All three UK PNH agents cleared NICE's standard pathway, not the ultra-rare HST route, so dosing convenience now decides NHS share.

NHS England commissions PNH complement-inhibitor therapy through 15 Highly Specialised Services centres, concentrated at Leeds, King's, Oxford, Addenbrooke's, and Glasgow, for an estimated 600 diagnosed UK patients. Ravulizumab (Ultomiris) holds the NHS anchor position, having cleared NICE as a standard Technology Appraisal (TA698, published 19 May 2021) on the strength of its q8w dosing versus eculizumab's q2w regimen. Despite PNH's ultra-rare prevalence, ravulizumab did not use NICE's Highly Specialised Technology pathway. It met the standard TA cost-effectiveness bar at its negotiated NHS price, the same route the rest of the class has since followed.

Iptacopan (Fabhalta) has since cleared its own standard Technology Appraisal as TA1000, NICE's 1,000th published appraisal, with a final positive recommendation now in place. The access uncertainty around its estimated £350,000 to £400,000 annual WAC has been resolved in the drug's favour, and NHS access no longer runs through Individual Funding Request. Crovalimab (Piasky) has also been recommended by NICE, for patients aged 12 and over weighing at least 40kg, on the strength of COMMODORE 2 data. Its subcutaneous self-injection every four weeks is now a live convenience argument in market, not a pending one.

With access settled for all three, the fight moves to dosing convenience.

Five questions this report answers:

Q1 - Why did all three PNH agents clear NICE's standard route instead of the ultra-rare HST pathway?

Q2 - Which NHS centres control PNH prescribing, and how is the ravulizumab-over-eculizumab switch reshaping share?

Q3 - Now that iptacopan and crovalimab are both recommended, how does the convenience contest play out?

Q4 - How does ravulizumab's q8w dosing versus eculizumab's q2w regimen drive the NHS switch programme?

Q5 - What did resolving iptacopan's £350,000-400,000 annual WAC uncertainty mean for NHS access?

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Mike || Global Pharma Commercial Marketing Head

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