NICE and the other children like Isita

What with Isita getting pneumonia on the hottest day of the year so far and spending the first half of last week at St Mary’s Paddington, and then once she was out taking her and Jamie to Legoland on the weekend, I haven’t had much time to write about NICE’s decision not to fund immunotherapy for high-risk neuroblastoma. This does not affect Isita as she had already started, but it does affect all the children who need the drug but who haven’t started yet. I don’t know how many there are but by the law of averages there must already be about 40 in this country diagnosed since last summer, the approximate cut-off date. It is tragic that they might not get access to this vital treatment.

NICE issued the result of its appraisal of dinutuximab beta on 4 May. A lot of the people who are now more affected by this than we are have protested. Solving Kids’ Cancer – which supports families of children with neuroblastoma – has issued a statement with some other charities. Channel 4 News’ Victoria Macdonald has conducted an extremely moving interview with one family involved.

NICE said that the “plausible estimate” of the cost of the treatment (£62,300-£79,900 per Quality-adjusted life year –QALY – gained) was “much higher than what NICE normally considers a cost-effective use of NHS resources.”  QALYs are the currency of NICE evaluations. Basically, for dinutuximab there isn’t enough evidence of long-term benefit, as the cost is divided by too few years. NICE want to see ten years of benefit.

One of the reasons that this cannot be demonstrated is that the drug is too new. NICE has recognised this – partly – and said that “given the promising clinical benefit shown by dinutuximab beta so far in the trial data and the potential for longer-term data within two to three years… a period of time in the Cancer Drugs Fund (CDF) would be the best opportunity for data collection to address the clinical uncertainties.”

So hooray? Not necessarily. CDF funding would allow doctors to make individual funding requests on a patient-by-patient basis and the NHS would pay so the drug could be trialled over a longer period. But unfortunately, NICE goes on to add that the drug is too expensive even for this.

It has therefore invited the company to make a proposal. In other words, to drop it price. It has come down to a plain haggle. The discussion is no longer about medicine; it is all about money. Can EUSA Pharma, the manufacturer of dinutuximab trim its margins enough to squeeze into NICE’s model? Can NICE be persuaded to widen its criteria so that a hundred or so children don’t fall into a dreadful gap just because they are being treated here in the UK and not in France, Germany, Norway or Spain? The final decision will be issued on 12 June. Let’s not abandon these young lives.

One thought on “NICE and the other children like Isita

  1. You are as always informative and to the point. I’m afraid “Great Pharma” is always about money.
    A Chinese doctor once put me metaphorically in my place by saying:” I am not in the business of making money. I am in the business of making you well.”
    V xxxx and love to you all.


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