Léim ar aghaidh chuig an bpríomhábhar

Dáil Éireann díospóireacht -
Tuesday, 5 Oct 2021

Vol. 1012 No. 1

Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2021: First Stage

I move:

That leave be granted to introduce a Bill entitled an Act to amend the Health (Pricing and Supply of Medical Goods) Act 2013 to establish specific criteria applicable for orphan medicinal products for the purposes of the Health Service Executive making a relevant decision regarding adding an item to the Reimbursement List.

Before I begin, I want to acknowledge the work of former Deputy John Brassil in regard to the introduction of this Bill, which, in essence, is a reintroduction of the work he undertook a few years ago. I also acknowledge that the Minister for Health is here today and that he made an extra €50 million available in the drugs budget in the last year, which is also to be commended.

Under the existing legislation, there is no differentiation between orphan medicinal products and other products in Ireland. In recent years it has become clear that there is an issue in Ireland whereby orphan drugs continually struggle to secure reimbursement, often despite their wide ability elsewhere in Europe. It is well acknowledged that Ireland is a lagging behind Romania, Hungary and other Eastern European countries in the provision of these drugs. This reimbursement processes is protracted, unnecessarily public and often antagonistic with patients in Ireland continually forced to protest publicly or engage with politicians to seek fairness and equity over the course of a two-year campaign from initial application to final decision.

I listened during Questions on Promised Legislation when Deputy Griffin mentioned the plight of people seeking approval for Zolgensma. Deputy Christopher O'Sullivan from west Cork raised the matter last week. Unfortunately, those families have the same journey to undertake as the sufferers of amyloidosis, who only recently got approval for the patisiran drug in the last couple of months. Unfortunately, that long journey awaits many of these families.

The issue of low availability of orphan drugs or treatments specifically designated for rare diseases is complex, with several factors contributing to the problem. These range from the appropriateness of the assessment mechanism to the current framework agreement between the State and pharmaceutical industry and industry pricing and prioritisation in Ireland.

This Bill is not a panacea to this complex issue. It does, however, seek to address one of the clear barriers to access in Ireland in how we apply a standardised assessment mechanism to medicines that are far from standard. Presently, the same health technology assessment, HTA, is carried out by the National Centre for Pharmacoeconomics, NCPE, for new medicines irrespective of their medicine type. This is a quality-adjusted life year, QALY, health technology assessment whereby quantitative data relating to the medicine and the disease it treats are used to assess its cost-effectiveness. In Ireland's case, this threshold is set at €45,000 per QALY. This mechanism presents several issues for orphan drugs, the quantitative data relating to which is often limited due to the low clinical data or the low epidemiology around a rare disease, given such a small patient population. As such, a QALY-based health technology assessment will result in huge uncertainty around an orphan drug. In the words of Professor Michael Barry, head of the NCPE, when he appeared before the previous Joint Committee on Health, "none of the drugs for rare diseases will reach the €45,000 euro per QALY [threshold]". The current system is designed for orphan drugs to fail.

The consequence of using this form of health technology assessment is that commercial negotiations between the State and the industry commence from a poor position whereby the information available to the HSE is of limited value. This is directly contributing to the two-year process of protracted negotiations as both parties seek to reach a fair and accurate agreement of the true value of medicine.

I will now speak to the Bill. Responsibility for addressing this issue has fallen between two stools, with previous Ministers for Health stating at every opportunity that statutory responsibility for medicine, pricing and reimbursement decisions lies with the HSE in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013. For its part, the HSE has informed Members of this House, through various health committees, that the 2013 Act does not make separate provision for orphan drugs and that, as a result, the processes and procedures do not make separate provision for distinct criteria on the assessment of orphan drugs. When challenged on whether it had any flexibility under the Act to allow for a tailored approach to assessing orphan drugs, the HSE stated that it had pushed legislation as far as it could.

The Bill seeks to resolve this issue by clarifying for the HSE the application of the health technology assessment for assessing orphan drugs as well as establishing set criteria to be considered when making a relevant decision on the reimbursement of an orphan drug. It clarifies that the HSE, when carrying out a HTA, should do so in a manner that does not rely on an incremental cost-effectiveness ratio threshold but should instead conduct a HTA that will provide a meaningful and credible assessment of an orphan drug. This is not to suggest we do not carefully consider an orphan drug but that we look to EU member states like Sweden that have adapted their HTA such manner.

The hardest thing I have had to do since I was elected was sit across a kitchen table from one of the families who have been seeking approval for orphan drugs and hear their individual story of being told that if they do not get medicine or a tablet by a certain date then the person will undoubtedly either die, suffer from severe mental trauma or whatever the case may be.

This Bill is costed and there are budgetary constraints on it. I again acknowledge John Brassil's efforts with it. We are not asking for a magic money tree to provide medicines for these people. We are asking that orphan drugs for illnesses very few people in this country suffer from - they are, by their nature, rare diseases - be subject to the same assessment, in the context of qualitative data, as other drugs.

Is the Bill opposed?

Question put and agreed to.

Since this is a Private Members' Bill, Second Stage must, under Standing Orders, be taken in Private Members' time.

I move: "That the Bill be taken in Private Members' time."

Question put and agreed to.