Kate O'Connell (Dublin Bay South, Fine Gael)
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307. To ask the Minister for Health if an independent expert to review the appropriateness, transparency and timeliness of the HSE's reimbursement process for orphan medicinal drugs will be appointed in view of a growing number of orphan drugs failing to secure reimbursement here despite being made available in other EU countries; and if he will make a statement on the matter. [52839/17]

Simon Harris (Wicklow, Fine Gael)
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The Health Service Executive (HSE) has statutory responsibility for decisions on the pricing and reimbursement of medicines, under the Health (Pricing and Supply of Medical Goods) Act 2013. The 2013 Act specifies the criteria to be applied in making decisions on the reimbursement of medicines.

The issue of orphan medicines, with very small patient populations, and the price barriers for health systems in supplying them to patients, is world-wide. Orphan product manufacturers have exclusive patents and are monopoly suppliers. They can, and frequently do, seek very high prices for these products.

The HSE seeks, where possible, to give patients early access to new treatments. However, it must work within the budget provided by the Oireachtas. In the statutory reimbursement process, supported by the 2016 Framework Agreement with industry, the HSE makes decisions on objective scientific and economic grounds. Criteria which must be considered include the population’s health needs, the product’s cost and clinical effectiveness, opportunity costs, the budget impact and available resources.

HSE decisions are supported by health technology assessments (HTAs), which systematically assess whether a drug is a cost-effective health intervention. Currently, most new medicines, including orphan products, undergo HTAs. However, not all applications have sufficient information for assessment and in such cases this must be sought. Also, as HTAs frequently cannot demonstrate value for money for items at the price sought by the company, lengthy price negotiations between the HSE and the manufacturer may be needed. These factors contribute significantly to the length of the reimbursement decision making process.

A new Committee called the “Rare Diseases Medicinal Products/Technology Review Committee” was established in June 2017 by the National Centre for Pharmacoeconomics. The terms of reference for the Committee have been finalised and the responsibilities of the Committee include the review of proposals for funding of new products for rare diseases or expanded indications for existing products for rare diseases. The composition of the Committee will ensure that the voice of patients, patient groups and clinical experts will be available to the HSE when considering reimbursement of drugs for rare diseases.

The Committee will also contribute to the development of clinical guidelines for relevant Orphan Medicinal Products and support the implementation of same.

Dr. Michael Barry was appointed the Chair of the Committee on the 13 September 2017. It is expected that Committee will begin its work in early 2018 after appointment of the necessary support staff.

With the potential costs involved and the competing demands across the health service, every effort must be made to secure the best outcome for the health service and the taxpayer. This includes seeking realistic and sustainable pricing and supply models for new treatments.