James Browne (Wexford, Fianna Fail) | Oireachtas source

I commend Deputy Brassil on bringing forward this important Bill on behalf of Fianna Fáil. The Bill will help to reform the current HSE reimbursement process and provide a better situation for assessing orphan drugs.

Approximately one person in every 12 will develop a rare disease.

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Orphan drugs are currently assessed by the same mechanism as all pharmaceutical products where the odds are completely stacked against them because the numbers affected are so small, the medicines are usually very costly and the prices often exceeds the criteria set out by the National Centre for Pharmacoeconomics, NCPE, and the HSE. While these drugs are very costly to develop and the low patient population and low success rate in bringing the drugs to market can make it very difficult to assess based on clinical data, effectively these drugs are often set up to fail under the current system. These include Kuvan, Translarna, Respreeza, Orkambi, Spinraza and an ever-growing list of drugs. These families and their children want hope and a fair chance.

I will focus on Translarna for Duchenne muscular dystrophy which affects approximately 12 people in the country. The drug available for its treatment, Translarna, can only be used for children of a certain age and only five children fall into that category. Those families have been outside the gates of Leinster House and have presented in the audiovisual room several times. Muscular dystrophy is a debilitating genetic disease that will unfortunately lead to these children dying. Their muscles eventually waste away and they eventually lose the capacity to breathe and ultimately their heart gives out. Translarna is the first licensed therapy to address the underlying causes of this disease. It is available in 22 countries across Europe but not here. It works, it saves lives and there are no alternatives. Those distraught families and their sons and daughters are up against the clock. They know that if they do no use this drug by a particular age it is too late.

I know that many of the pharmaceutical companies effectively operate a system of blackmail, and pit countries against one another. Nevertheless, we can put better processes in place in this country to effectively provide a proper system so that these orphan drugs can be fairly assessed. That is what these families find most distressing. They see a very opaque system which has no transparency, there are no dates for meetings and there is no information about what is happening and how some drugs are, or are not, being approved. They need hope and they desperately need these drugs.

We do not have time to more than touch on the waiting list for genetics services to assess children and have them diagnosed. We are talking about genetic diseases, there is nothing that the children can do about it.

Even if we look at cancer drugs, some 20% of new cancer drugs launched internationally in 2015 and 2016 were made available in Ireland, compared with 76% in Germany, 71% in Austria and 67% in Denmark. Patients' groups are saying that Ireland is an outlier. I ask the Minister to support the Bill tabled by Deputy Brassil and Fianna Fáil. It is a very good Bill which sets up a way for a proper and fair reimbursement process to help give these families hope for their children.

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