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Government gives Grace thumbs-down

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TENDING TO GRACE Young Mark O’Malley at the bedside of his sister Grace after she underwent surgery recently. Grace has been turned down by the Government for a life-saving drug.

HSE says life-saving drug ‘not cost-effective’

Áine Ryan

AS the HSE quibbles with a pharmaceutical company about the cost of a life-saving drug, nine-year-old Grace O’Malley’s ability to write and draw, which she loves, ebbs away.
Spinal muscular atrophy (SMA) is continuing to attack her little body, and her distraught mother, Lorraine O’Malley, told The Mayo News last night that she now has to be assessed for assistive technology to help her with her favourite pastimes, as well as for a new seating system for her wheelchair.  
Poignantly, Grace sent a hand-written letter to Minister for Health, Simon Harris last October pleading her case for access to the drug Spinraza, which is available in 21 other European countries.

Devastated
Six months after the establishment of the campaign for the introduction of Spinraza, the Hollymount family has been left devastated. Last Friday, they received a letter stating that a report by the HSE Drugs Group had found that it was not cost-effective to purchase the drug. It would cost €600,000 per patient for the first year, and €380,000 thereafter annually. This would have an estimated budget impact in excess of €20 million over a five-year period, the HSE said.
It cited cost and proven clinical effectiveness as the two fundamental criteria used in its assessment of the drug.
The HSE has contacted Biogen, the pharmaceutical company that manufactures Spiranza, and it now awaits its input, within a 28-day stipulated period, on a possible revised cost.

‘False hope’
Meanwhile, Lorraine spoke to The Mayo News about the family’s distress. “We are back to the start and it just feels like Grace’s life is in the hands of a drug company who want money, the HSE who say they have no money, and a Government who give false hope and promises,” she said.
“When we started this campaign six months ago, Grace’s ability to write was not any worry to us, now six months on she has had to be assessed for assistive technology. The thoughts of Grace not being able to write her name – and she just loves arts and crafts, making cards and writing letters – is just so difficult to bear.
“Also, six months ago Grace had the strength in her neck muscles to keep her head held up, now she has had to have an assessment for a new seating system for her wheelchair to try to help aid her body and [keep her] head in a correct position. Of course, we worry what will be next to deteriorate and how will we ever sustain the deterioration without the possible help of Spinraza.”

Budget ‘almost exhausted’
NOTING that Taoiseach Leo Varadkar told the Dáil two weeks ago that the HSE had  ‘approved 29 new drugs last year’ and that he was sure they would ‘do the same again this year’, Lorraine said that a few days later it was announced in newspapers that the HSE has ‘almost exhausted’ its 2019 budget two months into the year.
“So the budget is gone, and they can’t make Spinraza happen, yet we also see a reported multi-million-euro overrun on the restoration of Leinster House and the astronomical overspend on the new children’s hospital, which leaves us in despair. Again we must wait to see if the drug company Biogen will send in a reasonable offer that the HSE will accept,” Lorraine continued.
“In November, we presented the patient impact of SMA to the Rare Disease Technology Review Committee, outlining the disease and its real-life impacts and what we would wish for if we got Spinraza. We know that this group of experts recommended that the HSE make Spinraza available on a Managed Access Programme,” she said, explaining that such a programme would mean that if Spinraza was shown not to be working for a child after the sixth injection, that child would be taken off the drug, ensuring that money would not be wasted unnecessarily.

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Six months after the families affected by SMA demonstrated outside Dáil Éireann they will gather on Thursday next, February 28, World Rare Disease Day, for a Spinraza NOW Day of Action. The gathering will take place on Dublin’s Kildare Street, between 11am and 2pm. The families say this demonstration will ‘remind the Government that people with SMA need to have the only treatment that exists for their condition. People with SMA will not be easily dismissed’.

MPU Mayo