The Department published a progress report on the National Rare Disease Plan in February 2017 and it is available on the Department’s website at:
http://health.gov.ie/blog/publications/interim-report-on-national-rare-disease-plan-for-ireland-2014-2018/.
It provides an update on all 48 recommendations as outlined in the Plan and progress to date.
One of the principal recommendations in the Plan was the establishment of a National Clinical Programme for Rare Diseases which is responsible for assisting with mapping and developing care pathways for rare diseases; facilitating timely access to centres of expertise nationally and internationally; and developing care pathways with European Reference Centres for those ultra-rare disorders where there may not be sufficient expertise in Ireland.
The establishment of a National Rare Disease Office (NRDO) featured prominently in the recommendations of the Rare Disease plan. The office provides current and reliable information about rare diseases to the general public, health care professionals, researchers and policy makers. The National Rare Diseases Office is responsible for updating Orphanet Ireland. Orphanet is the international rare disease reference and information portal funded by the EU. The office also manages the National Rare Diseases Information Line (a Freephone service) and provides online information about rare diseases on http://www.rarediseases.ie/.
A number of recommendations about access to appropriate drugs and technologies were contained in the plan. One chief recommendation in this regard refers to the HSE developing a Working Group to bring forward appropriate decision criteria for the reimbursement of orphan medicines and technologies; and that the approach should include an assessment system similar to that for cancer therapies established under the National Cancer Control Programme. The HSE Acute Hospitals Division has developed the terms of reference, required membership and reporting relationship for this committee.
As such, a Committee called the “Rare Diseases Medicinal Products/Technology Review Committee” was established in June 2017 by the National Centre for Pharmacoeconomics (NCPE). The responsibilities include the review of proposals for funding of new products for rare diseases or expanded indications for existing products for rare diseases. The Committee will also contribute to the development of clinical guidelines for relevant Orphan Medicinal Products and support the implementation of same.
Dr. Michael Barry is the Chair of the Committee since September 2017 and it is expected that Committee will begin its work in early 2018 after appointment of the necessary support staff.
The National Rare Disease Plan continues to be implemented. It recommended that an Oversight Implementation Group of relevant stakeholders, including patients' groups, be established to oversee and monitor implementation of the plan’s recommendations. This group was established by the Department of Health in 2015 and it has met on a number of occasions.
The Department will consider the matter of a further plan as part of its Work Programme for 2018.
