Seanad Éireann debate -
Tuesday, 9 Mar 2010

Vol. 201 No. 7

Health Services

Senator Jerry Buttimer

I thank the Cathaoirleach for allowing me to place this motion before the House. I welcome the Minister of State and compliment his fortitude on this marathon day in the House.

The motion before us is about the need for a national plan on rare diseases. I pay tribute to the Irish Platform for Patients' Organisations, Science and Industry, IPPOSI, which has done considerable work alongside other umbrella bodies and organisations. According to IPPOSI, "to be considered rare a condition should not affect more than a limited number of people out of a whole population". Herein lies the difficulty. Given the rarity, fewer voices are calling for help. For this reason, we need a national plan for rare diseases. I pay tribute to Deputy Stanton who raised this matter with the Minister for Health and Children in the Lower House last week. It is important we create awareness.

IPPOSI has identified three large hurdles faced by patients with rare diseases, those being access to correct diagnosis, a lack of information and scientific knowledge and access to treatment if it exists. Living in the 21st century, it is important we recognise the steps humankind has taken. We put a man on the moon, yet there are people who are unable to get an answer as to why they are sick and why they are unable to identify what is wrong to find a treatment. As we all know, delays in access to treatment have a detrimental effect in some cases, setting back patients' recovery. It is important we provide a national plan that outlines practical solutions, puts research at its heart, allows patients access and provides for them.

Speaking at a recent conference entitled Patients and Researchers: Partners for Life, the Minister of State, Deputy Áine Brady, recognised: "that for rare diseases, the plans and strategies have to integrate the various elements of care including diagnostics, treatments, rehabilitation, access to drugs and information and that this will involve a national plan to bring all these strands together". We need a cohesive plan and I urge the Minister of State to put one in place as a matter of urgency. Those living with rare diseases live with uncertainty and the unknown. We must provide them with as much help and as many practical outlets as possible to make their lives the best they can be.

According to the figures provided by IPPOSI, 3% to 5% of our population, approximately 140,000 people, are affected by rare diseases. Professor Eileen Treacy stated: "Inherited metabolic disorders are 2-3 times as prevalent in Ireland as they are in the UK". This statement underlines our need for a national plan for those living with rare diseases.

In June, the EU Council of Ministers recommended a concerted action across the EU by 2013. Given the conference speech given by the Minister of State, Deputy Áine Brady, it seems that the plan is only in preparation. In the Lower House last week, the Minister for Health and Children discussed the need for a plan and preparation. Many organisations and groups aiding those living with rare diseases in their day-to-day lives are in serious fear. We should be able to assist in the sourcing of treatment but we cannot in many cases because there is no national plan. I am asking the Minister of State, as the Government's representative, when the plan will be published and whether it will fully address the three hurdles identified by IPPOSI.

As with all aspects of life, we must provide hope, assistance and, most of all, knowledge. Every body of work and knowledge on rare diseases has, at its core, the demand for greater collaboration. We must identify and link centres of expertise in the field of rare conditions to assist people. I hope the Minister of State's response will contain a positive outcome and that a national plan for people with rare diseases will be delivered.

Deputy Michael Finneran

I will be taking this Adjournment debate on behalf of my colleague, the Minister for Health and Children, Deputy Harney. The European Union has been developing an action framework in the field of rare diseases. Ireland has supported the proposals in this area which concluded with a Council recommendation in June 2009. The key elements are that member states, including Ireland, develop plans and strategies for rare diseases and that this be completed by the end of 2013. In the context of a proposed rare disease plan for Ireland, this will include actions in a range of areas.

The national plan will need to consider the appropriate information systems for rare diseases. There is added value in adopting international definitions for rare diseases and to learn the lessons on the most appropriate registries that can be used to plan and manage services. The identification of centres of expertise for rare diseases will be a key priority under the plan. Allied with this is the organisation of suitable health care pathways so that patients and professionals know where the most appropriate care can be provided. In Ireland's case, it will be important that the identified centres and reference networks link in with their European counterparts on all these issues including health care professional training. The development of a research infrastructure with appropriate financial support is a further priority. This is an area where there is obviously added value in working with our European partners on programmes for rare diseases research.

In November 2009, two important documents were launched, the action plan for health research prepared by the health research group and the Health Research Board's strategic business plan. While each document has its own particular focus, taken together they represent a coherent and complementary approach to building a health research system that will see Ireland become a leading international centre for world class health research.

Research represents hope for many of the rare disease patients who are without cure today. In Ireland, many people are affected by a rare disorder, but many more individuals could unknowingly have a rare condition. Having a national rare disease plan would be an important step towards establishing a systematic and co-ordinated approach to dealing with some of the many challenges posed by rare conditions.

Access to orphan medicines is an issue which is key to effective clinical management. In Ireland the elements exist to have a transparent process to ensure equitable access to orphan drugs as well as to incentivise orphan drug development through our strong pharmaceutical and bio-tech industry in this country. Because of the small market, investment in medicinal products and devices for people with rare diseases is limited. As a result, the price for many of the treatments is such that it is impossible for them to meet conventional criteria for cost-effectiveness. The Minister for Health and Children, Deputy Mary Harney, has asked the HSE to explore a mechanism whereby a ring-fenced drugs budget could be put in place to cater specifically for people with other rare diseases.

The action plan for rare diseases adopted by the Council of European Union will provide the basis for our national plan. The Minister is encouraged by the recent joint conference on rare diseases organised by IPPOSI, GRDO and MRCG and that there is strong support from all stakeholders for this development. She would like to acknowledge this as well as the support from individual disease organisations and industry.

The Seanad adjourned at 10.45 p.m. until 10.30 a.m. on Wednesday, 10 March 2010.