Progress with Access to Nusinersen in European Countries for Adults who have SMA
25th June 2020
Thank you to the SMA Europe member countries who responded to our request for information about the reality of access to treatment by adults in their country. Some provided other information about access in general. Responses included testimonials from adults who have SMA Type 3 and are being treated. We have a long way to go in the UK.
We have to discuss reimbursement for Spinraza again in Belgium. We had a temporary accord for 2 years with inclusion and exclusion criteria. Many adults are receiving treatment except those who have permanent ventilation and / or difficulties administering the drug. We hear many testimonies about the effect of Spinraza, but we don’t have the data. Biogen has been collecting this data over the 2 years. We don’t know if this data will be published. To evaluate the effectiveness of the drug, we would need more time: 2 years it’s short!
Biogen wants to continue the treatment of every person treated but they have to negotiate the future with our government. We have asked our members to write testimonies. We have already gathered 15 adult’s letters, and everybody wants to continue the treatment, it changes our lives! Some people feel a stabilisation and some other people feel stronger! The scales HFMSE, 6-minute walk test, RULM are sometimes enough to measure the improvement and sometimes not (they don’t show less fatigue, better energy, etc). Spinraza is definitely useful and important for adults, it brings SMN and stops the progression of the disease. Now we hope that Risdiplam will shortly be available for adults who have type II with spinal fusion or physical difficulties that don’t allow Spinraza injections. And we are trying to help people with permanent ventilation too.
Read Veronique’s testimonial here – she has SMA Type 3.
Unfortunately, we have no Danish adults being treated.
Spinraza has been available in France for more than 3 years. Today all Type 1, 2 and 3 can have an access. More than 90% of children are receiving treatment
Adults can have access but due to the difficulties with the injection, fewer than 50% are receiving treatment today
There is no access of Spinraza for Type 4
Regarding newly diagnosed Type 1 or 2, since the beginning of 2020, most of them are treated by Zolgensma with the early French access program.
All patients with SMA can have Spinraza, regardless of tracheostomy, peg, walking...
Some don’t want it because of the procedure and are waiting for Risdiplam. The EAP for Type I for Risdiplam has been running since March, but so far not many applications.
We have a pilot NBS since January 2018 in parts of Bavaria and North Rhine-Westphalia. We have applied for implementing NBS in the regular NBS program and this long process is under way.
I regret I can’t provide a breakdown, but the total number of patients (children + adults) who have been approved for nusinersen treatment is slightly above 700 while the number of those who have already commenced the treatment stands at mid-500s. I estimate the number of adults currently receiving nusinersen in Poland at around 200 (all ages). This is in relation to the total number of patients estimated at 800–1,000.
I have now sent a query to the medical committee that approves individual patients and will share any response that may come.
As to the procedures itself – a proportion of adult patients (approx. 10%) require injection under radiography (usually MRI). This does pose a challenge as most hospitals do not have teams ready to perform the procedure, so those more ambitious ones get the load of “difficult” patients. Timewise, it is prudent to book 1 full hour for a radio-guided injection, plus the time to move the patient to/from the MRI unit.
A few adults in Poland are receiving nusinersen via an Ommaya reservoir or another intracranial system. I have no further details as I did not closely follow on that.
As for effects, approximately 40% of adult patients in Poland report meaningful improvements within a year of starting the therapy. We are in the process of collecting information in a more systematic way and should have a report ready by September.
Here is a recent press article about a lady receiving SMA treatment.
We have approximately 50 adults who wanted to access Spinraza. They have either all started or will start by the end of this month. They were included in a negotiation that the partner of Biogen did with our Health care system, and them together with all the children (around 100) also started with Spinraza. Also, there are always efforts to be included also children which are newly diagnosed.
But we also have some adults, very few indeed, that did not want to be treated.
The situation is complicated. On the one hand everybody could be treated, but all payments for treatments are decided by the region where the person lives via an individual reimbursement procedure. (The regional budget purchases the drug for every single patient). There are NO all-Russia reimbursement SMA programmes in place, so due to the current legislation every patient has to apply to their regional local Ministry of Health (there are 85 different regional MoH) for treatment. The regional MoH then decides on the budget\coverage\possibility of delivery of treatment for each individual. This currently means there is a lack of clarity about who receives treatment. When we analyse that decision process, we don’t see that these decisions are based on type\condition etc.
We have some patients Type 2 and 3 treated with Spinraza, but currently only 1 adult treated - but this isn’t due to any official access restrictions operating. Two more adults will be treated in the coming weeks. In some regions, the first patients to receive Spinraza have been Type 3 while those who have SMA1 or SMA2 were rejected. So, there is no clear pattern. In this situation a lot of factors could influence the possibility to receive treatment. In almost at all cases patients have required legal support to obtain individual approval to receive treatment
But when discussions take place at Federal level, there are always attempts to make some sorts of prioritisations based on types and other aspects of the condition - but the final decisions on SMA Federal reimbursement have not been finalised yet.
Spinraza applications started in Serbia in July 2018 with children. We started with 4 patients who have Type 2. Now, we have 34 patients receiving Spinraza. We have a named patient programme depending on funding availability with no limitations regarding tracheostomy, NIV or PEG. Every newly diagnosed child automatically receives Spinraza. In general, we have all types receiving Spinraza but for Type 3 patients, even if they are ambulatory and are children it is hard to get access. We have some with access, others are waiting. It depends on a budget for rare diseases.
It is hard to get access for adults - we have 4 diagnosed with SMA, with age range 27-37 years old. They all are type 3, and they are wheelchair users. They began their Spinraza journey on September 19th, 2019. What is in common for all the patients during their journey, is that none of them have felt any disease progression since starting treatment and two people have shown some improved motor functions on the Hammersmith test. There were no difficulties with Spinraza injections for the two patients who had no scoliosis problems. With the two other patients that had had spinal surgery, there were some problems with administration of the medicine and several injection attempts had to be made. However, after the second dose there were no more problems.
Concerning doctors, their position is that everybody with SMA should receive some kind of therapy, and they are on our side and have strong support. We have 8 patients in the risdiplam Sunfish study (different study arms). We are in the process of setting up the CUP for Risdiplam (already 4 older children type 1 have applied) and we are waiting for Type 2s.
We have Rainbowfish and are trying to implement a pilot project for NBS with Roche and our Ministry of Science through projects they are financing.
In less than 2 years we have achieved 50% of patients who have SMA receive therapy.
The main problems are still type 3 patients no matter what age they are, and adults.
We would like to share our personal stories from people who have SMA Type 3 - read more.
Official restrictions on access to adults are linked either to permanent ventilation, a very low functional activity (EK2) or due to physical difficulty administering the drug. In reality, adult access in Spain has been quite good, although we are not free of “soft denials” (taking time to send the patient to a reference centre, or “this drug is not for you”, “you will not experience any improvement” “wait for the oral drug”). On the other side of things, we do have adults receiving the drug with guided lumber punctures, Omayah reservoir or other attempts to really try to get them on Spinraza.
The Netherlands (updated 29th October 2020)
In the Netherlands there is a reimbursement agreement for Spinraza for all SMA types up to the age of 9.5 years. Since January 2020 a conditional approval program started for all SMA types above the age of 9.5 years. This program will run for 7 years. Based on the results a decision will be made on Spinraza reimbursement for this group patients