Jim Daly (Cork South West, Fine Gael) | Oireachtas source

I thank Deputy Deering for raising this issue. I appreciate that a rare disease diagnosis places enormous stress on patients and their families and, as Deputy Deering is aware, access to potentially beneficial medicines for the treatment of rare diseases is extremely important for patients and their families.

I want to assure Deputy Deering that the Minister for Health works tirelessly to address issues around access for patients to new and innovative medicines. The Oireachtas has put in place a robust legal framework in the Health (Pricing and Supply of Medical Goods) Act 2013, which gives statutory powers to the HSE to assess and make decisions on the reimbursement of medicines, taking account of a range of objective factors and expert opinion, as appropriate. The Act specifies the criteria to be applied in the making of reimbursement decisions, to include the clinical and cost effectiveness of the product, the opportunity cost and the impact on resources that are available to the HSE.

While the 2013 Health Act does not include provision for a specific rule set when assessing orphan drugs, the HSE and the NCPE seek to take into account issues such as 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. In reaching its decision, the HSE will examine all the relevant evidence and will take into account such expert opinions and recommendations that are appropriate, including from the NCPE.

The Deputy rightly identifies that increasingly drugs are being developed to target rare conditions and that these drugs often come with very high list prices. In that respect, the HSE is required under the Act to have regard to the funding challenges that these drugs represent. It does this by drawing on the criteria contained in the Health Act, including the potential or actual budget impact of the drug in question and the cost effectiveness of meeting health needs by supplying a particular item rather than providing other health services. These are difficult decisions but recognise the core challenges of the availability of finite resources in the face of ever competing demands. Since the signing of the four year framework agreement on the supply and pricing of medicines in 2016, a significant number of orphan drugs have been reimbursed by the State.

Notwithstanding the challenges which orphan drugs present and the fact that the HSE and NCPE are mindful of these issues when assessing orphan medicinal products, a number of changes have been introduced to address those challenges. In June 2018, a technology review committee for rare diseases was established. This committee is now operational and has already completed work in relation to orphan products. As well as examining the methodologies for assessing orphan drugs, it will also consider the views of patients, caregivers and examine the wider issues surrounding health technology assessments. In addition, the composition of the HSE drugs group is being expanded to include two representatives from the National Patients Forum and more clinical expertise in the area of rare disease. Both of these measures are intended to provide greater balance and transparency to the assessment process as a whole.

The challenge of accessing innovative medicines at affordable prices is one shared by most, if not all, developed countries. It is estimated that in the region of 45 new molecules are due to receive market authorisation in Europe each year over the next five years. It is in this high-tech space, including orphan drugs, that the greatest challenges will arise in the years ahead. A significant development in Ireland’s international agenda was joining the BeNeluxA initiative on pharmaceutical policy in June 2018. This collaboration will support the Government's objective of co-operating with other European countries to identify workable solutions in an increasingly challenging environment to secure timely access for patients to new medicines, including orphan medicines, in an affordable and sustainable way.

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