Despite PNH being rare enough to qualify on prevalence, NICE has appraised every modern anti-complement therapy through the standard £20,000-30,000 Technology Appraisal route, not the Highly Specialised Technologies threshold, so a new entrant must defend its price against the confidential-PAS precedent set by ravulizumab, pegcetacoplan and iptacopan.
NICE runs two appraisal tracks for ultra-rare conditions: the standard Technology Appraisal, which applies a £20,000 to £30,000 QALY threshold, and the Highly Specialised Technologies route, which applies a far higher £100,000 to £300,000 threshold reserved for the very rarest conditions. PNH sits close to the prevalence boundary, but in practice NICE has routed every modern anti-complement therapy through the standard track. Ravulizumab was recommended under TA698 in 2021, then pegcetacoplan under TA778 and iptacopan under TA1000, each an ordinary single technology appraisal cleared at the standard threshold rather than the HST band. Eculizumab, the original C5 inhibitor, predates these and was commissioned directly by NHS England as a highly specialised service rather than through a numbered NICE appraisal.
That consistency is the pricing problem for any new entrant. A £20,000 to £30,000 threshold, roughly a tenth as forgiving as the HST band, is the bar ravulizumab, pegcetacoplan and iptacopan each had to clear, and each did so through a confidential patient access scheme discount off a high list price rather than through a lower list price. Crovalimab took a different route: a NICE cost-comparison appraisal (TA1019, November 2024) that recommended it as lower-cost than the C5 incumbents rather than re-litigating the cost-effectiveness case. A new anti-complement drug therefore has to model three things at once: the discount depth needed to clear the standard threshold, the confidential-PAS precedent the incumbents have already set, and whether a cost-comparison positioning against an existing agent is available instead. Our price ladder models each against the ravulizumab, pegcetacoplan and iptacopan comparator set.
UK PNH pricing — every modern anti-complement drug cleared the standard NICE threshold
| Agent | NICE Route | QALY Threshold | UK Access Status |
|---|---|---|---|
| Ravulizumab (Ultomiris) | Standard Technology Appraisal (TA698) | £20,000–30,000 | Recommended, confidential PAS; the most-used C5 inhibitor |
| Pegcetacoplan (Aspaveli) | Standard Technology Appraisal (TA778) | £20,000–30,000 | Recommended for residual anaemia after a C5 inhibitor |
| Iptacopan (Fabhalta) | Standard Technology Appraisal (TA1000) | £20,000–30,000 | Recommended, confidential commercial arrangement |
| Crovalimab (Piasky) | Cost-comparison appraisal (TA1019) | cost-comparison vs C5 incumbents | Recommended Nov 2024 as lower-cost than eculizumab/ravulizumab; SC every 4 weeks |
| Eculizumab (Soliris) | NHS England highly specialised commissioning | commissioned directly | Original C5 inhibitor, ~15 designated PNH centres |
Sources: NICE TA698 ravulizumab for PNH (2021); NICE TA778 pegcetacoplan for PNH; NICE TA1000 iptacopan for PNH; NICE TA1019 crovalimab for PNH (cost comparison, 2024); NHS England Highly Specialised Services specification for PNH.
What this model answers
Every section answers a named commercial question your team is asking, scoped to your asset.
Delivers
- The prevalence and precedent logic behind NICE routing every modern PNH anti-complement drug through the standard Technology Appraisal
- what that means for the threshold a new entrant must clear
Delivers
- Price ladder modelling against the standard NICE ICER threshold
- confidential-PAS discount scenarios benchmarked to the ravulizumab, pegcetacoplan and iptacopan precedent
Delivers
- How a NICE cost-comparison appraisal works, when an anti-complement entrant can position as non-inferior to an existing comparator, and the pricing implication versus a full cost-utility submission
Custom model delivered in 72 hours.
Commission This ModelWhat's inside
- Why the standard £20,000-30,000 STA threshold, not the HST band, is the bar every PNH price must clear
- Pressure-tested against the ravulizumab, pegcetacoplan and iptacopan appraisal record before the rest of the model is built out
- How the £20,000-30,000 standard threshold is calculated and where the incumbent PAS-negotiated prices sit against it
- What a new entrant's list price and net price would need to be to clear the same bar
- Which markets could reference a UK PAS-negotiated price
- Sequencing risk if a UK discount undercuts the German AMNOG outcome, or vice versa
- Ravulizumab, pegcetacoplan, eculizumab and iptacopan pricing as the comparator set
- What the analogue class supports for a new entrant's defensible price
- Modelling a new entrant's ICER against the £20,000-30,000 standard band
- Where quality-of-life evidence is strong enough to defend a premium within that band
- Statutory and PAS-negotiated discount components decomposed line by line
- The discount depth needed to clear the standard threshold at a given list price
- Sequencing recommendation across a full cost-utility STA versus a cost-comparison appraisal positioning
- Conservative, base and aggressive revenue scenarios under each route
- The open pricing questions your team must close before the UK launch price is locked
- Structured for an internal pricing committee session
Included with every brief
How AXLRx builds this model
Prepared by MoatRx analysts.
Every AXLRx pricing model is built from primary regulatory sources (NICE, MHRA, NHS England) and live technology-appraisal documentation, not secondary summaries. Findings are independently verified before inclusion.
PNH UK pricing sources: NICE TA698 (ravulizumab), TA778 (pegcetacoplan) and TA1000 (iptacopan), NICE TA1019 (crovalimab, a cost-comparison appraisal), and the NHS England Highly Specialised Services specification for PNH. Each appraisal was checked to confirm the standard Technology Appraisal route and threshold, not the Highly Specialised Technologies band.
- NICE standard Technology Appraisal QALY threshold range verified against NICE methods guidance
- Ravulizumab TA698, pegcetacoplan TA778 and iptacopan TA1000 confirmed as standard single technology appraisals against live NICE guidance
- Crovalimab's NICE recommendation via the TA1019 cost-comparison appraisal verified against live NICE guidance
- New-entrant pricing benchmarked against the confidential-PAS precedent set by the incumbent anti-complement appraisals
Frequently asked questions
Commission this model
AXLRx delivers PNH pricing strategy models built for market access and pricing teams navigating NICE's standard Technology Appraisal threshold and cost-comparison appraisal dynamics. Custom model in 72 hours.
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Research-verified pricing model in 72 hours with optional analyst readout.