The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drug schemes, under the Health (Pricing and Supply of Medical Goods) Act 2013.In reaching a decision, the HSE examines all the evidence which may be relevant in its view for the decision and will consider such expert opinions and recommendations as it may have sought.
The Rare Diseases Medicinal Products Technology Review Committee is engaged with prescribers and the patient representative group to establish clear reimbursement guidelines for sapropterin. These would be expected to target sapropterin for patient cohorts for which it may be a cost-effective intervention.
The reimbursement guidelines are expected to include a requirement for response testing, to detail the criteria to identify patient cohorts for whom reimbursement of sapropterin would be a cost-effective use of resources and to include appropriate monitoring and control mechanisms. These engagements are ongoing.
The HSE Drugs Group will review the output of such engagements at the earliest opportunity to examine whether the above aims were achieved and will consider a reimbursement recommendation at that time.