Although precautions are needed in its use, there is evidence that Ravulizumab, like its predecessor eculizumab, is effective in doing its job in those whose complement is dysregulated. Whether that dysregulation is due to genetic defects in the components of their complement system or in other systems in the body which hamper the effectiveness of complement controls, it works.
It may even work for other conditions where thrombotic microangiopathy is at play.
But is it cost effective, is it affordable? Two questions that will be asked by healthcare providers and policy makers around the world, even by private health insurers.
For it to be used at all Ravulizumab is going to have to be listed as a therapy that a country or a insurence company approves as a technology that is licensed as safe and effective and whose cost can be covered from the funds they have for such medical technologies.
How that happens depends on the way aHUS patients medical costs are covered. Only very rich people with aHUS can pay for it out of their own pockets, most aHUS patients must hope, either that any provision they have personally made to insure against cost of potential illnesses, or that their nation’s social medical funds will pay for it.
There will be many people around the world who will have the job of making the decision “ can we afford to pay for Ravulizumab for an aHUS patient?”
Whether eculizumab has been approved in their country in the past and Alexion has sold it there is perhaps the best predictor of whether Ravulizumab can be afforded too. As Ravulizumab is also indicated for PNH and approvals for use of ravulizumab for PNH are occurring around 9 months ahead of aHUS evaluations, what happens to PNH patients is also indicative of what could happen for aHUS patients.
So far this has happened for PNH patients in the USA , Germany and Japan, and in recent months in Australia. The Australian medicines approval authority took just 180 working days to decide to list Ravulizumab for PNH. Ravulizumab for aHUS has subsequently been approved in the USA and soon Japan.
Cost effectiveness and affordability depends on what Ravulizumab is compared with.
In the “Spotlight on Ravulizumab 1” about its dosing, we reported that the volume of the drug used is less than that of eculizumab over an 8 week period.
But whether the cost is less depends on the price per vial of Ravulizumab. Reuter’s has reported that Alexion has filed the price of a vial of ravulizumab at about $6400 about $150 less than for eculizumab.
So with less volume and a slightly lower price Ravulizumab would appear to be more cost effective and affordable than eculizumab. If eculizumab is being reimbursed now it would cheaper for the payer to switch to Ravulizumab.
But compared with the less clinically effective plasma therapy and long term damaging and burdensome dialysis , can it be cost effective?
If the comparison is made with the highest volume dose cost, as was mostly the case when eculizumab was evaluated, then the gap in cost between the treatment modes will be very big, but so will the clinical benefits in terms of longevity and quality of life of aHUS patients. But by most cost effective benchmarks its cost would still be unacceptable.
In eculizumab health evaluations the average age determined the amount of eculizumab dose. The top dose level for eculizumab would be reached by 12 years of age. The avaerage age of a patient would be about 28.
Ravulizumab has more dose weight steps prescribed with a top weight band of 100kg. The dose level for the average aHUS patient weight is going to be less than 100 kgs. so the volume of drug needed is going to be less. A weight profile across the spectrum of aHUS patients would determine the average volume of drug needed.
Assuming an average weight of an adult to be around 60kg and a child of around 20kg, then 10 vials of ravulizumab would be needed every 8 weeks for adults and 7 vials for children. The overall average would be between the two , say 9 vials per 8 week cycle . That would be around 58 vials per annum ( 6 .5 treatment cycles in 52 weeks) . The equivalent number of vials for exulizumab would be 26 treatment cycles @ 4 vials or 104 vials per annum for adults and 26 x 2 or 52 vials for children, around about 80 vials per aHUS patient overall. Treatment of aHUS with Ravulizumab would require just less than three quarters the volume of eculizumab , 22 vials less, or about a 27.5% reduction in cost.
But there is more to consider. Ravulizumab prescription specifies that treatment should be for 6 months and then the treatment should be individualised. This is a recognition of the real life data that has been collected on the likelihood that for some aHUS patients withdrawal from treatment is safe to do even if occasional relapse happens..
The exact figure is yet to be determined but if was the case that a third of aHUS patients needed treatment for life , a third could withdraw from treatment and not need it again and the rest may withdraw from time to time, that would mean that only 50% of aHUS patients would need treatment at anyone time.
So 50 % of patients treated with ravulizumsb needing 70% of the dose volume for eculizumab would reduce avarage cost of treatment of aHUS considerably. Even more so when compared with that top weight dose level of eculizumab for life which is the topline “alarmist” figure that is frequently used by health providers/policy makers to deny treatment to aHUS patients.
Real world evidence of personalised treatment of the average weighted aHUS patient would reveal a much smaller gap between the cost of ravulizumab and its alternatives of PEX and dialysis; whilst the huge gap in the relative clinical ineffectiveness of the latter remains the same.
With more precise work on the “pharmacokinetics” and “pharmacodynamics” of ravulizumab an even better case could emerge for the affordable use of this precious resource.
That completes this series of articles about ravulizumab , the most information provided by any aHUS patient organisation. Unless those who have gone through or are going through the transition from eculizumab to ravulizumab would be willing to contact us to tell us about their experience.
Further articles about ravulizumab by the aHUS Alliance can be found at :