NHS body urged to approve wonder drug ‘before it’s too late’ at vigil for Forest Gate baby

Maryam with her father Zainab. Pic: Rachael Burford (Image: Archant)

Members of six-month-old Maryam Malji’s family and supporters including Imam Yunus Dudhwala and West Ham MP Lyn Brown held a vigil outside the London office of the National Institute for Health and Care Excellence (NICE).

They urged NICE to approve the drug Spinraza before it is too late.

Maryam’s uncle, Irfan Malji, made a heart felt plea: “You can imagine how Maryam’s parents will feel if they provide this medication. Hopefully the decision is in our favour.”

Asked about Maryam, he said: “She is deteriorating slowly. The longer this goes on the weaker she will be.”

NICE’s appraisal committee met in Manchester on Wednesday to discuss whether or not to grant approval.

But it could be another five weeks before a decision is made public.

Lyn Brown said: “It is completely and utterly outrageous. Maryam and many other babies need that drug now.

“They are playing with babies’ lives and should be ashamed of themselves.”

About 30 people stood behind a banner bearing Maryam’s picture and the words ‘NICE - Do the right thing!’ before prayers led by Imam Dudhwala and the Rt Rev Peter Hill, the Bishop of Barking.

Maryam has the rare genetic condition, type 1 spinal muscular atrophy (SMA).

Medics told her parents Zainab and Shakil she could have just months to live without Biogen’s drug.

Maryam’s brother, Abdullah, also had SMA1 and was one of the first children to receive Spinraza when he was placed on a medical trial.

He died aged one in December 2015, from an infection, but the trial was successful and led to the drug being government-funded across Europe.

Despite it being free in Scotland and 25 other European countries, NICE had said it was not recommending it due to its price.

Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said: “When the committee discussed Spinraza prior to agreeing the previous draft recommendations last August, it noted that the evidence showed a substantial benefit for the treatment, but felt there were significant uncertainties, particularly around its long-term benefits.

“Since then we and NHS England have been discussing with Biogen how they might address the uncertainties identified by the committee, while demonstrating the potential for Spinraza to be considered cost effective and managing the risk to the NHS of allowing access to this treatment.

“It has taken us longer than we would have liked to get to the position we’re in today where our independent committee is able to discuss a revised submission from Biogen, alongside the feedback we received during public consultation on our draft recommendations.

“We recognise that this continuing uncertainty has been concerning for the community but we felt it was important to give all parties the opportunity to get to a point where we could have a meaningful discussion about Spinraza and what it would take to make it available to patients in England.

“That meaningful discussion is now taking place and will conclude today. We will do our best to ensure that the committee’s decision is made available as quickly as possible.”

A Biogen spokesman said: “Like families and patient groups, we await the outcome of the committee meeting.

“NICE and NHS England previously agreed that flexibility and collaboration was important in the case of Spinraza, and we are hopeful that this will be honoured when the final decision is published.

“Biogen understands that patients in England, Wales and Northern Ireland, and their families, desperately need equality in access with 25 European countries (including Scotland).

“We are committed to continuing to play our part in helping the NHS make Spinraza available to as many patients as possible, as quickly as possible.”