Dáil Éireann Debate, Wednesday - 7 July 2021
165. Deputy Bernard J. Durkan asked the Minister for Health the number of rare diseases currently identified in Ireland; the extent to which various medications are available, are approval-pending or awaiting reimbursement approval; the number of patients affected; the expected timescale to bring matters up to date; and if he will make a statement on the matter. [30474/21]
View answerThere are approximately 8,000 rare diseases affecting around 30 million people in the EU.
In the EU, medicines for rare diseases must be authorised by the European Medicines Agency (EMA) and the European Commission under the ‘centralised procedure’. It is therefore not open to Irish authorities to grant a marketing authorisation for orphan medicines outside of this framework.
The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drugs schemes, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013.
In line with the 2013 Health Act and the national framework agreed with industry, a company must submit an application to the HSE to have a new medicine added to the reimbursement list.
Reimbursement is for licenced indications which have been granted marketing authorisation by the EMA or the Health Products Regulatory Authority.
As outlined in the national framework agreed with industry, and in line with the 2013 Act, the HSE will decide, within 180 days of receiving the application (or a longer period if further information is required from the company), to either add the medicine to the reimbursement list or agree to reimburse it as a hospital medicine, or refuse to reimburse the medicine.
In making a reimbursement decision, the HSE is required under the Act to have regard to a number of criteria including efficacy, the health needs of the public, cost effectiveness and potential or actual budget impact.
While the 2013 Health Act does not include provision for a different ruleset when assessing orphan drugs, the HSE seeks as far as possible to take into account issues such as the small patient numbers and the nature of the condition to be treated when evaluating these medicines. The criteria that apply to the evaluation process allow sufficient scope for the HSE to take on board the particular circumstances that pertain to orphan drugs and rare diseases.
There has been a number of key changes in the HSE assessment process of medicines within the confines of the 2013 Health Act. These have included changes to the HSE Drugs Group, whose membership has been expanded to include representatives from the National Patients Forum and more clinical expertise in the area of rare diseases.
In June 2018, the HSE Leadership appointed a Technology Review Committee for Rare Diseases (RDTRC). The Committee’s recommendations regarding reimbursement of orphan medicines are not intended to replace any part of the existing medicines appraisal or reimbursement process but rather complement it.
The additional funding of €50m provided for new medicines in Budget 2021 will allow the HSE to provide access to medicines which have been recommended by the HSE Drugs Group, on the basis of efficacy and value for money in line with the 2013 Health Act, and ultimately provide more medicines to Irish citizens. As of 16 June, the HSE had progressed a total of 26 new approvals for reimbursement of medicines in 2021.
The number of pricing/reimbursement applications currently underway for new medicines, including with respect to rare diseases, is an operational matter. As such, it is a matter for the HSE. However, members of the Oireachtas are advised that the HSE is currently unable to access the information to answer Parliamentary Questions due to the recent cyber-attack, which has required a temporary shut-down of HSE IT systems. The disruption to service is on-going, and the HSE is working hard to restore its IT capacity and resume normal services.
Members of the Oireachtas will be advised as soon as the HSE is again in a position to provide responses to PQs and are encouraged to resubmit their Parliamentary Questions at that point.
