Bernard Durkan (Kildare North, Fine Gael)
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381. To ask the Minister for Health the extent to which rare and orphan medicines continue to be tested and make available here; the extent to which progress is slow on any particular medicine; and if he will make a statement on the matter. [27129/22]

Stephen Donnelly (Wicklow, Fianna Fail)
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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.

Reimbursement is for licenced indications which have been granted market authorisation by the European Medicines Agency (EMA) or the Health Products Regulatory Authority.

The Committee for Orphan Medicinal Products (COMP) is the EMA's committee responsible for recommending orphan designation of medicines for rare diseases. This designation is for medicines developed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or very serious. The European Commission decides whether to grant an orphan designation for the medicine based on the COMP's opinion. The application process for this designation takes a maximum of 90 days from validation.

In the EU, medicines for rare diseases must be authorised by 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. Evaluating a marketing-authorisation application under the centralised procedure can take up to 210 days, not counting clock stops when applicants have to provide additional information. It is possible for an orphan medicine sponsor to submit an application through an accelerated assessment procedure. This procedure reduces the timeframe for the EMA to review the marketing-authorisation application to 150 days, not including any clock stops. It will then be up to the sponsor company to respond promptly to requests for additional information or data to support its application to avoid extended clock stops.

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. 

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 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 principal factors determining the speed of reimbursement are the price at which a manufacturer applies for the reimbursement of a product, the availability of Exchequer funding, and the outcome of the HSE’s rigorous assessment process as to its clinical and cost-effectiveness.

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 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.

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. However, the HSE anticipates that a number of medicines with orphan status are likely to be approved throughout 2022.