TD calls for long overdue reform to access to rare disease treatments on Rare Disease Day
Fianna Fáil TD Pádraig O’Sullivan has called for long overdue reform to the system governing access to medicines for people living with rare diseases in Ireland, including conditions such as Friedreich’s Ataxia and Duchenne Muscular Dystrophy.
According to Rare Diseases Ireland, 1 in 17 people in Ireland lives with a rare disease, an estimated 300,000 people nationwide.
The Deputy’s call comes as 28th February marks International Rare Disease Day. He said he fears another year may pass without meaningful reform to a system that continues to block access to life saving treatments for so many patients.
Deputy O’Sullivan said he is deeply concerned that Ireland’s reimbursement process remains one of the slowest in Europe, leaving patients waiting years for medicines that are already approved and in use elsewhere in the EU.
Speaking, Deputy O’Sullivan said:
“Imagine needing a life changing treatment that already exists, but being told you will almost two and a half years to get it. This is not a worst case scenario, it is the current average in Ireland for accessing treatments for rare diseases.
“These medicines have passed EU wide safety checks and are already being used across Europe. In Germany, patients can access them in under 100 days.
“A child with a degenerative condition cannot pause the progression of their illness while the State negotiates. An adult with a rare cancer cannot wait indefinitely for a therapy that could extend or improve their life."
Deputy O’Sullivan has previously introduced a Bill to the Oireachtas aimed at reforming how orphan drugs are accessed, and the legislation is currently sitting at the third stage of the legislative process.
The TD highlighted several structural issues that have placed Ireland’s reimbursement process in this position. These include rigid cost effectiveness thresholds that do not reflect the realities of rare diseases, budgetary caution that treats high-cost therapies as a threat to the wider medicines budget and fragmented governance, where multiple bodies operate in sequence rather than in parallel.
Deputy O’Sullivan continued:
“I have spoken to families who have spent years fighting for medication that is already available elsewhere. I have heard from young adults trying to hold on to their independence while their condition slowly chips away at their strength. I have seen parents exhausted by a system that seems more focused on paperwork than on people.
“It is heartbreaking and it is entirely preventable. This system must be reformed and it cannot wait any longer.”
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Link to Deputy O'Sullivan's Bill: Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2021 – No. 122 of 2021 – Houses of the Oireachtas