Q and As about Nusinersen’s availability in the UK
SMA Support UK has been asked many questions by families and invited the SMA Trust and Muscular Dystrophy UK to add any additional ones to the list. SMA Support UK then liaised with clinicians who had been involved with the clinical trials and with Biogen for accurate technical answers to the questions that required their input.
These are the responses we had in March 2017. We have continued to update answers. These Q and As were last updated on 2nd June 2017:
Q.1. Have all clinical trials for Nusinersen now stopped or is it still possible to enrol on any clinical trial for Nusinersen?
A. All clinical trials are advertised here. There are currently no trials recruiting for nusinersen. There is, however, an Expanded Access Programme in the UK for Nusinersen for children with SMA Type 1. For the latest information on this programme, please click here. On 13th April 2017 Biogen announced that it would not be extending this programme any further than SMA Type 1. You can read this announcement here.
Q.2. We have heard about the Roche clinical trials (Firefish, for SMA Type 1 and Sunfish, for SMA Type 2 and 3). If we enrol our child in one of these trials, does that mean s/he won’t be able to access nusinersen through the EAP for SMA Type 1 or if it is approved for licensing in the UK ?
Q.3. Now that the European Commission has given approval for marketing for treatment for 5qSMA, which includes SMA Types 1, 2 and 3, will NICE and the rest of the UK follow?
A. The drug needs to be assessed for cost-effectiveness before it is made available in the NHS. The National Institute for Health and Care Excellence (NICE) is the body that conducts any such assessment in England; this guidance also applies in Northern Ireland. Wales does have its own process for determining cost-effectiveness through the All Wales Medicines Strategy Group but may follow the decisions made by NICE. Scotland has a separate appraisal process through its equivalent to NICE, the Scottish Medicines Consortium. You can read more about the NICE process here.
Q.4. How long will it take from now to the point when it can be accessed in the UK?
Q.5. How will the UK price be determined? Could it be too expensive for the NHS to fund?
Q.6. What are the eligibility criteria for getting nusinersen in the UK going to be? For example, will it be for all 5q SMA? Will there be lower or upper age limits?
A. At the moment, no-one knows the answer to this. The European Commission decision to grant marketing authorisation for 5qSMA, which includes SMA Types 1, 2 and 3, gives an indication of potential prescription criteria. However, NICE could further limit eligibility based on cost criteria. What NICE can’t do is expand the criteria once they are set by EMA.
Q.7. Will it be available to anyone with any type of SMA, including Types 3 or 4?
Q.8. What Centres will offer it? Will it be restricted to certain Centres or more widely available?
A. By the time a decision has been made about whether the drug is going to be made available in the UK, there are likely to be Centres across the country that are, under the Expanded Access Programme, offering the treatment to children with SMA Type 1. These Centres would have experience of administration of the treatment.
It is likely that, if NICE does assess the treatment, they will express an opinion as to whether it’s administration should be restricted to certain Centres.
Importantly, we have been urging, and will continue to urge, NHS England and NICE to adopt commissioning frameworks that allow for equitable access based on clinical eligibility not on a postcode lottery which is dependent on where someone lives.
Q.9. If nusinersen gains approval for reimbursement in the UK but my local health authority won’t pay for me / my child to have nusinersen, what can I do? Could I move to an area where they are paying for it?
A. If the treatment were to be finally agreed and provided as one that is highly specialised, it would be commissioned nationally by the specialist clinical commissioning group (SCCG). Your local area would not be paying for it. There may though be other criteria for access. It is too early to answer this question in any really helpful way.
Q.10. Now it's licensed by the European Commission, will I be able to access it privately?