Mary Butler (Waterford, Fianna Fail) | Oireachtas source

This Bill is a response to the medicines crisis faced by Irish patients suffering with rare diseases and I compliment Deputy Brassil on his work on it. The Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2018 seeks to reform the HSE's current reimbursement process by establishing for the first time a unique process for assessing an orphan drug for reimbursement in Ireland. The Bill brings a long overdue and credible solution to the ongoing crisis surrounding access to medicine for patients with rare diseases in Ireland. Orphan drugs treat, diagnose or prevent life-threatening or chronically-debilitating diseases which affect fewer than five people in every 10,000. They are currently assessed under the same guidelines and procedures as standard drugs despite the clear differences between the two. Rare, high-tech drugs will naturally treat an extremely low patient population. To measure their effectiveness using the same criteria as common conditions will naturally cause difficulty. Other EU countries have adapted the process for this reason and we believe it is time we did likewise.

In recent years, I have become aware of two orphan drugs. The first such drug is Respreeza which I first learned of at a presentation in the audiovisual room where I met a gentleman, Johnny Hannon, who was on a trial through a compassionate access programme. Respreeza is used for maintenance treatment to slow the progress of emphysema in adults. The drug has had a game changing effect on his life. However, that day I met another lady in the room who did not have access to the drug on the compassionate access programme. Every breath she took was a struggle. She told me how basic things such as getting up, washing and going into town to do some shopping was a struggle. It is wrong that people have to take to the streets to fight for access to a drug that can change their life. It is not acceptable that citizens suffering from severe illnesses should be forced to do that or to go public in the media to make their voices heard.

Orkambi is another drug that people are familiar with. Jillian McNulty who had benefitted from the drug which, as she said, transformed her life ran a high profile campaign. An estimated 60% of the cystic fibrosis population in Ireland have begun Orkambi treatment, which is very welcome. Gerry McElvaney, professor of medicine at the RCSI said, "In general the results from Orkambi were better than I expected and I think it has given significant relief to a significant number of patients". He has also said that we have a duty and obligation to ensure that patients with rare diseases receive the same treatment and care as any other patient in Ireland.

Access to drugs such as these can result in less time spent in hospital, fewer acute beds being used and can allow patients to spend more quality time at home which means cost savings to the State. This Bill is a timely and logical step and I hope the Minister will support it and work with Deputy Brassil for it to be enacted as soon as possible. I cannot imagine what it is like for a parent of a child with a rare disease. All one would want to do would be to give the child every chance at life. If there is a drug that would give children such as these a fighting chance then the onus is on us as legislators to ensure those children are given every chance.

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