Newcastle North MP, Catherine McKinnell, met yesterday afternoon with the Secretary of State for Health and Social Care, Matt Hancock, to press him to ensure that an innovative treatment for CLN2 Batten Disease can be made available to patients on the NHS.

She was joined at the meeting by her Throckley constituents, the Rich family, as two of their three children – Nicole and Jessica, aged 7 and 3 – have been diagnosed with this rare, genetic condition and are currently receiving Brineura, an enzyme replacement therapy, at Great Ormond Street Hospital in London.

Licensed by the European Medicines Agency in 2017, children receive the treatment by an infusion into the brain over four hours once a fortnight. However, clinical trials and the compassionate use programme currently enabling the therapy to take place in England do not provide security of access to what is the first and only treatment for any form of Batten Disease. The clinical trial is due to end next year, whilst the compassionate use programme could be withdrawn at any time.

Catherine secured yesterday’s meeting with the Health Secretary when she raised concerns at Health Questions in the Commons about the provisional decision by NICE – the National Institute for Health and Care Excellence – not to recommend making Brineura available on the NHS, after a decision-making process that had lasted over a year. The Newcastle North MP has previously extensively lobbied Ministers, NICE and NHS England on this issue since she first met with Nicole and Jessica’s mum, Gail Rich, almost two years ago.

The Rich family – including dad Matthew – met up with Catherine in Parliament ahead of the discussion with the Secretary of State, to reiterate just how beneficial Brineura has been for Nicole and Jessica, and the importance of making the treatment available on the NHS to the very small number of children affected by what is a debilitating, rapidly-acting condition. They were joined by other affected families, and their MPs.

Catherine and the Rich family used the meeting with the Health Secretary to urge him to convene an emergency meeting with NICE, NHS England and BioMarin – the manufacturer of the treatment – to find a way of overcoming the barriers to making Brineura available on the NHS, and to look at whether an exception should be made in this case given the very small number of children affected by CLN2 Batten Disease who want to access the treatment on the health service.

Speaking after the meeting, Catherine said:

‘I am extremely grateful to the Health Secretary for agreeing to yesterday’s meeting – and for the time he took to carefully listen to Gail and Matthew about the incredible impact that Brineura has had on Nicole and Jessica and their quality of life. I know it meant a great deal to the family to be able to explain this to him directly, and to emphasise how incredibly important it is that a resolution to this situation is found as soon as possible. 

‘I left our discussion feeling really positive about the Health Secretary’s determination to ensure we get the right outcome for the Nicole and Jessica, and other affected families – and being able to meet with them, and see for himself the lifesaving difference that Brineura has made, will clearly have helped with that.’

Gail Rich said:

‘We could not have asked for a better end to the day than for the Secretary of State to meet our daughters. Nicole and Jessica are living proof that this treatment works and he got to see that for himself. 

‘We have more hope than ever before, and we have every faith we will get the positive outcome we and all affected families are desperate for.  

‘It was a huge step forward for our family and for the Batten Disease community, and we cannot thank Catherine enough for getting us to this point.’