John Brassil (Kerry, Fianna Fail) | Oireachtas source

Under the current Act, there is no differentiation between orphan medicinal products and other products. In recent years, it has become clear that there is an issue in Ireland whereby orphan drugs continually struggle to secure reimbursement here, often despite their wide availability elsewhere in Europe. This reimbursement process is protracted, unnecessarily public and often antagonistic, with patients 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 in Ireland to its final decision.

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

This Bill is not a panacea to this complex issue but looks 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. Currently, the same health technology assessment 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, based health technology assessment whereby quantitative data on the medicine and the disease it treats are used to assess its cost effectiveness against an incremental cost effectiveness ratio, ICER. In Ireland's case, this threshold is set at €45,000 per quality-adjusted life year.

This mechanism presents several issues for orphan drugs, which often possess limited quantitative data due to 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 great uncertainty around orphan drugs. Last month before the Joint Committee on Health, Professor Michael Barry, head of the NCPE, stated that none of the drugs for rare diseases will reach the €45,000 per quality-adjusted life year.

The consequence of using this form of health technology assessment is that commercial negotiations between the State and industry commence in a poor position and 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 the medicine. From a broader perspective, this two-year delay does not serve patients who need orphan drugs because, as per the European Medicines Agency licensing requirement, they have life-threatening or chronically debilitating diseases. The delay can swiftly exceed two years as companies deprioritise the Irish market in favour of European access. This is in light of Ireland's growing reputation for having a slow and challenging access environment in respect of drugs used to treat rare diseases.

I will now comment on the Bill. Responsibility for addressing this matter has fallen between two stools. The Minister for Health has stated 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 informed the Joint Committee on Health that the 2013 Act does not make separate provision for orphan drugs. As a result, the processes and procedures do not make separate provision for distinct criteria for the assessment of orphan drugs. When challenged on whether the HSE had any flexibility under the Act to allow for a tailored approach to assessing orphan drugs, the executive's representatives informed the joint committee that it has pushed the legislation as far as it can. The Bill seeks to resolve this issue by clarifying for the HSE the application of the health technology assessment for evaluating orphan drugs, as well as establishing set criteria to be considered when making a relevant decision on reimbursement in respect of an orphan drug. It clarifies that the HSE should carry out health technology assessments in a manner that does not rely on an incremental cost-effective ratio threshold. Instead, it should conduct assessments that will provide meaningful and credible evaluations of orphan drugs.

This is not to suggest that we should not carefully consider orphan drugs. Rather, we should look to EU member states such as Sweden, which has adapted its health technology assessment process in such a manner. In addition, the Bill places on a statutory footing some important considerations for the HSE to assess before making a final decision on an orphan drug. These include the availability of the drug elsewhere in Europe, guaranteed input of patients and the level of certainty that can be provided by industry through risk-sharing agreements. The Bill also acknowledges the limitations of traditional quantitative data. Importantly, the Bill does not seek to undermine the ability of the HSE to seek an affordable price from industry. Important criteria, such as the potential budget impact of the drug and the resources available to the HSE, are maintained as statutory considerations. As such, I see no specific budgetary implications. This Bill simply aims to speed up the process and ensure that it starts on the right footing.

It should be acknowledged that there are ongoing reforms in the HSE with regard to orphan drugs, driven in part by the work carried out by the members of the Joint Committee on Health during the past two years. Reforms include the creation of a rare disease technology review committee. This is welcome, as is the Minister's engagement with other EU member states via the BeNeLuxA and Valetta initiatives. It has been acknowledged by several Members that European co-operation is a logical but long-term solution that is unlikely to materially resolve the orphan drug issue, which is immediate and urgent. The technology review committee will ultimately consider a flawed health technology assessment and, as such, this Bill remains a necessary reform that must proceed.

A collaborative approach is required with the input of the Minister for Health and his officials in conjunction with the Joint Committee on Health. On 27 September last year in a debate on the availability of the drug Ocrevus, the Minister stated:

If people feel that the current system is broken, I would like to hear their views on what should replace it. I think it is broken too. Coming up with a better model is the conundrum.

I am providing the Minister with an answer to that conundrum. I am asking the Minister to work with me and to support the Bill to stop the endless marching and protesting that seems to be required for every drug that comes before us. I urge the Minister to support this measure and I urge every Member likewise.

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