The recent incident in British Columbia about access to eculizumab, which was referred to in a recent blog ( click here) , illustrates the noise and fury that a case of an untreated aHUS patients can cause.
The result is that aHUS patients now have the eligibility rights that those with PNH in British Columbia, treated with the same drug, have had since 2010.
The emotions it raised to attract the media, provided a spotlight on the person, or groups, who make life or death decisions about people.
It was not the first incident in the world; and it is unlikely to be the last.
Another petition going the rounds also seeks priority for another aHUS patient, and others, affected by aHUS in British Columbia. Some of those supporting the petition have given reasons for signing it. Those reasons varied and ranged from a personal connection with the patient ,through own experience of the disease and its treatment ,to broader human rights/ justice. Some also addressed the issue that there is clinical evidence to support its use, which has been a key stumbling block.
All good reasons,so why has the health care provider been so obstructive?
British Columbians are entitled to healthcare under the Province’s Medical Services Plan, which is a social insurance funded health service paid for by premiums based on what an household’s income is. It has an additional level of cover called Pharmacare to recover the costs of eligible drugs and protect against “potential catastrophic drug costs”. Eculizumab would be a catastrophic cost for a household to bear. The key word however is “eligible”.
To be eligible drugs have to be reviewed and evaluated by committees set up to do that.
A committee did it for eculizumab for aHUS and this is what they concluded:
Drug coverage decision(i.e. Non Benefit) is consistent with the Drug Benefit Council recommendation.
The clinical efficacy review did not identify any randomized controlled trials to be included in the review.
The clinical efficacy review included three uncontrolled trials. These studies had several important limitations including: no control group (eculizumab was not compared to anything), short duration of trials and no data on clinically important outcomes.
As a result of these limitations, it is unclear what advantages and disadvantages eculizumab has compared to other standard means of treating aHUS.
With unclear comparative clinical advantages and very high per-patient costs (about $730,000 in the first year and $700,000 in subsequent years), eculizumab is likely not cost effective and would result in a significant budget impact
Click here for full statement
That is a rational decision based on evidence, or lack of it, and with no emotion, it was done in 2013.
The first point about no randomised control studies with a placebo, has been a handicap for aHUS patients where ever eculizumab has been evaluated like this. Although they are a “norm”, such trials are not compulsory to do ; in fact it can be unethical to do it where death and significant harm can result using a placebo ;but without it , it leaves an opportunity to conclude that those who improved would have done so anyway without treatment.
There were 100 aHUS patients included in the trials for testing eculizumab and all got eculizumab. The alternative would have been that 50 of them would have got a placebo, to prove the end point that those with eculizumab would not have died, or moved on to chronic kidney failure, in the 26 weeks of the trial.
Eculizumab for PNH was also evaluated by these committees and gained a “Non benefit- on case by case basis” recommendation ,which meant that ,for some individuals ,access would be possible as a “special authority ” on an exceptional basis. The PNH trials were randomised with placebo. All in the trial could get a blood transfusion , if ,and when, the normal triggering point was reached at any time in the 26 week trial . So there was no difference in standard treatment. The worst outcome would be increased anaemia , although the risk of a catastrophic thrombosis existed anyway with standard treatment.
Those with aHUS would have had thrombotic microangiopathy and most likely acute kidney failure to enter a trial. Their risk was present and evident, would it have been ethical to have just received a placebo in such crcumstances. The committee could have given some leeway ,but chose not to ,and the remaining reasons flowed from that.
It is as it is. There can be no going back.
The PNH decision was made in 2010 .
Other than the randomised trial, all else was the same. The 26 week trial period , 5 years outcome data and the price.
There are around 20 PNH patients in British Columbia and about 10 with aHUS. The dose levels for PNH are 75% of the full dose for aHUS, although the dose mix level for aHUS is less than for PNH in reality because of the number of children with the disease receiving lower doses.
Although benefiting from the same treatment the disease courses are different, it is unlikley that those with PNH will spontaneously go into remission, in aHUS some patients can and do; and more could and would with eculizumab prescribed when needed for as log as is needed.
In either case the budget impact is regarded as significant; not because of the number of patients ,but the perceived cost of treating one person. A classic rare disease scenario. The same number of dollars is affordable if it benefits those with more common illnesses. Inequity .
The budget is the budget. Affordable means affordable. Cost effectiveness is cost effectiveness.
The spot light of the publicity has created some movement – aHUS patients can ,like PNH patients have since 2010, be considered for a special authority on a case by case basis. It is not universal coverage, but it is progress from no chance at all.
Now the issue becomes what is “exceptional” on a case by case basis.
Movement to fully eligible status for all with aHUS within Pharmacare scope will need more persuasive evidence to show that the impact on its $2 billion drugs budget will not be as significant as claimed.
This could be helped by some movement by Alexion Canada on its absurd list price in Canada.
Also the cost of the average patient is much less that the “full dose list price”; and there are number of clinically acceptable and safe ways of achieving that. Personalising the treatment for some in the short term, can prevent unnecessary millions of dollars being spent on alternative treatments for them in the long term.
It is no longer 2013. The reasons for the decision are no longer rational.
What can be learned from British Columbia can be learned elsewhere too. What happens in BC does not have to stay in BC.