A meeting has recently taken place where SMA Support UK and Muscular Dystrophy UK, representing SMA charities, met with the Clinical Director of Specialised Commissioning at NHS England, James Palmer, for urgent talks on the Expanded Access Programme for Spinraza to treat infants with SMA Type 1. They were also joined by representatives from the drug company, Biogen and Great Ormond Street Hospital clinicians.
What happened at the meeting?
James Palmer advised that although he understood the urgency for families, NHS England is not yet able to confirm any support for the EAP. This is because of a rule which requires companies to have their trial results published in a peer-review journal before they qualify for NHS England support. As soon as this happens, it will review the possibility again.
This rule is totally at odds with the severly life-limiting nature of SMA Type 1 and the urgency facing babies and their families. This requirement is not even insisted on by major regulators such as the European Medicines Agency and the Food and Drug Administration in the United States (both of whom have now approved Spinraza), nor for decision makers at NICE.
Additionally, NHS England has said they have no power to speed-up the NICE process in any way. NHS England suggested that Biogen considers making a decision to include treatment costs as part of their commitment to the EAP.
What you can do
You can write to the Chief Executive of NHS England, Simon Stevens, urging fast action on Spinraza, or to your member of the Scottish Parliament in Scotland, your assembly member in Wales or for Northern Ireland, to the Assembly.
You can read the full notes of the meeting, which took place on 25th April.
The EAP is a separate programme, funded by Biogen for eligible infants affected by SMA Type 1.
Now that the Committee for Medicinal Products for Human Use (CHMP – the body that conducts the scientific review) has made a positive recommendation to the EMA to grant a marketing authorisation for nusinersen, we are awaiting the next steps towards a decision if the drug can be centrally funded by the NHS here for SMA Types 1, 2 and 3.
The European Commission must first ratify the EMA’s decision. This is expected to happen within 60 days of the CHMP opinion.
In the coming weeks, NICE is expected to announce whether they will be evaluating the drug for clinical and cost effectiveness. In order to do this, they must have the topic referred to them by a Health Minister. SMA Support UK, MDUK and The SMA Trust are calling for this to happen in the shortest timeframe possible.
NICE’s rulings apply to England. Northern Ireland is officially linked to the NICE process, and Wales will often follow NICE guidance.
The Scottish Medicines Consortium takes decisions on new medicines in Scotland. It is expected that this process will run at the same time as a NICE evaluation.