Biogen have provided the following update to the SMA Support UK and SMA Trust in response to their queries:
Statement to SMA Support UK and The SMA Trust on current status of EAP
Based on the unmet medical need in SMA and with a great sense of urgency, Biogen opened one of the largest global expanded access programmes (EAP) for eligible patients in infantile-onset SMA (consistent with Type 1) in Autumn 2016, before filing with the regulatory agencies. However, as you are aware, the EAP in England has met with ongoing infrastructure and capacity challenges within potential administering sites resulting in the EAP not yet being fully established.
In order to address these ongoing challenges, a number of discussions are taking place with NHS England and members of the SMA clinical community to ensure that the programme is put on a more sustainable footing in order to minimise piecemeal and ad hoc access, not only now but in the future.
Since the last community update on the EAP, a number of clinicians have been in touch with Biogen enquiring about their hospital becoming an EAP administering site. However, approval of these requests are dependent on the necessary feasibility checks undertaken by the hospital to determine that all required ancillary resources are available; e.g. appropriately trained personnel, clinic time and space and costs and time associated with supportive care and management. Progress is being made, but for those sites that are up and running, patient numbers have been necessarily limited as a result of infrastructure and service set up resulting in the need for treating physicians to clinically prioritise accordingly.
The primary outcome of these early discussions is to review options for both number and location of potential EAP sites as well as the prioritisation of patients that are most clinically in need of treatment. In conjunction with the SMArtnet network clinicians, Biogen is in discussions with the SMA clinical community to gain broad clinical consensus on the number of administering sites to operate as ‘hub sites’ to accommodate patients from a larger geographical area, from referring ‘spoke’ sites, as well as the criteria to define the highest clinical need and necessary prioritisation.
Biogen will therefore submit a proposal for NHS England to consider a commissioning framework to support the implementation of the EAP.
Biogen acknowledges and understands the frustrations and disappointments experienced by those families hoping their child can access nusinersen via the EAP and is working expeditiously with clinicians, commissioners and SMA Patient Organisations to find an appropriate solution.
We want to assure you that we are committed to working with all stakeholders to ensure access to the EAP in a timely fashion whilst continuing to plan for a viable reimbursement pathway should nusinersen be approved. We believe this is the best way to provide broad and sustainable access to the SMA community. We will continue to address your requests for more information.
Biogen – 7th March 2017
Source: SMA Support UK