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A UK website about atypical Haemolytic Uraemic Syndrome (aHUS)



When aHUSUK was informed that the evaluation of eculizumab would be referred to NICE it was because the Health Minister wanted more information about the affordability of the drug that AGNSS had recommended for the treatment of aHUS (and PNH previously). Subsequent announcements and letters from Ministers and the Department of Health still referred to its cost effectiveness, value for money and even “was it a wise use of resources?”.

All these terms are vague and mean different things, so it is no wonder that people get confused, including those who use them. A recent BBC news item about the drug Kadcyla which adds six months of life on average to women dying with an aggressive form of breast cancer illustrates the confusion. NICE criticised makers Roche for not setting an affordable price, in its updated draft guidance. The drug costs £90,000 per patient but Roche said it had offered a lower – undisclosed – price in recent talks .The two organisations have been in negotiations* since the first draft guidance from NICE (the National Institute for Health and Care Excellence), rejecting the drug, was published in April.

“Although Roche proposed a discount to the full list price of Kadcyla, it made little difference to its value for money”- Sir Andrew Dillon, NICE

Kadcyla would be used to treat breast cancer for 14 months compared to just 8 months with the other drugs and, in accounting terms, would cost the NHS budget around £90,000 gross ( i.e. before savings of the budget cost of the alternative treatments)  per patient for that period ( i.e. the number of doses needed in that period multiplied  by the actual price of a dose).

NICE rejected the drug on grounds of cost effectiveness compared to other existing drug treatments for breast cancer. To be cost effective Kadcyla would have to be less than the NICE benchmark of £30,000 per QALY** (or £60000 per QALY for an end of life drug) but NICE found it to be £166,000 per QALY. All other things being equal it would require a list price of around £15,000  to £30000 for Kadcyla  to be cost effective in economic terms. Whether Roche can supply Kadcyla at that price in a safe and sustainable way is for them to answer in accounting terms.

Highly Specialised Technologies do not get evaluated using the economic cost effectiveness methodology but affordability has to be evaluated somehow. To illustrate their concern about the scale of resource required over the next five years, NICE calculated a sum and asked could the NHS afford to spend that level of resource. That NICE forecast can be shown to far exceed what will actually need to be afforded over that period in accounting terms.

However, even for that considerable  lower level of NHS budget resource to be affordable in economic terms ,NICE have to assess which  NHS resource currently used for other treatments would need to be given up to permit eculizumab for aHUS to be available and affordable. How practical and how extensive that search for other treatments to be given up goes, is very uncertain but this, in  economics  theory, will produce what  is known as the opportunity cost of eculizumab.

This is not going to be easy to do. The methodology is only just beginning to be developed and is quite crude. It is too easy say that the choice is between eculizumab for PNH or aHUS, or between aHUS and Cystic Fibrosis; or between aHUS and repeat abortions; or between aHUS and vanity surgery; or between aHUS and  treating non paying uninsured foreign visitors; or between aHUS or any condition which has resulted from avoidable lifestyle choices. The media is full of stories about such comparisons.

aHUS is a horrific disease for those who have faced it, and for those who survive a life on dialysis does not  compare favourably with having working kidneys. People with aHUS would no more wish to suffer from PNH or Cystic Fibrosis than those with PNH or Cystic Fibrosis, if they knew about it, would wish have aHUS. However, those with PNH or Cystic Fibrosis would argue vehemently that they should be preferred to aHUS patients and not give up the funds that  they have been given under different appraisal processes to the one that aHUS patients have, and will continue to face; even though eculizumab for aHUS compares more favourably than eculizumab for PNH or kalydeco for cystic fibrosis.

Similarly, women would not wish to give up their right to choose again and again to abort  a life with health resource that could save an aHUS baby’s life.  As those needing vanity surgery to boost their egos and earning capabilities, see the NHS resources used on them as something that should not be given up to avoid people living their life around a dialysis machine and having to accept progressive scarring to their bodies just to stay alive.

Cancer too is horrific and only those in the position appreciate the worth of an extra few months of life.  How can that be compared to a child with aHUS controlled by eculizumab,  who could expect a normal life span?

“It’s impossible to put a price on life’s precious moments. But it’s not impossible to put a fair price on drugs: Sally Greenbrook, Breakthrough Breast Cancer”

Eculizumab gives very high clinical value, and, if the methodology was available to demonstrate it, high societal value for money too but it comes at a high price, although slightly less now that the Health Minister has negotiated a new Prescription Pricing Regulatory Scheme (PPRS) in 2014 ;  but  that is  just an accounting matter.

When does it become possible to have a fair price in not only accounting and economic terms but in humane terms too for aHUS patients to finally be told that an effective treatment will be theirs in a safe and sustainable way when they need it for as long as they need it?



* NICE does  not negotiate the price of a drug or therapy, nor does the NHS , it is the Department of Health which does so . This is done primarily through the PPRS in which the supplier can list their drug at a price they chose, but in doing so have to supply supporting information which demonstrates that the elements ,e.g.  production cost  or spend on research , which make up their prices ,are within acceptable” tolerances” to the Department of Health . There is also an opportunity for the supplier to offer to enter a patient access scheme ,while their drug is being evaluated by NICE,  and this can include simple or complex adjustments to the price to improve cost effectiveness. PPRS now includes a suppliers’ rebate process where each quarter members of the scheme return a percentage of their sales to the Department of health who in turn hand over the additional funds  over to the NHS ( and not just NHS England) but this does seem to have an effect on cost effectiveness in NICE’s opinion.   

**QALY stands for quality adjusted life year i.e. how many additional years of life will result from using the proposed treatment compared with the alternative and the difference it would make to the quality of a patient’s health in those additional years( measured on a scale of between 0 and 1).   The result of multiplying additional years with the difference in quality of health is divided in into net cost of treatments to produce a “cost per qaly”.  Think of QALY as a Mars bar ,or a Rolls Royce and what NICE is calculating is the unit cost of a product  which in this case is  a “health product” rather than a common confectionery or luxurious car .

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