• The Terms of Reference of the HTA were agreed between HIQA and the National Clinical Lead in Child Health Public Health in the Health Service Executive (HSE).
• An Expert Advisory Group was convened comprising representation from relevant stakeholders. These included the HSE, the Department of Health, Children’s Health Ireland, patient representatives, and clinicians, nurses and medical social workers with specialist expertise. 
• A protocol for the work to be undertaken was reviewed by the HIQA Expert Advisory Group, and published on the HIQA website (link). 
• The epidemiology of these conditions in Ireland and the associated burden of disease was described. 
• A high level description of HSCT and the outcomes associated with its use for these indications was provided. 
• The current standard of care and treatment pathway for these patients, and the proposed pathway if HSCT treatment for all paediatric conditions were to be repatriated to Ireland, were described.  
• The governance, organisational and legal implications of HSCT service expansion, including the impact on the resilience of the service, were assessed.  
• The budget impact of providing HSCT in Ireland was provided across multiple perspectives.  
• The ethical implications that the provision of HSCT in Ireland may have for patients, their families, the general public and the healthcare system in Ireland were reviewed.
• The draft report was reviewed by the Expert Advisory Group. A final draft report was submitted to the Board of HIQA for approval. Following its approval, the completed assessment was submitted to the Minister for Health and the HSE as advice.
   

Allogeneic haematopoietic stem cell transplant (HSCT) is the recognised standard of care for paediatric patients with particular inborn errors of metabolism (IEM), inborn errors of immunity (IEI) and certain haemoglobinopathies (depending on the form and severity of the disease). Compared to the general Irish population, patients who required HSCT for these conditions are more likely to be Irish Travellers or of non-Irish ethnicity

HSCT is provided by Children’s Health Ireland (CHI) for paediatric patients with haematological malignancies and certain benign conditions. However, paediatric patients with IEM, IEI and haemoglobinopathies must travel abroad to obtain HSCT. This is associated with a significant administrative, logistical, financial and emotional burden for families given a treatment period abroad of between two to six months.

Repatriation of HSCT services for these conditions would necessitate an additional 10 to 13 allogeneic HSCTs, on average, each year at CHI, potentially doubling the number of such procedures currently undertaken (average of 12 per year). From the perspective of the HSE, while subject to substantial uncertainty, the estimated five-year incremental budget impact is expected to result in cost reductions. Over a five-year time horizon, depending on whether costs arising from ancillary procedures are excluded or included, the reduction is estimated as €2.3 million (95% CI -€5.8m to +€1.1m) or €1.5 million (95% CI -€5.0m to +€1.9m), respectively. 

Additional considerations relevant to a decision to repatriate HSCT services for these conditions, include the following:

• While there will be potential fluctuation in demand, on average, there would be sufficient capacity for all patients when services move to the new National Children’s Hospital and the number of dedicated transplant beds increases from four to six.
• The ability to accommodate any increase in HSCT activity would be contingent on the recruitment of additional staff, such as skilled nursing staff and support staff across a range of disciplines.
• Repatriation would reduce the burden on ethnic minority groups who are both disproportionately represented in this cohort and who are disproportionately impacted by challenges associated with travelling abroad for care.
• Patients undergoing HSCT in the UK have access to procedures not currently provided in Ireland by the HSE; these include extracorporeal photopheresis for the treatment of graft-versus-host disease and the provision of procedures aimed at preserving fertility. The provision of such services in Ireland for paediatric HSCT patients would have considerable organisational and resource implications.

In the event of a decision to repatriate HSCT services for these conditions:

• A phased approach to implementation may be required to support the build-up of sustainable capacity within the service.
• Given the potential fluctuation in demand, there may be a requirement for contingencies at times when there are multiple high-priority patients for transplant and demand exceeds available capacity. This could include continued agreements with centres abroad.
• Careful consideration of the process and criteria applied for the prioritisation of patients for transplant (including criteria to determine which patients are referred abroad for treatment when demand exceeds capacity), would be required to ensure equity and fairness.