Article No. 434
10 May 2021
The Scottish Medicines Consortium (SMC) has approved the use of Ravulizumab for treating aHUS in Scotland. This follows on from its earlier decision to use it for PNH treatment too.
The SMC acknowledges the contributions from patients to their decision making process. Whilst quite slick, the evaluation has taken from early Autumn until now for a decision to be made. It involves written and oral evidence from Alexion and clinicians too.
But now it is made, the 2 or 3 new aHUS patients on-setting in Scotland each year will not need to apply individually, with trepidation, for access to Ravulizumab, Such access has been denied to some. There will now be assured universal approval and funding.
Treatment will be given free of charge at point of delivery when needed.
Similarly, those 10 to 20 or so Scottish aHUS patients ,who may have accessed eculizumab, will be permitted to transition to Ravulizumab and in doing so reduce treatment costs.
Past aHUS patients, if any, not on eculizumab would also be eligible, maybe to permit a kidney transplant.
There are some qualifications to the decision in that the patient access scheme price discount continues and the cost effectiveness evidence provided by Alexion proves to be correct in practice.
Every country goes about approving drugs like Ravulizumab in their own way ( NICE will be announcing its decision later this month) but the process is essentially the same. The clinician decides if it is of clinical benefit, the hospital/ health policy maker agrees to include it in its pharmacy, someone agrees to pay for it and then Pharma delivers it.
In some countries getting someone to pay for it has to done by hundreds of patients individually. It takes time and it is a burden for patients and their families at a difficult time in their lives. It is frequently not without financial costs to them.
Alternatively, there are countries where there is one application and a patient organisation coordinates the patient input. This takes a lot of effort. Calls for evidence are made and must provided. Not emotionally but rationally with authority. It takes hours of work to draft answers to the questions being asked.
It is not something anyone can do and it is done with feeling the weight of expectations of those who would benefit. Pressure is felt for the duration of the review , be it for six months or several years.Hundreds of hours invested , sometimes for nothing.
That weight on advocates shoulders is lessened if the voice of patients is gathered to provide evidence relevant to the decision being made. Collectively the voice is louder.
In SMC case evidence was based on the research undertaken last August when 18 aHUS patients told aHUS alliance Global Action what their experiences and expectations of Ravulizumab were having been treated with eculizumab. Together their words were insightful and influential.
aHUS Patients, who they may never know or who may not know about them, will benefit in the future thanks to them. That is what the aHUS community can do together.
A copy of the abridged research reports used can be read in our info centre HERE.
These days health policy decision making entities like SMC have people to work with patient advocates to guide them through the evaluation process. aHUS alliance Global Action would like to put on record our thanks to the public involvement advisory team at Health Improvement Scotland /SMC for their advice and support which made a difficult job much less of a challenge.
The SMC decision means that those at risk of this very disease in Scotland will not only be less harmed by it but also their life will be much freer and easier on Ravulizumab.