Rare Disease Day highlights need to reform reimbursement system for new medicines

Fine Gael Senator and Member of the Oireachtas Health Committee, Seán Kyne, has said the process for the reimbursement of medicines for rare diseases needs to be reformed given the lengthy delays in making new medicines available here.

Senator Kyne was speaking in advance of Rare Disease Day, which takes place tomorrow, and aims to raise awareness of such illnesses and their impact on patients and their families.

Senator Kyne said: “The differences in the length of time that it takes to reimburse the cost of new medicines for rare diseases are worrying.

“In Ireland for example, it takes on average just over three years for the successful reimbursement of new medicines for rare diseases that are designated by the EU as ‘orphan medicinal products’. In contrast however it takes on average less than four months in Germany.”

Senator Kyne continued: “There are approximately between 6,000 and 8,000 rare diseases globally with anywhere between six and eight per cent of the population affected at some point in their lives. This means that between 280,000 and 320,000 people in Ireland will be affected by a rare disease at some point. However, only about 5% of rare diseases have a licenced treatment option.

“A Technology Review Committee for Rare Diseases, as recommended by the National Rare Disease Plan for Ireland, was appointed in late 2018 with responsibility for reviewing proposals received from industry or expert groups in Ireland for funding of new products for rare diseases.

“The Committee meets in response to requests for assessment of new products for rare diseases or expanded indications for existing products for rare diseases.

“However, it is my opinion that the current system for the reimbursement of new medicines in Ireland stacks the odds against the reimbursement of new medicines for rare diseases.

“The system is heavily focused on examining quantitative data such as large clinical trials. Such a focus makes it more difficult for medicines to treat rare diseases because by their very nature each disease only impacts a small number of people.

“In addition, the threshold in Ireland for measuring the cost effectiveness of a new medicine, in the context of its ability to improve the quality of life or extend life, is set at €45,000. This threshold can seldom be achieved by medicines designed to treat rare diseases.”

Senator Kyne concluded: “We need to examine the reimbursement system in countries like Germany where new medicines, and especially medicines to treat rare diseases, are reimbursed much faster and are available to patients much sooner.

“It is also incumbent on pharma companies to work with Government to set a fair and reasonable price for their potentially life-saving products.

“The HSE Service Plan states that approximately €2.4bn is paid by the HSE each year to drug companies. This equates to 12% or nearly one in every eight euro invested in running our health and social care services.

“The speedy development of vaccines for Covid-19, which has taken place without sacrificing rigorous research into safety and effectiveness, shows that is possible to test new medicines and provide a decision on reimbursement much sooner.

“In the development of its work plan for this year, I have requested that the Oireachtas Health Committee examine this issue to help bring about the reform that’s needed.”