You may remember some time ago we posted that MHRA had approved an EAMS process for accessing risdiplam. Just to remind everybody what an EAMS process is, it stands for Early Access to Medicines Scheme, and is designed to provide those who currently have unmet clinical needs to get access to life-saving treatment before it is licensed in the UK.

This program replaced the initial Compassionate Usage Program set up by Roche, and was felt by Roche that this was a more appropriate and streamlined method of people receiving the treatment. The theory is that the clinician now makes the ultimate decision as opposed to a pharmaceutical company. Despite these assurances, the reality seems very different.

As ever, the devil is in the detail, and things are not progressing as we would have hoped. The process for getting access to risdiplam is complex. Firstly, a consultant needs to agree that the patient is eligible for the treatment and that there are no other alternatives available to them due to clinical difficulties. Ironically, that appears to be the easiest part of the process, the second part is that before the consultant, who has agreed you can have it, can prescribe the treatment, it needs to go through another process called a Drug Therapeutic Committee.

These committees are often managed by the local trust themselves, although sometimes they are area-wide including other elements of the local health economy such as Clinical Commissioning Groups (representing groups of GP surgeries and other health functions). The consultant cannot prescribe unless it has been agreed at the local committee regardless of need or clinical circumstances. This is where we are seeing some challenges.

Under the guidance issued from the MHRA the following statement applies:

“A positive EAMS Opinion will simplify governance around the use of the medicine. NHS organisations should not duplicate the provision of an EAMS Scientific Opinion at local level in NHS governance and clinical decision making.”

We believe that this statement is quite clear, local hospitals should not be second guessing the eligibility or methodology used by MHRA to arrive at their scientific opinion. The local Drug Therapeutic Committees should be considering how to implement and what additional resources are required, they should not be reconsidering the clinical efficacy or any data.

Firstly the good news, it would appear that of the hospitals we know so far who have had their local committees, all have been approved for paediatric patients. We can’t give you a list as yet because we are not entirely clear of which areas have been approved, but we wanted to provide you with this information as soon as possible so you can query the position with your own neuromuscular centre. At the moment this means that any paediatric patient who is unable to receive Spinraza for clinical reasons, and is cared for by one of these centres who have approved risdiplam, should now be able to proceed with treatment.

Now comes the bad news, adults have once again been left out in the cold. We know of at least a couple of organisations who have approved it for paediatrics but have denied it for adults on the basis that there isn’t enough efficacy data to support treatment. Bringing you back to the statement above, directly from MHRA, we would challenge that this is irrelevant. The local hospital should not be reassessing efficacy data as this would be second guessing the MHRA process.

We have sought help and advice from Roche in terms of additional data, but as yet that hasn’t been forthcoming. We are urging Roche to help with this process or provide other potential avenues of access. It may be that the data isn’t available, and that’s something we will need to accept, but it still doesn’t explain why local hospitals feel that they can second-guess MHRA. We have also contacted NHS England and the MHRA directly to get their views on the position, we are awaiting replies.

This is now the second life-saving drugs that adults have been denied. As you may remember, last year at Spinraza was approved in June, including for adults, but to the best of our knowledge, only two adults nationally are actually receiving treatment. We have been subjected to excuse after excuse from hospitals across the country as to why they are unable to provide this fully licensed treatment to those who are eligible.

Now we have a second, a new treatment, which requires minimal involvement from the hospital. What makes this even more frustrating is that Roche are providing the treatment free of charge up to the point that it is approved by NICE and available on the NHS. While hospitals may cite lack of evidence, they are actually exasperating the problem by not prescribing because there will never be evidence if that’s the case. They also don’t appear to understand the mathematics that SMA is a rare disease and that there won’t be huge amounts of efficacy data for some time to come. This argument is also going to be made when it comes to applying for NICE approval, therefore it’s important people get onto the EAMS as soon as possible.

We mustn’t sit back and watch ourselves or our loved ones continue to deteriorate, and in some cases, lose their battle when there are available treatments. If this was a cancer treatment there would be no hesitation in providing access as the government has a specific cancer drug fund available for just this scenario. Time for us to once again rattle the appropriate cages and make sure all organisations, pharmaceutical companies, NHS, local hospitals, know that we aren’t going to go away quietly.

What do we need from you?

Firstly it would be really helpful if you could contact your local neuromuscular care centre and ask them what their current position is with risdiplam. For example, has it been approved through the local Drug Therapeutic Committee, or named equivalent? If so, has it been approved for paediatrics and adults or just paediatrics?

Secondly, we need to start writing to local MPs again making them aware that the NHS, more specifically, local hospitals, are once again denying treatment to those who require it urgently.

Thirdly, and finally, provide us with any press contacts you may have locally so that we can coordinate an appropriate campaign to stop history repeating itself.