC Sickle Cell Disease Payer & HTA The GCC's largest rare-disease programme by patient volume sits in a commercial vacuum. Crizanlizumab and voxelotor are both withdrawn, leaving 8,000-10,000 NPHC-managed SCD patients ahead of 2025-26 gene therapy registration. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC Fabry Disease Payer & HTA NPHC has a 72%-cost-reduction incentive to switch amenable-mutation Fabry patients from ERT to migalastat. A single HEK293 assay lab in the entire GCC is the only thing standing in the way. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK Spinal Muscular Atrophy Payer & HTA All three funded SMA therapies in England reached the NHS through a conditional route. Zolgensma was recommended under HST15 and extended under HST24; nusinersen and risdiplam spent seven years in managed access before TA1162 moved them to routine commissioning. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK Pompe Disease Payer & HTA NHS England's Pompe commissioning policy defines a 45-50 patient switch-eligible cohort for avalglucosidase alfa (NICE TA821). That is a manageable NHS budget event; broader first-line uptake would be a materially larger one. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK Fabry Disease Payer & HTA Migalastat's HST4 recommendation in 2016 was the first oral mutation-specific rare disease therapy NICE approved. Agalsidase beta was never formally appraised at all. Together they underpin an estimated £144M NHS Fabry programme, with a £70-130K/patient/year switch incentive still unrealised at scale. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK ATTR Amyloidosis Payer & HTA NICE rejected tafamidis for ATTR-CM in May 2021 (TA696) and recommended it in June 2024 (TA984). Acoramidis (TA1121, January 2026) and vutrisiran (TA868 for ATTR-PN, TA1115 for ATTR-CM) complete the UK amyloidosis market. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC Spinal Muscular Atrophy Payer & HTA SMA is the most mature rare-disease access model in the GCC. All three modalities are NPHC-covered, with Zolgensma's outcomes-based milestone rebate the GCC-first template other programmes follow. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK Myasthenia Gravis Payer & HTA NICE's June 2025 rejection of efgartigimod (TA1069) leaves UK myasthenia gravis with no NICE-recommended novel agent. Eculizumab's own appraisal (TA636) was withdrawn by the manufacturer in 2020 without a cost-effectiveness verdict, and the NHS IVIg cost-offset argument is now the strongest lever for a future resubmission. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC ATTR Amyloidosis Payer & HTA GCC tafamidis costs roughly a tenth of its US price, yet uptake is not limited by affordability. Tc-PYP scintigraphy access at fewer than 8 GCC centres is the real constraint. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK Hereditary Angioedema Payer & HTA NICE TA606 recommended lanadelumab in 2019 with a confidential simple-discount patient access scheme, and the first national UK audit found total attacks fell 87.7% at 6 months. Berotralstat's TA738 recommendation is measured against that benchmark. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC Pompe Disease Payer & HTA NPHC's well-established Pompe programme still gates avalglucosidase behind a 12-month failed-response switch criterion. Sanofi is pursuing NPHC first-line approval to bypass it. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK Dravet Syndrome Payer & HTA NICE recommended both Dravet therapies through standard Technology Appraisal, not the ultra-rare HST route. This covers what the Fintepla Cardiac Monitoring Scheme costs the NHS, and why the UK treatment algorithm is now closed to new entrants without a significant clinical edge. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Payer Hta DE PNH Payer & HTA Iptacopan's orphan-drug status let it clear Germany's AMNOG process with an established additional benefit and no comparator dossier at all. Ravulizumab's only PNH-specific G-BA review found no added benefit. DE · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs UK Sickle Cell Disease Payer & HTA NICE's SCD gene therapy appraisal may be the largest NHS rare disease budget event in history. It hinges on an annuity payment model that current NHS SCD management cost cannot yet clearly justify. UK · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC IgA Nephropathy Payer & HTA IgA nephropathy has no NPHC programme at all in the GCC. Access runs entirely through hospital pharmacy committees or private insurance, gated by a biopsy available at fewer than 20 GCC centres. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

P&HTA · Rare Disease · Market Access · Medical Affairs GCC Dravet Syndrome Payer & HTA The most effective Dravet agent is the least accessible in the GCC. Cannabidiol's Schedule-1-equivalent narcotics classification caps exceptional-import approval at 35-40%. GCC · In-Market · 24–32 pp · PDF · Excel · PPT

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