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Update on the UK Expanded Access Programme (EAP) for Nusinersen for Children with SMA Type 1

20 January 2017

We have been talking with Biogen about what progress there has been on extending the EAP in the UK.

There are two strands of approval and negotiation before a UK Centre can offer the programme and enrol children. These are:

  • The application to Biogen for supply of the drug. Biogen will then review each request in line with their necessary feasibility and governance requirements. If the application is agreed, the drug is then supplied free to the Centre.
  • The discussions that a Centre has within its own hospital and with its NHS Trust about administering the treatment. These discussions need to cover whether or not the staff, facilities and funding required can be made available. Both the hospital and the Trust are managing many competing needs.

A Centre may apply to Biogen before or after the Centre has concluded its internal discussions with the hospital and Trust.

Biogen’s EAP for Nusinersen for children affected by SMA Type 1 in the UK has broad overall inclusion criteria. As long as the Centre is administering the drug to children who meet these criteria they will supply the drug. However, in view of the costs in terms of staffing, facilities and available funding for the administration of the treatment, an individual Centre is likely to be required by their hospital / NHS Trust to establish ‘local criteria’ which will restrict access to their particular EAP Centre. This may be criteria such as: prioritising babies that the clinical trials have indicated are likely to receive the greatest benefit from the treatment; the distance from individual centres/ travel; the severity of the condition; the age of the child.

Biogen has let us know that they have either been approached by or had conversations with several Centres other than Newcastle and Great Ormond Street Hospital (GOSH) in London, across the UK. These Centres will have either already carried out, or be in the process of carrying out, their own internal discussions as to whether or not it is feasible to offer the programme and, if so, what local inclusion criteria will apply.

This dual process may mean that, though families may hear that Biogen has approved a Centre for an EAP, how many and which children may have access to that Centre’s EAP is entirely for the clinician/hospital to decide. As these decisions involve many complex considerations, they may still be under discussion despite Biogen’s ‘green light’.

Biogen has advised us that they are committed to doing all they can to process applications as quickly as possible. Centres, including Newcastle and GOSH, are also working hard to progress the programme.

We appreciate that this must be immensely frustrating, confusing and distressing for families. At the moment we have no further information about exactly what is happening where in the UK but we will continue to ask questions and let you know as soon as we hear more information.