Mr. John Hennessy:

I thank the joint committee for the invitation to discuss orphan drugs. As Mr. Judge said, I am joined by my colleagues, Professor Michael Barry, Mr. Shaun Flanagan and Mr. Ray Mitchell from our parliamentary affairs unit. Our preparations focused on the committee's report of February 2018 and the measures taken or under way to address the recommendations contained in it. Mr. Judge has covered the main policy and legislative elements. I will focus on the operational aspects.

On recommendation No. 4, I confirm that discussions have commenced with the pharmaceutical sector to provide clarity on pricing and measures to speed up the negotiating process. The discussions have also explored the issues of risk sharing and post-approval review to ensure ongoing expenditure is justified by evidence of clinical effectiveness.

On recommendations Nos. 6 and 7, there is already a high degree of communication between the HSE and the pharmaceutical sector, as well as, of course, with patient advocacy groups. However, steps have been taken to strengthen this engagement through the provision of additional resources at both NCPE level and within the corporate pharmaceutical unit. This is intended to enable representations to be considered at both the HTA and negotiation stages. There is also routine engagement with the pharmaceutical industry, while the composition of the drugs group has been expanded to include patient representatives and more clinical expertise in the area of rare diseases. Enhancements have also been made regarding medicines for older persons and medical ethics.

The HSE has written to hospital groups setting out the importance of having a clear understanding and informed consent arrangements in place prior to the commencement of clinical trials and compassionate access programmes. The establishment of a specific budget for high technology and orphan drugs is also being examined. This was also a recommendation made in the report. To a large extent, it is already something we have in place in that high technology and orphan drugs are, by and large, funded centrally, rather than from individual hospital budgets.

The measures now under way to strengthen the representation of rare diseases in the assessment process and to involve patient representation and medical ethics should also refine the process of assessing medicines in the manner intended and recommended by the committee. The issues highlighted in recommendation No. 10 are being pursued further with the HRB, which appears to be the most obvious route to influence research in collaboration with the university sector. With regard to recommendation No. 12, the HSE has established a national rare disease technology review committee. The role of the committee is to enable clinicians and other stakeholders to input into the assessment process and in the post-health technology assessment phase to review proposals, prescribing guidelines etc. for consideration by the drugs group. This is designed to operate similarly to the National Cancer Control Programme therapeutic review committee, which has been operating successfully. Such guidelines will, where appropriate, include recommendations relating to the prescribers who should be enabled to access orphan medicines. I will ask my colleague, Professor Barry, to elaborate on the work of this group. A model of care has also been prepared by the HSE’s clinical programme for rare diseases, which aims to improve access to care for patients with rare conditions and improve value and quality in this area.

As the process for evaluating applications was covered in detail at our meetings with the committee in 2017, I will not repeat this other than to emphasise again that the criteria for assessment are as detailed by the legislation, the Health (Pricing and Supply of Medical Goods) Act 2013, and as set out specifically in section 19(4) of the Act. The important point to bear in mind is that the legislation requires the HSE to have regard to the cost-effectiveness and the clinical benefits of the product being applied for. The particular features that relate to orphan conditions are obviously considered during the assessment process and factors such as clinical effectiveness and the number of potential patients affected are clearly set out in the reports compiled. As members are aware, the HSE currently spends in excess of €2 billion per year on drugs and medicines, and on a per capitabasis the Irish health service continues to be one of the highest spenders on medicines in the OECD area, with expenditure still increasing annually. I will now hand over to Professor Barry for further detail on the rare diseases technology review group.