Bobby Aylward (Carlow-Kilkenny, Fianna Fail) | Oireachtas source

I thank the Leas-Cheann Comhairle for selecting this matter for discussion. The drug Vimizim is produced by BioMarin in its manufacturing operations in Shanbally, Co. Cork. It has been granted market authorisation in many countries worldwide for treatment of Morquio A syndrome. This is a rare, degenerative, potentially life threatening genetic condition that affects major organ systems in the body. It can substantially limit the quality of life of those affected and patients rarely live beyond the second or third decade of life.

This drug, Vimizim, is the first and only enzyme replacement therapy designed to target the underlying cause of this disease, which currently affects only ten known patients in Ireland. As my colleague has said, one of these patients is an 11 year-old girl from my constituency, Carlow-Kilkenny, who has battled bravely to lead a robust and fun filled life despite this challenging medical condition. This little girl’s family were delighted when their daughter was one of two Irish children included in the trials for Vimizim which began in September 2011. I want to read a previous statement from her mother, who is in the Gallery, into the record:

This is a progressive and damaging condition. I don't know how she will be without the medicine. It gives her stamina and energy and it is our only chance for her.

Unfortunately this brave young girl could lose her access to this drug very shortly. The voluntary drug supply that has been given to her is being withdrawn next Friday. From next Friday, this child will not have this drug that has given her the quality of life of the last couple of years.

The HSE drugs group has denied a number of applications for reimbursement. It has denied it twice so far. BioMarin submitted an application for reimbursement to the HSE in 2015. On 15 June 2017 it received a letter from the HSE confirming the decision not to reimburse the drug Vimizim in Ireland. The decision not to reimburse the drug on the grounds of insufficient clinical data and lack of value for money is surprising, given the nature of rare disease medicines and the acceptance of these in other countries. BioMarin met with the HSE again in July 2017 and resubmitted additional evidence and a new commercial offering for consideration at the HSE drugs group meeting on 18 September 2017. On 13 November 2017 while attending the HSE on another matter, BioMarin was informed that the HSE would not reimburse Vimizim for Irish patients. That was the second time.

The negative feedback from the HSE for a second time was particularly unfortunate, given that the drug is currently available to children and adults with Morquio A syndrome in many countries following an assessment of the very same data. The drug is currently commercially available to patients in the United Kingdom, our next door neighbours, Northern Ireland which is joined to us, Germany, Spain, France, Italy, the Czech Republic, Hungary, countries in the Middle East, the Russian Federation, Brazil, Canada, and the United States.

BioMarin requested a meeting with the HSE again in December 2017 and agreed to submit a new application, which was due to be discussed at the HSE drugs group meeting on 18 January 2018, just a couple of weeks ago. However, since this date they have had no communication from the drugs group, which leaves both patients and the company in the dark as to whether the drug will be reimbursed or not. We understand from parliamentary questions, of which I submitted many, however, that the drug was discussed at this meeting. I know very well the Minister of State will state that responsibility for decisions on reimbursement is solely that of the HSE drugs group and he is bound by legislation not to intervene. Surely the Minister can contact the HSE drugs group directly on behalf of the young people who are here today, their families and the other eight known Irish people with this condition and demand that they urgently report on their most recent meeting of 18 January.

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