Several eagerly anticipated trials are investigating the clinical effectiveness of new treatments that will work in combination with those that focus on the role of SMN protein in SMA (nusinersen, risdiplam and Zolgensma). You can read about these new ‘anti-myostatin’ treatments and the results of trials to date on this page.

November 2022: NHS England had made a decision that meant that if a person’s nusinersen or risdiplam treatment was funded via a Managed Access Agreement, this would stop if they joined one of these trials. This affected people in England, Wales and Northern Ireland but, as far as we had been advised and were aware, not Scotland.

You can read more about:

Why NHS England made this decision >

Why patient groups worked with clinical leads to challenge this decision >

The Principal Investigators from the Trial Centres met with representatives from NHS England and NICE and raised all the reasons why this decision needed to be challenged (see link above).

Similarly, the Patient Groups wrote to these representatives asking for an urgent face-to-face meeting of all parties.

The Patient Groups also agreed that they would alert other Rare Disease Groups that have, or may have, a treatment for their community funded via an MAA, of the issues it raises. We asked NHS England and NICE to meet with all these groups and appropriate clinical representatives to discuss the issues.

Clinicians and Patient Groups asked Pharmaceutical Companies to advise NHS England and NICE what impact they considered the current agreements would have on their future interest in the UK as a market for future trials and drug development.

22nd December 2022: NHS England advised us they were, “…committed to taking your responses on board, including any wider implications, and are taking legal advice on this matter.”

17th January 2023: we were notified by NHSE that this advice was now being worked through and that we would be notified of the outcome as quickly as possible.

23rd January 2023: As trials are internationally competitive, we were concerned that anyone in the UK who met the trial clinical eligilbity criteria whose SMN disease modifying treatment was not funded via an MAA should be invited to make contact with the trial centres. This included the possibility of people coming from Eire as the Common Travel Agreement ensures access to healthcare for Irish citizens post Brexit.

25th January 2023: We were delighted to be able to let the community know that NHS England had reviewed their decision as follows:

"In response to communications from stakeholders, NHS England has carefully considered all of the information about this complex situation. We have an ongoing concern that this may impact the ongoing data collection within the managed access agreement if patients already accessing nusinersen or risdiplam through one of the managed access agreements (MAAs) join a commercial trial for another potentially disease-modifying drug. We do however acknowledge that, in this specific case, there has been a relatively long period of data collection (especially for nusinersen), including for some of those individuals who may now be eligible to join the Sapphire trial. In this specific case, therefore, NHS England will exceptionally continue to fund nusinersen or risdiplam through the MAAs for those patients accessing the Sapphire trial (link). NICE will consider how this will impact the data collection agreement going forward and work with the key stakeholders to ensure the data collection can address the key uncertainties identified by the NICE committee.

This decision has been made on an exceptional case basis. Depending on the nature of the trial, NHS England may not always be able to commit to continuing the funding nusinersen or risdiplam through an MAA where a patient then joins a commercial trial for another potentially disease-modifying drug. Cases will be considered on a case-by-case basis on their individual merits.

Clinicians should always contact NHS England before committing to such commercial trials or to individual patients / families."

Sapphire Trial >

Clinicians will be discussing access to any other proposed trials in the UK with NHS England. These would include:

Resilient trial sites proposed in the UK >

Manatee trial sites – current and proposed in the UK >

We will continue to work with clinicians and NHS England and update you when we have further news.