Bernard Durkan (Kildare North, Fine Gael)
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689. To ask the Minister for Health the extent to which provision continues to be made for pharmaceutical companies providing for rare and orphan diseases; the extent to which applications are in hand for approval; and if he will make a statement on the matter. [22639/22]

Bernard Durkan (Kildare North, Fine Gael)
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690. To ask the Minister for Health the number of applications that remain on hand for approval for drugs to treat rare and orphan diseases; the length of time taken to process such applications; and if he will make a statement on the matter. [22640/22]

Stephen Donnelly (Wicklow, Fianna Fail)
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I propose to take Questions Nos. 689 and 690 together.

The Health (Pricing and Supply of Medical Goods) Act 2013 gives full statutory powers to the HSE to assess and make decisions on the reimbursement of all medicines taking account of a range of objective factors, clinical benefits, cost effectiveness and expert opinion as appropriate.

HSE decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds, on the advice of the National Centre for Pharmacoeconomics (NCPE).

The HSE Drugs Group is the national committee which the HSE has in place to make recommendations on the pricing and reimbursement of medicines. The role of the Drugs Group is to make a recommendation to the HSE Executive Management Team (EMT) in relation to each individual application having considered the criteria under the 2013 Act.

The Drugs Group considers the NCPE assessment, the outputs from commercial engagements, patient interest group submissions, any inputs provided by the Rare Disease Technology Review Committee (RDTRC) and any other pertinent information in advance of providing its recommendation to the HSE EMT.

As the decision-making authority within the HSE, the HSE EMT decides on the basis of all the demands with which it is faced (across all services) whether it can fund a new medicine, or new uses of an existing medicine, from the resources that have been provided to it in line with the 2013 Act.

The 2013 Health Act does not include provision for a different ruleset when assessing orphan medicines. However, the criteria that apply to the evaluation process allow the HSE to have particular regard for the unique circumstances surrounding orphan drugs, such as small patient populations and unmet clinical need. The overriding factor in medicines, including orphan medicines, making it through the process to reimbursement is the availability of Exchequer resources.

Budget 2021 allocated €50 million for the approval of new drugs and 19 of the 52 new medicines approved by the HSE in 2021 were orphan medicines, representing over one third of approvals.

The pipeline of new medicines coming to market remains strong and this government wants to secure fast access to medicines for the most vulnerable of our patients, including those for the treatment of rare diseases. The budget allocation of €30m funding for new medicines in Budget 2022 will provide for the reimbursement of both orphan and non-orphan new medicines this year.

To date in 2022, 24 approvals for reimbursement have been progressed by the HSE. There are a further 125 applications for reimbursement of new medicines in process with the HSE. A number of the medicines undergoing assessment currently have orphan designation and the HSE anticipates that a number of medicines with orphan status may be approved throughout 2022.

The Committee for Orphan Medicinal Products (COMP) is the European Medicine Agency’s committee responsible for recommending orphan designation of medicines for rare diseases. The European Commission decides whether to grant an orphan designation for the medicine based on the COMP's opinion. The HSE does not maintain a register of which medicines are designated orphan drugs, as the orphan status for a drug is subject to change at European level.