Spinraza treatment only for some?

Dear Community,

Welcome to our latest update, and please note this is a very important update, therefore please could we suggest you read the entire post. As you know on Monday we shared the entry criteria for the Managed Access Agreement (MAA) which is the criteria of which it is decided whether people with SMA can receive treatment. When the MAA was released we expressed significant concerns and have continued to express concerns all week.

Whilst we have had some initial positive conversations, we have still not received any word of amendments to the entry criteria. This is a concern and we, the community, need to consider our next steps.

Firstly, let’s remind you of the entry criteria and why we think it needs to be changed:

TreatSMA view

No permanent ventilation (≥16 hours/day for 21 consecutive days in the absence of acute reversible infection)/ tracheostomy requirement at baseline;

This requirement needs to be removed. The chances are this will only affect a small number of people, but the treatment would still help those both in terms of improvements in fine motor skills and endurance. This is a fundamental principle behind the treatment, with stability being as important as improvements. The exit criteria states that a patient who is on treatment and deteriorates to the point of requiring permanent ventilation then treatment should be stopped, we agree with this point. If the person is not seeing any improvement and is continuing to deteriorate then this is valid grounds for removing treatment. In summary, we have requested that this be removed as an entry requirement but kept in the exit criteria.

Intrathecal injection must be technically feasible in the opinion of the treating clinician and not contraindicated;

In principle we agree with this requirement under the criteria. The treatment is only licensed in the UK for intrathecal injection, and therefore this is the only option available for clinicians. Clearly if it is neither safe nor feasible then the treatment would not be able to be administered.

Must not have received spinal fusion surgery following a diagnosis of scoliosis which prohibits safe administration of nusinersen;

The wording of this requirement needs to be amended, and if not, could exclude a significant number of people. The word “must” does not accurately reflect the true situation of people who have spinal fusions. There is also significant concern around the impact this may have on people planning to have fusions. They may decide to delay or not have one altogether, which has its own severe health consequences. There are many types of fusions and it is important that we encourage NHS hospitals to investigate thoroughly the options available to them. We do not want to find ourselves in a position where a hospital is able to simply reject the patient on the basis of a historical fusion. There are large numbers of examples across the world where people are still receiving treatment despite their lumbar region not being available. These range from ports, injections into alternative locations along the spine and having small holes drilled in the vertebrae to allow access. We have asked for the language to be softened and to make it clear that under the NHS Constitution of Patient Choice, if one hospital is unable to provide the treatment then people should have the right to find alternative centres who can provide the treatment, of course subject to the above requirements.

Must not have severe contractures which in the opinion of the clinician prohibits measurement of motor milestones;

We have requested that this requirement also be removed due to its somewhat arbitrary nature and that it is not in line with the draft NICE guidance that was released. We believe it was introduced due to a misunderstanding around the expectation of people reaching milestones. As mentioned above, stability is just as critical as improvements.

If gained independent ambulation prior to initiation of therapy must still be independently ambulant. Independent ambulation is defined as per the WHO definition: patient takes at least five steps independently in upright position with the back straight. One leg moves forward while the other supports most of the body weight. There is no contact with a person or object;

We have also requested that this requirement be removed. We believe this could be the most significant exclusion criteria limiting treatment for the largest number of children and adults. This suggests that someone who has previously walked, but has deteriorated and has lost the ability to walk while not receiving treatment, is no longer eligible for treatment. This is absurd as there is no clinical justification for this requirement and it leads to significant inequality with access to treatment. For example, you could have two children, both SMA type 2, but because one of the children walked, albeit very limited, they wouldn’t be eligible but the other child would be. It also assumes that the patient who has lost the ability to walk wouldn’t regain it if they started treatment. It also completely ignores the fundamental principle that this is not just about improvements, it’s about stability.

Must not have severe contractures which in the opinion of the clinician prohibits measurement of motor milestones;

With regards to people with SMA type 4, those who have not had symptoms at or after the age of 19, we have proposed an amendment to the wording to include, “unless in the opinion of the treating clinician the patient is on the verge of losing independent ambulation or another motor milestone.” This gives clinicians the ability to exercise their own judgement and patient history in the decision whether to proceed with treatment.

Must not be type 0 SMA patient.

This is a difficult requirement because type 0 is not clinically defined. This would affect a very small number of newborns but nonetheless we have requested further clarification as to what type 0 actually means in relation to the criteria. The team have seen a number of examples where there has been a misdiagnosis of type 0 and therefore, we need far more clarity around this exclusion.

Please read these comments as sent to NICE and the NHS

Hopefully that is clear but if you have any questions please don’t hesitate to ask below. The important point to note in all of this is that while we have provided robust challenges to the wording of the MAA we have still not seen any evidence that the NHS are prepared to modify the criteria.

Next steps

This puts us in a difficult position because TreatSMA has always been very clear that we advocate for all people to receive treatment, and not a subset. We believe the MAA no longer meets the statement made by the NHS Chief Executive: “The NHS has now reached one of the most comprehensive deals in the world”.

At this point in time TreatSMA remains unequivocal in its commitment to campaign for access to treatment for all sufferers of SMA. This means that as of now, we are prepared to formally challenge the MAA criteria. We have up until 17 June to submit an appeal.

Risks of appeal

There are risks to appealing that we wanted to share with you, and the most fundamental risk is the possibility that it delays access to the treatment for those who are eligible under the MAA. An appeal can take anywhere from 3 months to one year, but by way of comparison, if the MAA is accepted as written it cannot be amended for a further 5 years.

So we have a significant conundrum whereby if we appeal we run the risk of delaying treatment for those who will be able to receive it under the current proposed MAA, but equally if we don’t appeal, we have abandoned our principles of “treatment for all”.

We need your input

To this aim we would be very grateful to hear your views so we can take these into consideration. Of course, anybody can appeal the criteria individually, but we are stronger together as a community and can leverage the relationships we have already built up. We are considering several options this week including looking at revitalising the press coverage we have received previously, so no options are off the table at present.

We know the NHS doesn’t like negative publicity, and after the press coverage following the approval, the last thing the NHS we want is to be seen as rolling back on this decision. So please do give us your views and suggestions.