NICE Asks for Comments on its Appraisal Consultation Document (ACD) for Risdiplam

02 June 2021

(Page updated 3rd June)

NICE’s initial ‘no’ to approving risdiplam for NHS funding is not unexpected. Their ACD outlining the reasons for this is here.

They are now open to comments from anyone up until 5pm on 23rd June on the following:

  • Has all of the relevant evidence been taken into account?
  • Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?
  • Are the provisional recommendations sound and a suitable basis for guidance to the NHS?

We can expect Roche to be working hard on their response and for discussions and negotiations on the treatment cost and the cost effectiveness to be ongoing with NHS England. Patient groups and clinicians will also be preparing their comments on the questions raised in the consultation and urging the company and NHS England to rapidly reach a position whereby NICE’s response can be changed to a ‘yes’ to NHS funding of risdiplam for all who have SMA Type 1, 2 or 3.

All responses must be submitted using this link.

The next committee meeting will be on 13th July.

What happens next?

NICE’s website no longer shows that it expects to publish its final recommendation on 21st July 2021. We will need to wait for the outcome of the 13th July committee meeting to know what will happen next. There could be further committee meetings.

The final recommendation can be a ‘No’ or a ‘Yes’ - some medicines don’t get recommended at all for anybody. For some, like nusinersen, there is a Managed Access Agreement (MAA). This recommends the treatment is funded for a number of years during which time further evidence about uncertainties is collected. Towards the end of an MAA this evidence is reviewed by NICE which then makes a further recommendation whether there should be ongoing NHS funding for the treatment. 

If NICE makes a positive recommendation, when can we expect risdiplam to be delivered by Centres?

We recognise that some people who could now access nusinersen are hesitating about treatment decisions and are waiting for the outcome of NICE’s risdiplam decision. If the decision is positive, because there has been an Early Access to Medicine Scheme (EAMS), the NHS has to provide the treatment within 30 days. If there hadn’t been an EAMS, treatment would have had to be provided within 90 days.

Some Centres that have offered the Early Access to Medicine Scheme would be in a good position to get going quickly, others who have not may take more time. With risdiplam, there wouldn’t be the same pressure on hospital resources as there has been with nusinersen – for example, establishing theatre space and skilled staffing to deliver the lumbar puncture procedure. However, people would still need to meet with their clinical team to discuss the risks and benefits of this treatment compared to any other current treatment they may be on, so not everyone could be started on treatment straight away.

Clinicians emphasise that the earlier the treatment is started the better.

On 10th May, NHS England issued guidelines and outlined which groups should take priority for nusinersen treatment. You can read this here.

The risdiplam EAMS is now only open to new people who were identified before 20th May and who have been assessed and for whom all paperwork has been completed and submitted for the EAMS by 1st July 2021. See the notice about this here.

If you are interested in treatment, we suggest you contact your clinical team to discuss your position as soon as possible.

You can read more about the NICE technology appraisal process on their website, here.