Joint North-South Rare Disease Conference
To celebrate International Rare Disease Day (28th February 2018), a Joint North-South Rare Disease Conference took place in Riddel Hall, Belfast on Monday 5th March 2018. The Conference offered participants the opportunity to be part of a global call on policy makers, researchers, companies and healthcare professionals to increasingly and more effectively involve patients in rare disease research.
The following is the opening speech delivered by Philip Watt, Chair of the Rare Disease Task Force; Chair of the Medical Research Charities Group and CEO of Cystic Fibrosis Ireland on the occasion of the North/South Rare Disease Conference, 5 March 2018 in Belfast.
Good morning everyone. Thanks to everyone who made it today, especially for those of you had to overcome an added level of adversity due to the combination of disability, health and weather.
I am very proud to be here today as a member of the Rare Disease Taskforce to be part of International Rare Disease Day.
Our key message today remains ‘If you or a loved one has a rare disease, you are not alone’.
We hope that today will in time be seen as an important step on the long and often desperate journey for equal rights for people with rare diseases in Ireland, North and South.
Today we are once again in Belfast as we were in 2017 in Dublin to renew both important friendships and joint working that has been feature of patient groups on the island of Ireland. I particularly would like to acknowledge our friendship with Fiona McLoughlin and the NIRDP. We also take this opportunity to congratulate Christine Collins who was recognised in the 2018 New Year’s honors list for her work on rare diseases in Northern Ireland over many years.
My name is Philip Watt and my presence here today is as Chairperson of the rare disease taskforce, where I represent the Medical Research Charities Group and Cystic Fibrosis Ireland where I work. The Task Force is also inclusive of Rare Disease Ireland and IPPOSI and the Irish Platform for Patients, Science and Industry
This taskforce, established 6 years ago, has brought together patient groups and key stakeholders from industry, academia and clinical services who have shared a vision of a better deal for people with rare disease and their families.
For too long many parents in Ireland North and South have had to cope with a sick child with a rare disease but with little information as to what the disease is, let alone where they might find treatment.
For too long GP’s have been frustrated by being unsure where the best center of expertise is to treat a particular rare disease.
For too long there has been an insufficient sharing of health resources North and South on our small Island. There had been some signs of change in cooperation in respect of children’s heart surgery, lung transplants and cataract operations for example.
In recent years there have also been several important rare disease health research partnerships North and South that were funded through EU programmes such as Horizon 20/20.
We urge that these beginnings of cooperation and joint research projects will not be up-scuttled or side-tracked by Brexit and the continuing lack of an administration in Northern Ireland. These are very worrying times for patient groups on both sides of the border.
I am delighted to see the support of the Northern Ireland Human Rights Commission for today’s conference. The participation of Chief Commissioner Les Allamby sends out an important message on both the importance of PPI- Public and Patient Involvement in health policy and the need to locate rare diseases more robustly within a human rights and equality framework.
In this context, we take this opportunity for the Minister for Health responsible for disability in the South, Finnian McGrath TD, to ensure that Ireland fulfils its oft repeated promise to ratify the UN Convention on the Rights of Disabilities that was signed by Ireland in 2007.
A key part if this convention (article 19) is concerned with the ambition for Independent Living for all adults living with a disability or chronic disease. Ireland remains the only county in Europe not to have ratified this vital Convention, which sends out poor message nationally and internationally.
In organizing today’s conference we once again draw attention and celebrate the fantastic work of rare disease patient groups in Ireland North and South.
There are some 7,000 rare diseases in Ireland
The fortunate few – perhaps around 30 or 40 - have a patient group to represent them.
Many patient groups are reliant on volunteers or one or 2 staff members at most. They are understaffed and overworked but they do an amazing job. In many cases they are run by the mothers or fathers of someone with a rare disease. Please show your appreciation for the work of these voluntary groups.
Today we also call on the Irish and British government to bring in urgently needed reforms to make drug therapies for those with a rare disease more accessible in Ireland North and South. For example, it is very unfair that the border can decide your access to a ground-breaking life enhancing drug. For example, the decision by the Irish government to approve the Cystic Fibrosis Drug Orkambi is in stark contrast to the British Government which continues to deny this vital drug to England, Scotland, Wales and Northern Ireland. Vice-versa the decision of Stormont to approve the drug Translarna for those with Muscular Dystrophy Duchenne is in stark contrast to the Irish Government which continues denies this drug to two patients.
Today we also take this opportunity to call on the British and Irish Governments for better support for health research and clinical genetic services that have such an impact on people living with a rare disease. Clinicians and researchers on the island of Ireland have been increasingly been involved in fantastic research focused on rare diseases – this work must continue to be fostered in spite of recent political challenges.
Today as we enter the final year of the of the operation of the existing rare disease plans north and south we acknowledge progress to date but we also call for the many remaining promises in these plans to be fully implemented and we call for second plans to be developed, including a focus on North/South and East/West sharing of the limited existing resources for those living with a rare disease.
Finally we call on both the Irish and the British Governments to tackle the often unstated or unintended carelessly though through mindset that ‘it is better to treat the many rather than the few’. For example - that it is better to give a million people a new drug at a lower price than provide funding for an expensive rare disease drug.
We say again today ‘We must continue to treat the many and the few equally’ and to do otherwise is a breach of human rights.
Chairperson Rare Disease Taskforce
Medical Research Charities Group (MRCG)
CEO Cystic Fibrosis Ireland.