The NCPE completed a rapid review of onasemnogene abeparvovec intrathecal (Itvisma®), Novartis’s intrathecal gene therapy for spinal muscular atrophy (SMA), making this the first national HTA assessment completed for a medicine undergoing an EU Joint Clinical Assessment (JCA) under the EU HTA Regulation. The JCA report was endorsed by the Member State Coordination Group on 16 July 2026. The NCPE’s national rapid review was completed and published only 16 days after EU marketing authorisation. 1
The NCPE recommended that Itvisma should not be reimbursed at the price submitted by Novartis. This is the first instance where Ireland has declined reimbursement of a medicine that has completed the EU JCA process.
The EU JCA assesses only the clinical evidence — whether the medicine works and for whom. It does not assess cost-effectiveness or set a price. Each member state then makes its own national decision on whether to reimburse and at what price. This is different from the NICE process in England, where cost-effectiveness is assessed as part of the technology appraisal itself, so price is built into the approval decision. In Ireland, the NCPE’s rapid review considers cost-effectiveness separately from the clinical evidence, and the HSE Drugs Group then negotiates the price. The NCPE’s “not at the submitted price” recommendation is therefore not a final rejection; it is a signal that the submitted price is too high and that reimbursement remains possible if Novartis reduces it in subsequent negotiations.
‘Notably, Ireland completed and published its national rapid review before the European Commission’s publication of the JCA report, demonstrating how Member States can incorporate the confidential JCA assessment into national HTA processes ahead of its public release while enabling faster reimbursement decision-making.’ 1
HSE Drugs Group — Rare Disease Drug Delays Draw Public Criticism
On 15 July 2026 RTÉ’s Prime Time broadcast a report on rare disease drug delays in Ireland. Campaigners and patients gathered outside Leinster House ahead of a HSE Drugs Group meeting, urging a positive reimbursement decision. One patient, Emily Felix from Kilkenny described having waited more than 700 days since the HSE first began assessing a medicine for her Friedreich’s Ataxia. The report highlighted the continuing gap between NCPE recommendation and final HSE reimbursement decision as a source of significant distress for patients with rare diseases. 2
End-to-End Review — Timeline
The Minister for Health’s commissioning of an end-to-end review of Ireland’s drug approval system, announced on 11 June 2026, is expected to produce its findings by December 2026. No further formal announcements on scope or process have been made during July 2026.
Blueteq comment: The NCPE’s completion of the first national HTA building on EU JCA data — in just 16 days from marketing authorisation — is impressive. However, rejecting Itvisma at the submitted price, and the continuing public pressure over rare disease delays, illustrate that speed of initial assessment does not resolve the governance challenge. After a medicine is accepted for reimbursement then ensuring it reaches only eligible patients and that the expenditure is tracked and audited at patient level remains the unresolved gap that Blueteq addresses.
References
1. Ireland Issues First JCA-Based National HTA Decision, Declines Reimbursement of Itvisma at Submitted Price. NAVLIN Daily. July 2026. https://www.navlindaily.com/article/31281/ireland-issues-first-jca-based-national-hta-decision-declines-reimbursement-of-itvisma-at-submitted-price
2. ‘Heartbreaking’ — Campaigners criticise rare disease drug delays. RTÉ News Prime Time. 15 July 2026. https://www.rte.ie/news/primetime/2026/0715/1583457-rare-disease-drug-delays/
