Is it OK to spend $2 billion plus on aHUS patients?


Imagine yourself as an aHUS patient and advocate for other aHUS patients in your country and you have been asked to appear on a live day time TV show discussing current affairs.

The context of your appearance is that the day before your country’s health organisation had approved eculizumab for all its aHUS patients who need it. You will be invited in to the show by telephone.

The show goes out live and when you are at work so you find a quiet room. a storage cupboard will do, and call the number you have been given.

The programme format is there a host and three panel members who give their opinion and then debate what is likely to be a controversial topic.

The host has already asked the panel “is it ok that the health organisation has agreed to pay $2 billion to treat a few hundred patients with aHUS”?.

The panellists in turn give their views. All were not in favour of doing it.

Their arguments were that far more patients could be treated with that much money than a few with a rare disease.

Cancer is worse and should be the priority for health spend.

And Pharmaceutical companies charge too high a price for their drugs.

No argument in favour of treating you, you are just deemed not to be worth it.

And then you join the discussion without knowing what has been said.

What do you say to get the panel , the studio audience and probably most of those watching to change their opinion?

You have to introduce yourself and tell them what aHUS is and that it kills or leads to kidney failure and a life on dialysis You had been on dialysis for 16 years thinking there was no chance of a kidney transplant because of aHUS. One transplant had failed because aHUS returned without eculizumab.

One of the panel, a well known TV actor, says in his role as a medic in a hospital drama series says he had learned about dialysis for a storyline. He understood how difficult that might have been and would not want to see someone living on dialysis for ever.

So one of the panel becomes sympathetic.

But the other panellists doubled down on cancer sufferers being denied treatment when you were getting too much health resource spent on you. Don’t you think cancer patients deserve to be treated?

Yes you say if there was a drug for them which improved their quality of life, they should get it too. The health service could afford it. After nearly 20 years experience of being a patient and being in hospital you have seen example of waste of resources which if avoided could be used for care.

One of your attackers agrees that there is a lot of waste in healthcare and out of the blue gives an example of a hospital spending $150,000 on a statue for its grounds, instead of care.

The other says that too much is spend on hospital management and not enough on frontline care.

Then you are asked to justify the prices that drug companies charge. You cannot and say so. (Neither could the drug company). You say the prices are very high but the drugs are only for a small number of patients. It will be too difficult to explain health economics simply and quickly. You have no idea of what the $2 billion figure related too. But you say that the drug has allowed you to have a kidney transplant, come off dialysis and all its complications and restrictions on diet and your freedom. You are working full time and giving something back.

By now the panel having got to know you, and are recognising you as a person. You acknowledged your good fortune and wish it for others with rare diseases or cancers.

They all wish you well.

Below is an article written by the now closed down aHUS charity in the UK, aHUSUK, about the excellent news for aHUS patient and the reception it was given in the media. It says what could not be said in a TV show slot.

It’s The Right Stuff

Posted on January 30, 2015 by aHUSUK • 0 Comments

So that is it, the NICE announcement caused a buzz of interest , aHUSUK’s website and Facebook/Twitter were very active and a number of supportive/sympathetic appearances on radio, TV and newspapers, involving Chair Emma, followed too.

Eculizumab is now a treatment in England, eculizumab  trialists will switch from compassionate provision by Alexion to NHS England funding .So all are now within scope.

It is time to reflect on the reaction to a news story which aHUSUK and its members have been part of for a number of years.

The focus of commentators has been of the price of drug, it is a very high price, all figures quoted are about a list price for the highest dose , but for those in the know and who look at what NICE really says in its guidance find that it does not have to be a high cost.

Certainly not the £1.8 billion mentioned on The Wright Stuff

  • Firstly roughly only 2/3rds of patients will be on the highest dose and 1/3rd will be on an average of the cost of the lower doses, which brings the  overall average cost down by as much as 25%.
  • Secondly there is great uncertainty about aHUS patient numbers, the number 200 is mentioned but by late last year only just over 50 were receiving  Eculizumab. Indeed if  past forecasts of patients needing eculizumab by 2015 had been correct  there should be more than three times  the number on eculizumab than there actually are , so concerns about the budgets are vastly overblown.
  • Thirdly NICE has said it is not over until its over, and has asked that NHS England and Alexion to continue to explore  ways to reduce cost further and Alexion are already returning a rebate on their sales to the DOH  through the Prescription Pricing Regulatory Scheme 2014 which by next year will effectively be a 10% discount on list.
  • Fourthly the research that NICE call for will bring about tailor made doses and intervals and where it is safe to do so ,patients will be able to exit from treatment. 3 week instead of 2 week intervals if sufficient drug remains to inhibit complement by the time the next dose is due reduces cost by 33%
  • Fifthly all prices and costs are just for Eculizumab and the savings from the alternative woefully ineffective  treatments are ignored.

So below the headlines the real situation is that Eculizumab is, and will be more affordable than it appears on the surface.

Something those ill-informed critics of the high cost of rare disease therapies may want to ponder before jumping to erroneous conclusions.

What came across very strongly was that eculizumab is very clinically effective, as it has been for treating PNH for several years now.

Although those sharing the same drug as aHUS patients may not be well known, nor visible in supporting aHUS patients claim in how safe and effective eculizimab is at blocking complement ,PNH patients have been benefitting  from access to Eculizumab during the four years that  aHUS  has been  through the AGNSS, CPAG and NICE  evaluations, and in doing so give testament to the drugs clinical effectiveness and lack of side effects. Indeed because PNH is usually later in onset  than for aHUS, Eculizumab for aHUS produces substantially more quality life years than it does for PNH.

A baby onsetting with aHUS today and receiving  eculizumab  today can look forward to a normal life span.

Commentators  frequently said how much more clinically effective Eculizumab was than many much more common therapies which cost a lot but deliver so very  little clinical benefit.

But the question remained about how could the NHS afford something  new like Eculizumab, however much more clinical benefit it gives to those at the margin of the margin of the health service because they are so rare, when the NHS funds were finite and other treatments for many more people would have to be given up. Switching between two aspects of finance (budgets) and economics (opportunity cost) in one go.

Inevitably the announcement linked the NICE decision to recent decisions about cancer drugs, and the very emotive issue of needing drugs which give a few precious  additional weeks of life. Funding treatment of cancer has produced enormous clinical benefits , more live now after cancer than now die from it. It is a wise  use of NHS resource. But why  give the impression it is just a choice between treating aHUS and treating cancer? Why also should the opportunity cost comparison be limited to other specialised rare disease like PNH or Cystic Fibrosis, because why should their treatment be given up, because Eculizumab for aHUS is much more clinically and  cost effective than treatment of PNH and Cystic Fibrosis patients?

The opportunity cost search has got to be NHS wide , and if each NHS  pound was as  rigorously evaluated as the pounds for aHUS  have been ,a different picture would emerge , and it would no longer be down to a  hypothetical choice  between cancer and rare diseases as billions of pounds of ineffective waste would be found.

Indeed on the day of the NICE announcement about aHUS there was another NICE announcement about asthma. In that NICE says that about 1.8million  of 5.4 million asthma sufferers in UK are getting treatment they do not need; and, as over £1billion is spent on treating asthma patients each year, that is quite a lot  of resource being used ineffectively and only a fraction of several billions ( at £300 million per year*) from that which could be saved over 25 years as a result ,would be needed to fund (£30 million per year) one of the most effective clinical therapies there is.

So, in more ways than it appears on the surface, it is clear  the NICE decision about Eculizumab for aHUS is The Right Stuff.

*What did the asthma patient organisation say about the potentially £300 million saving. Give it to cancer patients. No what ever is saved should be reinvested in the care for other asthma patients.

Article No. 655

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