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Further Q and As about Nusinersen’s availability in the UK

(last updated on 7th December 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. What about other clinical trials? If a child / adult signs up for one and then nusinersen becomes available, will they be able to access nusinersen treatment?

The nusinersen EAP for those with SMA Type 1 excludes any child who has previously or is currently participating in a clinical trial with an investigational gene therapy for SMA. It also excludes patients who have participated in a study with an investigational therapy for SMA within 6 months or five half-lives of the investigational drug, whichever is the longer, prior to the first dose of nusinersen.

If you are thinking about signing up for a trial of another treatment and are concerned is this could affect your potential eligibility, check directly with Biogen at Patientcenter@biogen.com

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Q.3. What if a child / adult is being treated with nusinersen, could they also sign up for a clinical trial?

If the trial criteria state that participants cannot have  received gene or cell therapy, SMN2 antisense oligonucleotidetherapy or any SMN2 splicing modifier then the answer is ‘no’ as this would include nusinersen.

We understand that salbutamol is not regarded as a treatment for SMA but may be part of approved care for SMA. This means it would not prevent enrolment on a trial or for treatment. However, this would need to be confirmed with the treating medical team / pharmaceutical company.

Anyone considering applying to be enrolled in any future possible UK trial for another treatment that is developed would need to seek advice from the pharmaceutical company and their medical team with regard to their eligibility.

Q.4. How will the UK price be determined? Could it be too expensive for the NHS to fund?

A. Answer

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? 

A. Answer