HAS split PNH into two pricing tracks: ravulizumab's SMR important, first-line rating against iptacopan's ASMR III, second-line-only restriction, negotiated by CEPS against a manufacturer-declared population of just 270 patients.
France assesses PNH therapies on two separate scales, not one. SMR (Service Médical Rendu), the actual medical benefit rating that sets the statutory reimbursement rate, rated ravulizumab (Ultomiris) important, and HAS positions it as first-line therapy, citing its every-eight-week dosing advantage over eculizumab's every-two-week schedule. ASMR (Amélioration du Service Médical Rendu), the added-value rating that anchors CEPS price negotiation, rated iptacopan (Fabhalta) III, a moderate improvement, in HAS's 5 December 2024 economic opinion, but restricted that rating to second-line use only, for adult patients with persistent hemolytic anaemia, haemoglobin below 10 g/dL, after at least six months on a C5 complement inhibitor.
That two-tier split shapes the price CEPS negotiates for each drug. Ravulizumab's published price, €14,988.66 for the 1100mg vial and €4,087.82 for the 300mg vial, applies across an unrestricted first-line population. Iptacopan's published price, €29,590.96 per box of 56 tablets, was negotiated against a target population the manufacturer itself estimated at only 270 patients nationally. Iptacopan reached French patients before that negotiation even closed: HAS's Collège granted an accès précoce (AP1) authorisation on 2 May 2024, fifteen days ahead of the drug's EU-wide marketing authorisation, with rebates accruing on invoiced turnover, capped at up to 80 percent, from day one. The definitive ASMR rating did not land until seven months later. A pricing strategy built on the US or UK label alone would miss both the second-line restriction and the accès précoce timeline that precedes it.
France PNH pricing — ravulizumab's first-line SMR-important track versus iptacopan's second-line ASMR III track
| Agent | SMR | ASMR | Position | Published Price |
|---|---|---|---|---|
| Ultomiris (ravulizumab) | Important | — | First-line | €14,988.66 (1100mg) / €4,087.82 (300mg) |
| Fabhalta (iptacopan) | — | III (moderate) | Second-line only (Hb <10 g/dL, ≥6mo prior anti-C5) | €29,590.96 per box of 56 tablets |
Sources: HAS Commission de la Transparence, Avis économique FABHALTA (iptacopan), 5 décembre 2024; HAS Commission de la Transparence, ULTOMIRIS (ravulizumab); HAS, Décision n°2024.0128/DC/SEM du 2 mai 2024; French public medicines database (base-donnees-publique.medicaments.gouv.fr).
What this model answers
Every section answers a named commercial question your team is asking, scoped to your asset.
Delivers
- HAS's two-track SMR/ASMR assessment
- the Hb <10 g/dL and six-month prior anti-C5 criteria gating iptacopan's second-line eligibility
- the every-eight-week dosing rationale behind ravulizumab's first-line position
Delivers
- The AP1 pre-AMM mechanism and its 2 May 2024 authorisation date, 15 days ahead of EU marketing authorisation
- the seven-month gap to the definitive ASMR rating
- the rebate mechanism reconciling the indicative price to the definitive CEPS price
Delivers
- Iptacopan's €29,590.96 negotiated price against its 270-patient target population
- ravulizumab's weight-based €14,988.66/€4,087.82 pricing as the first-line comparator
- the rebate cap of up to 80 percent of invoiced turnover
Custom model delivered in 72 hours.
Commission This ModelWhat's inside
- Why HAS's two-track SMR/ASMR split, not a single technology appraisal, determines iptacopan's negotiated price
- Pressure-tested against the ravulizumab first-line precedent before the rest of the model is built out
- How CEPS anchors price negotiation to the ASMR rating and the target population size
- Where iptacopan's 270-patient population changes the standard negotiation levers
- Why ravulizumab cleared SMR important and first-line while iptacopan cleared only ASMR III, second-line
- The Hb <10 g/dL and six-month prior anti-C5 eligibility gate
- Iptacopan's AP1 authorisation, 2 May 2024, 15 days ahead of EU marketing authorisation
- The rebate mechanism accruing on invoiced turnover, capped at up to 80 percent, before the definitive CEPS price lands
- Ravulizumab's weight-based €14,988.66/€4,087.82 pricing as the first-line comparator
- What the analogue class supports for a new entrant's defensible price
- The indicative accès précoce price, rebate layer, and definitive CEPS price decomposed line by line
- The reconciliation mechanism once the standard SMR/ASMR appraisal closes
- Sequencing recommendation across the accès précoce window and the definitive CEPS negotiation
- Conservative, base, and aggressive revenue scenarios tied to the target population size
- The open pricing questions your team must close before the France 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, HAS Commission de la Transparence opinions and CEPS pricing documentation, not secondary summaries. Findings are independently verified before inclusion.
PNH France pricing sources: HAS Commission de la Transparence economic opinion for iptacopan (5 December 2024), HAS Commission de la Transparence documentation for ravulizumab, HAS's accès précoce decision n°2024.0128/DC/SEM (2 May 2024), and the French public medicines database for reimbursement pricing.
- Iptacopan's ASMR III rating and second-line restriction verified against the live HAS Commission de la Transparence economic opinion
- Ravulizumab's SMR important rating and first-line status verified against HAS Commission de la Transparence documentation
- Accès précoce authorisation date and rebate cap verified against HAS decision n°2024.0128/DC/SEM and the Ministère de la Santé's remises/majoration procedure sheet
Frequently asked questions
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