Access to life changing treatment

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Our fight for Vimizim

NICE leans towards a no for the treatment of Morquio A (MPS IVA) - 12 November 2021

NICE have publicly released their interim decision not to recommend elosulfase alfa (Vimizim®), for the treatment of individuals with MPS IVA. The full decision and committee papers can be found here. Read the full update from MPS Society here.

What next?

We are doing all we can to engage with the NICE process and work with all those involved to ensure that our patient community is treated fairly. If you want to help, you can:

  • respond to the consultation before 3 December 2021

  • write to your MP to raise your concerns

  • read our frequently asked questions about the process 

 

Useful links

 

For parents

For sibling support

About MPS IV Morquio

Helpline

 

Our helpline is open 9-5pm

Monday-Friday: 0345 389 9901

Out of hours support line open

5pm-10pm Monday-Friday and weekends: 07712 653 258

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How we got here

The fight to make Vimizim available was a massive undertaking for the MPS Society and all the families that fought with us. Download the timeline to find out about the journey we took. 

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When will guidance be available and how can we respond?


Guidance and templates are available on our website now. The deadline for responses is the 3 December 2021. These need to be submitted via the NICE website (you will need to register your details to access the questions and response boxes). The evaluation document and supporting evidence is available to view on the NICE website. We have posted a sheet with the questions NICE are asking you to respond to, along with some example responses based on feedback received already. Please include anything that you consider relevant or that you feel has not be captured. Even though data has been collected already and the stories we tell have already been collected, there is absolutely no harm in telling those stories again, emphasizing how they impact lives and the benefits you’ve witnessed. The more ways we can tell the story the stronger our voice collectively is.




Who can we ask for support?


If you need any help or support you can contact a member of the support and advocacy team via telephone 0345 389 9901 or email advocacy@mpssociety.org.uk.




What does this mean for MPS IVA patients who are already enrolled in the MMA? How will they be treated?


Currently access to treatment has been extended to the 31st March 2022 whilst the re-evaluation takes place.

There appears to be confusion from all sides in relation to the two review approaches for existing and new patients. Clarity is needed. The MPS Society and clinical experts continue to raise these questions but please feel free to raise them within your response also.




If newly diagnosed people do not get offered the treatment, what can we do?


We will all need to stick together to support them. It’s the people who have the treatment who have the best opportunity of getting it for others.

There is a strong opinion of “all in this together”. It is important that everyone gets this – it doesn’t matter if you have it already or are newly diagnosed. This approach would raise all sorts of issues in terms of ethics if the decision was a no. Patients should get what they need not what they want.




What can I do to help?


  • Respond to consultation by the 3 December 2021 via NICE website.
  • Use our guidance document if you need help with your responses.
  • Share the link so friends and family can respond as well.
  • Write to your MP to raise your concerns.





 

Latest update​ from NICE - 10 June 2021

BioMarin has requested an extension to the timelines for the re-evaluation for elosulfase alfa (Vimizim) to respond to clarification questions on the evidence submitted to NICE.

 

NICE has accepted this request to ensure that BioMarin has the best opportunity to present more complete data on patients receiving treatment with elosulfase alfa (Vimizim). Therefore, the committee meeting on 10 June 2021 has been rescheduled to take place on 6 October 2021.

The revised timelines for the publication of guidance will now extend beyond the current end date for the managed access agreement (15 December 2021). In response to the amended timelines for the re-evaluation, NHS England and NHS Improvement will work together with BioMarin on an agreement that ensures continued patient access to treatment while the re-evaluation is undertaken.

 

NICE will provide an update to all stakeholders when further details about the arrangements for the extension period are confirmed.

 

Update from NICE - 03 June 2021

Yesterday, NICE released the below update in respect of the committee meeting for Elosulfase alfa (Vimizim). The MPS Society is in close contact with NICE and the company and will provide any updates to the rescheduled timelines as soon as these are available.

BioMarin has requested an extension to the timelines for the re-evaluation for elosulfase alfa to respond to issues concerning their evidence submission to NICE.

 

NICE has accepted this request to ensure that BioMarin has the best opportunity to present more complete data on patients receiving treatment with elosulfase alfa.

 

As such, we will need to cancel the committee meeting on 10 June and re-arrange the committee meeting date. We will update you as soon as these new proposed timelines are available. Read the full update here.

Update from NICE - 16 November 2020

NICE, NHS England and NHS Improvement (NHSE&I) and BioMarin have reached a commercial agreement which will enable the NICE re-evaluation Vimzim to resume. While the re-evaluation continues, they have also agreed a commercial arrangement that will ensure patients can continue to access elosulfase alfa over the duration of the NICE re-evaluation (until December 2021). . Read the full update here.

23 June 2020

NICE have confirmed that NHSE are in discussions with Biomarin regarding the current MAA arrangements, including any additional extensions to the agreement should this be necessary for the re-evaluation of elosulfase alfa (Vimzim).

Statement​ from NICE - 30 March 2020

NICE has sought clinical expert advice in line with the wider NHS response to COVID-19 during these exceptional circumstances around the safe management of patients receiving treatment as part of a managed access agreement. The following statement aims to try and ensure consistency in approaches across managed access treatment centres and that all patients receive equivalent services across the country. Read the full statement here.

26 March 2020

NICE have suspended all appraisals that are not related to COVID-19. This includes the Vimizim appraisal.

16 March 2020

We have been informed that due to mediation by the MPS Society a meeting took place today (16 March 2020) between NICE and the company. Our understanding is both parties had constructive dialogue and have agreed to meet again urgently. We hope that clarity is forthcoming and we will keep you informed of every development that happens. 

12 March 2020

The MPS Society would like to reassure our community, that we are actively engaging with all parties (NICE, Pharmaceuticals, NHSE) and have had a number of sensitive discussions to resolve the current situation. We are unable to comment on these at the moment but will inform you of any developments and plans as we know more. 

 

In the meantime the MPS Society and RDRP (MPS Commercial) have written and submitted their patient organisation report to NICE in the anticipation that the process will continue. Thank you to all of you who completed the survey and gave your personal accounts that has enabled us to put forward a compelling case from our patient community.

5 March 2020

We feel this unfortunate situation has been caused by a misunderstanding between NICE and the pharmaceutical company. The MPS Society are working with both parties to hold conversations and help facilitate a meeting in order to resolve this issue.

28 February 2020

NICE have today (28 February 2020) released the following statement in regards to the re-evaluation of Vimizim under the terms of the Managed Access Agreement (MAA).

NICE statement​

We are writing to let you know that the re-evaluation of elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643] is now suspended.

 

This is because the company has not provided an evidence submission that is adequate for the committee to make a decision, and will not accept the terms of the charging procedure for highly specialised technologies. Whilst suspended, the terms of the Managed Access Agreement (MAA) for elosulfase alfa will remain the same. Access to elosulfase alfa will continue for new and existing patients, providing they meet the conditions for treatment. The re-evaluation of elosulfase alfa for treating mucopolysaccharidosis type IVa (re-evaluation of HST2) [ID1643] was originally scheduled so that the outcome would be known prior to the expiry date of the MAA in December 2020. NICE is following up with the company about its submission and options moving forward. We will write to you with any further updates in due course.

MPS Society response

NICE has released the above statement with regards to the re-evaluation process under the terms of the Managed Access Agreement (MAA) for elosulfase alfa for treating MPS IVA Morquio.

 

The MPS Society are deeply concerned with this news and we are actively engaging with all parties concerned to gain further clarity and assurance that there will be a fair process for our patient communities. We recognise that our members may be understandably concerned on hearing this news and we aim to give you regular updates on the situation as it develops.

 

Meetings have been scheduled over the next couple of weeks between the MPS Society and both the company and NICE to seek a resolution to this unacceptable situation and to ensure our patients have ongoing access to effective treatments and therapies both now and in the future.

​If you are concerned or want to talk to our support and advocacy team about how you or a family member may be affected please call on 0345 389 9901 or email Sophie Thomas, our Head of Patient Services.

What Vimizim means to our members

Sam


“Vimizim is changing Sam’s life. This is clinically proven, but I don’t need clinical evidence. I can see it every day.”




Kamal


“Kamal’s only wish for Father Christmas is ‘to be a normal boy’, and by having Vimizim it gives him the chance to be as normal as can be.”




Angela


“…just being able to get on with life and enjoy my leisure time as everyone else does, without constantly feeling exhausted, has had a very big impact on my quality of life.”




Gracie


“We can’t even bear to think about the fact that it may not be funded. It would feel like we had given Gracie the best chance, to have it snatched away from her."




Sarah


"I have noticed my body really craves the enzyme replacement treatment, I’m very scared about it discontinuing and going back to that dark place.”