Dáil debates

Thursday, 20 June 2019

Saincheisteanna Tráthúla - Topical Issue Debate

Medicinal Products Availability

4:20 pm

Photo of Caoimhghín Ó CaoláinCaoimhghín Ó Caoláin (Cavan-Monaghan, Sinn Fein)
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Gabhaim buíochas leis an gCeann Comhairle.

The date 11 June 2019 was and will always be a red letter day in the lives of those children and their families who will now finally get the chance to have new life experiences and prospects, thanks to the HSE leadership team's decision to approve the drug Spinraza. It could and should have been a red letter day in the lives of all SMA sufferers, but, sadly, approval only extended to children up to the age of 18 years. We must all be conscious of the adult SMA population in Ireland who have been left behind by this decision and are feeling very disappointed and isolated. What will happen when children turn 18 years of age?

I will read extracts from two emails I received in the wake of the decision. One is from an adult Irish woman, a mother and a wife, who has SMA, while the other is from an adult American male who also has SMA but who has access to Spinraza. Both are in their 50s. The adult Irish female states in her correspondence what it has been like living with spinal muscular atrophy since she was a teenager. She recently celebrated her 50th birthday. I quote:

Last year our SMA Ireland family was formed. It was and is wonderful to be part of a support network because some of us adults have been battling this disease for decades on our own, apart from family, physiotherapists, and occupational therapists. I was thrilled on Tuesday last, 11 June, that at last Spinraza was approved for children but devastated for myself, my husband, my daughter, my family, and for the other SMA adults living in Ireland.

Addressing the Minister for Health, Deputy Harris, she stated:

You also have a daughter, Minister Harris. You know how much love a parent has for their child. I had to greet my daughter at the school gate on that Tuesday with the devastating news that her mother will not get Spinraza. I tried to explain the inexplicable. She cried as I have done. Time is not on my side.

Yours in anticipation.

I turn to the adult American male. I will only quote extracts from his email:

Denying adults access to this drug is heartless and inhumane. I am living proof that Spinraza does work for adults suffering with SMA. I am an American male, 54 years of age, who was diagnosed with SMA in my early 20s. This was a terrifying time in my life. After diagnosis I spent the next 20 years watching this disease slowly rob me of my ability to play with my children, my hobbies, my ability to perform even basic home maintenance, and eventually my ability to work and provide for my family. I started treatment with Spinraza back in October of 2017. Since starting treatment, my disease progression not only stopped but I began to see improvements in my walking gait, my overall strength and my endurance. Before treatment I needed a walker just to stand up from a lift chair. That walker is no longer needed. Before treatment I needed assistance in order to lift my leg into a vehicle. This is no longer needed. These achievements may seem minuscule but for someone who is facing the inevitable total loss of independence, Spinraza is offering a new chance of life. Please make Spinraza available for all because every life matters.

I close with the request that the Minister of State respond positively to the appeal of the SMA community and that she, please, ensure approval for the reimbursement of Spinraza for all SMA sufferers.

Photo of Catherine ByrneCatherine Byrne (Dublin South Central, Fine Gael)
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I thank the Deputy for raising this issue which he has raised on a number of occasions. We were all delighted when it was announced in June that Spinraza would be made available to children. Spinraza is indicated for the treatment of 5q spinal muscular atrophy, SMA, which causes progressive muscle atrophy and weakness. It is a genetic condition that affects the nerves in the spinal cord. It causes problems with movement, muscle weakness and difficulties in breathing and swallowing. It is estimated that there are approximately 25 children living with SMA in Ireland. As Deputies are aware, the Oireachtas put in place a robust legal framework in the Health (Pricing and Supply of Medical Goods) Act 2013 which gives full statutory powers to the HSE to assess and make decisions on the reimbursement of medicines, taking account of a range of objective factors and expert opinions, as appropriate. They include the clinical and cost effectiveness of the product, the opportunity cost, the potential or actual budget impact and the impact on resources available to the HSE.

I am informed that on 11 June the HSE leadership team approved access to the drug Spinraza for children with SMA types I, II and III on an exceptional and individualised basis. The HSE’s decision is in respect of children with genetically confirmed SMA type I, II or III, in accordance with the controlled access criteria recommended by the Technology Review Committee for Rare Diseases. That committee’s recommendations on access criteria were clearly targeted at the youngest and most severely affected SMA patients. This group is the clear priority for the HSE.

The actual patient assessment and approval process will be the means used in determining access on an individual case by case basis. Such a managed access programme will operate within the remit of the HSE medicines management programme. The HSE's decision process for Spinraza involved a health technology assessment, followed by detailed consideration by the HSE expert groups on new drug therapies, including the Technology Review Committee for Rare Diseases and the drugs committee. Evidence of the clinical effectiveness of the new drug therapy was also reviewed.

We acknowledge that the protracted deliberations on the reimbursement of Spinraza have caused great stress and anxiety for the families of patients. However, the HSE has finite resources and a responsibility to ensure they are deployed to the greatest effect. The Minister for Health, Deputy Harris, is pleased that, through commercial negotiations, the HSE and the manufacturer managed to reach an agreement that will benefit the most vulnerable children.

I do not have much more to add on the specific question the Deputy asked. While we are all delighted that Spinraza has been approved for children, the Deputy asked that it also be made available to adults. I do not have a reply to that question and if I come back into the Chamber again, I do not believe I will have anything further to add. I cannot give a commitment on behalf of the Minister that Spinraza will be supplied to adults any time soon.

Photo of Caoimhghín Ó CaoláinCaoimhghín Ó Caoláin (Cavan-Monaghan, Sinn Fein)
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On behalf of Members, I ask the Ceann Comhairle to reflect on the unsuitability and inadequacy of having the Minister of State respond. She is, as she rightly acknowledged, not in a position to expand on the current position. The Minister for Health should be here. He has the same duty to be held as accountable to the House as the Ministers, Deputies Flanagan and Bruton. He certainly never appears in the House whenever I table a Topical Issue matter. The reply the Minister of State has read does not address the serious deficiency in the decision made. It should have included all SMA sufferers. Not everyone who will have the opportunity to access Nusinersen, or Spinraza, its branded name, will respond positively to it, as in the example I gave of the adult American male. One hopes they all will - certainly the 25 children who will now have access to it - show spectacular improvement in their physical condition and life prospects. There is, however, a cohort of adults.

Their number is uncertain but is certainly no more than 50, as has been speculated on time and again. These are people who have survived with SMA from childhood. In many of their cases, the symptoms presented in later life rather than in early years. They are equally entitled to access to Spinraza. The families who are now pleased and who have every right to celebrate because their children have hope are equally vehement in supporting the case for access to Spinraza for all, including the adult cohort. When the Minister of State says the HSE has prioritised this group, this is welcome but it has to include all SMA sufferers. I am not going to labour my point. The voice and intervention of the Minister of State could also be helpful. I ask her to stand with us in this appeal to the Minister, Deputy Harris, to the Government and to the HSE to continue the engagement with Biogen and as speedily as possible to conclude an arrangement for access to Spinraza for all sufferers from SMA across this land.

4:30 pm

Photo of Catherine ByrneCatherine Byrne (Dublin South Central, Fine Gael)
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We would all hope into the future that those who suffer from SMA will be able to treated, as everyone who is sick should be. I am not in a position to give Deputy Ó Caoláin an answer on this today. However, I will commit to speaking to the Minister, Deputy Harris, and to trying to influence those who need to be influenced to the effect that all people who have SMA should be treated equally. I am sorry that I do not have the direct decision the Deputy would like but I will bring it back to the Minister, Deputy Harris.

Photo of Caoimhghín Ó CaoláinCaoimhghín Ó Caoláin (Cavan-Monaghan, Sinn Fein)
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I thank the Minister of State.