In March 2018, the Report on Evaluating Orphan Drugs published by the Joint Committee on Health, was received by my Department. The Committee’s report put forward a number of recommendations which were carefully considered by my Department, the Health Service Executive and the National Centre for Pharmacoeconomics.
I am satisfied that the Oireachtas has put in place a robust legal framework in the Health (Pricing and Supply of Medical Goods) Act 2013, which gives full statutory powers to the HSE to assess and make decisions on the reimbursement of all medicines, taking account of a range of objective factors and expert opinion as appropriate. This assessment process has delivered results for the State and Irish patients. In 2019, the HSE has approved 26 new medicines and 5 new uses of existing medicines, 17 of these medicines are for the treatment of rare diseases.
Since the publication of this Report, I am pleased to report that there has been substantial engagement between my Department, the HSE and the NCPE on each of the recommendations, which has resulted in a number of key changes in the HSE assessment process of medicines within the confines of the 2013 Health Act. These have included changes to the HSE Drugs Group, whose membership has now been expanded to include two representatives from the National Patients Forum and more clinical expertise in the area of rare diseases.
In June 2018, the HSE Leadership appointed a Technology Review Committee for Rare Diseases which is responsible for:
1) reviewing proposals received from industry or expert groups in Ireland for funding of new products for rare diseases including orphan drugs, or expanded indications for existing products for rare diseases and making recommendations as to the implementation of the relevant recommendations from the National Rare Diseases Plan 2011-2018; and
2) providing contributions to the development of clinical guidelines for relevant Orphan Medicinal Products (OMPs) and supporting the implementation of guidelines in conjunction with the National Drugs Management Programme Office where applicable. The Committee’s recommendations for reimbursement of OMPs are not intended to replace any part of the existing medicines appraisal or reimbursement process but rather complement it.
The Committee also recommended the appointment of an independent academic with knowledge of pricing and reimbursement systems and orphan medicines, to conduct a review of the current process and its role in orphan drug availability in Ireland.
My Department has recently completed a tender process for a review of the HSE reimbursement and pricing decision-making process. Mazars has been appointed as the consultancy firm to complete this work. The review will make recommendations, as considered appropriate, in respect of any required improvements to the HSE's systems, structures, processes, governance arrangements or use of specialist resources in respect of the review.
The Committee in its report conveys it support for collaborating with other EU member states. A significant development since the publication of the Committee’s report was the joining of the Beneluxa Initiative on Pharmaceutical Policy in June 2018. This collaboration is supporting my objective of co-operating with other European countries to identify workable solutions, in an increasingly challenging environment, to secure timely access for patients to new medicines in an affordable and sustainable way. The patient is at the centre of this collaborative international approach. This is a long-term strategy but a strategy that we expect will complement existing domestic policy in this area.