Catherine Byrne (Dublin South Central, Fine Gael) | Oireachtas source

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.

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