A Wythenshawe dad of a terminally ill baby says time is running out for the NHS to provide a “miracle” drug which could massively improve the quality of his son’s life.
Eight-month-old Haris, from Newall Green, who has Spinal Muscular Atrophy was given three months to live when he was diagnosed with the condition in February.
Until February last year, the wonder drug Spinraza which can prolong and dramatically improve quality of life was available on the NHS, but was withdrawn on cost grounds.
Haris’ mum and dad Renata and Shakeel have been campaigning for the NHS restore funding for the drug, which is available in a number of countries including Scotland.
And in March the family and their supporters lobbied a meeting of the National Institute for Health and Care Excellence, the body which makes the recommendations for the funding of drugs on the NHS.
Following the meeting, NICE’s Chief Executive, Sir Andrew Dillon, wrote to Wythenshawe MP Mike Kane saying they were hopeful of positive outcome with the drug company Biogen, who manufacture the drug.
But nearly two months after their protest, there is still no decision on the funding of the drug which is deemed too expensive.
With Haris is now back home with his family in Newall Green, Shakeel says the NHS, NICE and Biogen all share responsibility for the failure to provide the drug with will have a massive impact on his son’s quality of life.
Funding for the drug will be considered again at meeting of NICE next week (Wednesday May 8) and NICE have released this statement:
Following the committee meeting on the 6 of March 2019, the appraisal committee asked NHS England and Biogen to enter into commercial discussions to see whether a managed access arrangement could be agreed for Spinraza consistent with NICE’s framework for determining cost effectiveness.
We have been informed by the parties that no agreement has yet been reached. Nevertheless, we consider it necessary to return to the appraisal committee next week in case an agreement is reached, or if no agreement is reached, for the committee to consider the consequences for guidance development.
This topic will be considered by the committee on Wednesday 8 May. Given the nature of the discussion, this will be held in a private.
Shakeel says the fight will continue. Boxer Amir Khan is supporting the campaign and Shakeel says he has received tremendous support from the local community and from the charity New Life which has funded a special cot costing £4,000 and a buggy costing £3170. Haris also has a Just Giving page to raise funds in the event that the final decision is to reject funding the drug and the family has to go abroad to get treatment.
Spinal muscular atrophy is caused by dysfunctional or missing gene that affects the production of a protein essential for motor neurons. This condition is manifested by a shrinking spinal cord.
One in 40 people are a carrier of the faulty gene and in the most severe cases, such as Haris, children rarely live beyond two years, making SMA the leading cause of death in babies and toddlers in the country.
But Spinraza has been known to have significant effects on the length and quality of life for patients.
Manfuacturer’s Biogen markets Spinraza at $750,000 dollars in the USA during the first year and $375,000 per subsequent year. The company offered the drug at £450,000 to the NHS and an undisclosed discount, but it still was not enough.
One report has said Biogen’s growth is heavily dependent upon SMA medication.
