On the 19th of May, Biogen attended a meeting arranged between 4 patient advocacy groups: Muscular Dystrophy UK, SMA Trust, SMA Support UK and TreatSMA.
During the meeting we have received an update and were able to ask certain questions. After the meeting with Biogen, all 4 patient advocacy groups discussed how we can combine our efforts to ensure the success of the overall campaign. Below are the main points which community must know.
1. MDUK, SMA trust, SMA Support UK and TreatSMA are going to continue a coordinated effort to advocate and push forward for the treatment for ALL those affected by SMA.
2. Biogen has not been officially requested by NICE to make a formal submission and it is not likely to happen before elections. Therefore, due to regulatory position, EAP is the only achievable target and will continue to remain the focus of the current campaign. However…
3. EAP is used as a stepping stone to ensure that right procedures and resources are already in place within hospitals and NHS for when the regulatory situation changes and further tangible results can be achieved. Furthermore, if the most severely affected patients are loaded through the EAP it should free up the resources down the line when larger number of patients will become legible.
4. Five hospitals in England already running EAP, but a low capacity. Three more hospitals have been given green light, but still have internal issues. Three hospitals in Scotland have been given green light, with one running already and two to join soon. One hospital in Wales has been approved. One hospital in NI already running. Biogen has not refused a single hospital who requested EAP. They are very willing to work with hospitals and NHS, unfortunately, NHS is being difficult. It is down to NHS England to sort this mess out or individual trusts if NHS England refuses.
5. There are 17 children on EAP in the UK right now.
6. Children on EAP will continue receiving the treatment for as long as needed regardless of it being reimbursed or not in the UK. However, when NICE/NHS make decision the recruitment into EAP will stop.
7. Whilst appreciating the hard decision to take you child to Paris, Biogen cannot guarantee continuation of treatment in Paris once reimbursement route starts. This means that we must get EAP running in the UK.
8. If a person currently classified as type 2 is reclassified as type 1 by their medical team, he or she may be eligible for EAP.
9. Newcastle and GOSH are working because they have their own separate commissioning programme. Regional arrangements are unfair though and not substantial and this is why Biogen approached NHS England.
10. NHS has asked that Biogen part or fully finds the auxiliary cost of the program. Biogen are considering contributing towards auxiliary costs, however they must understand where these costs are coming from. Looking at GOSH as an example, it is clear that throwing money into a system is not the answer, since it is physical capacity that is the problem.
11. MD UK is going to provide an audit of 23 hospitals that may be suitable for EAP in order to find out the current constrains within those hospitals. These results will be fed back to parties involved, including Biogen, so appropriate resources and funding can be allocated to move things forward.
12. TreatSMA will require a number of parents whose children are not on EAP already to write to CEO of their hospital which is running or not running EAP and ask why there are delays and what is been done. It is important to keep the pressure on CEO for as long as needed. Weekly letters are generally a good way forward.
13. Charities, TreatSMA and parents should all write to Executive of NHS requesting to know what is going on. Though, families must make letters very personal. This is going to be coordinated a little bit later to maximise the impact.
14. MD UK has proposed a long standing media strategy which is currently reviewed by those involved. With media we must ensure that the relationship between community and reporters grows strong as we will need them for the duration of the whole campaign and not just EAP. We will need many families to join from all types, on EAP, newly diagnosed etc. This will be a massive effort as we are competing with election coverage.
15. Because EAP in the UK has been such disgrace there will be no EAP for type 2.
16. Biogen is going to try to sort out peer reviewed publication by the end of Q2. Meanwhile, there are some ideas of how this can be expedited. (In response to NHS England demand.).
17. Due to time constrains we were not able to ask all questions. Therefore Biogen has agreed to deal with questions by email through Q&A type approach. They cannot provide answers directly to families as this will be in breach of conduct and a possible penalty may be a complete suspension of the process, therefore please ask questions either through TreatSMA or other charities. The questions posted on FB will be collected into an email and sent to Biogen soon.
18. There will be further updates from MD UK, SMA trust and SMA Support UK about other aspects and the information coming from these charities is also very important.
We hope that the information here provides some clarity to the situation. Nothing is perfect and a lot of hard work will continue to be put in by parties involved. At #TreatSMA we believe that all SMA sufferers deserve a chance to treatment.