Fresh hope for drug-plea girl
JUDGES RAISE CHANCES OF WALKING AGAIN
A LITTLE girl’s hopes of getting a drug which could change her life have risen after the Court of Appeal ruled that she should be reconsidered for it.
Ten-year-old Sophie Basma, known as Fifi, has a rare genetic neuromuscular disease and can no longer walk.
She has been denied Nusinersen – marketed as Spinraza – a drug which could, potentially, help her regain the ability to walk.
Fifi met six out of seven of the criteria required for her to qualify for the drug.
But in 2019 the National Institute for Health Care and Excellence (NICE) approved Spinraza for use on the
NHS in England with patients like Fifi required to walk at least five steps unaided prior to receiving treatment.
Two doctors from Manchester Royal Children’s Hospital and Great Ormond Street Hospital in London declined to refer Fifi for the drug. They decided this after she had been documented in a one-off assessment walking four steps unaided at the Manchester Hospital. There is no cure for Fifi’s condition, Type III Spinal Muscular Atrophy (SMA), which has left her with muscular weakness and progressive issues with her mobility.
Fifi’s family, from Wilmslow, appealed against the decision not to refer her for the drug but it was rejected.
Her mother, Sara, then instructed specialist public law and human rights lawyers at Irwin Mitchell to launch judicial review proceedings to challenge the decision.
A High Court hearing followed in October 2020, where the Judge upheld the decision not to provide the drug for Fifi.
As a result, Fifi’s legal team applied to the Court of Appeal in a bid to overturn this judgement. An appeal hearing took place on February 11 and this week the court ruled that the NHS Trusts must reconsider their decision taking into account all of the evidence.
In her judgement Lady Justice King said: “This seemingly simple matter was of critical importance to Sophie as her proven ability to walk those five steps determined whether she would qualify for treatment with a newly-approved drug, Nusineresen. Nusinersen is agree to be clini
This case is one example of the benefit of a judicial review for people such as Fifi
Katy Clarke, public law and human rights lawyer
cally appropriate for Sophie. Whilst it is unclear how effective it might be in the long term, there is good evidence of clinical effectiveness.
“It may have the potential to be life changing, in that it may help Sophie to regain her ability to walk and to maintain significant levels of upper body strength against, what is otherwise, an inevitable gradual decline to complete immobility. Nusinersen is very expensive but that is not the issue here.” She says it is ‘common ground’ that Sophie satisfies all but one of the criteria to qualify for it, namely the ‘controversial’ five steps criterion, ‘which relates to whether she was able to walk five steps unaided in the 12 months before she became entitled to be considered for the drug in October 2019.’
Katy Clarke, public law and human rights expert at Irwin Mitchell, said the judgement was ‘a huge step forward’
for Fifi and her family. She said: “This case is one example of the benefit of judicial review for people such as Fifi and her family whose lives have been impacted by decisions made by public bodies.
“It also highlights the importance of individuals being able to access legal aid to obtain advice about their rights and to enable them to bring judicial review proceedings if necessary to challenge decisions made.”