Has the NHS put the CAR-T before the horse?

CAR-T is going to be made available on the NHS for children who have an advanced form of a blood cancer, acute lymphoblastic lymphoma, that has not responded to other treatments. The BBC reports that an estimated 15 children a year will be eligible to receive the treatment, which works by extracting, editing and re-purposing the child’s own immune system. This treatment is exceptionally expensive, both for the drugs themselves and for the extra care patients require as a result. The treatment has been reported as costing upward of $300,000, with care costs topping $1 million in the USA. While care costs are usually substantially lower in the NHS, this will still be a very expensive treatment.

So if it is being made available on the NHS, does that mean it’s cost-effective? In a word, maybe. Realistically, probably not. Under the health economic model of cost-effectiveness, if the NHS funds treatments that are not cost-effective, someone else loses more health than the patient receiving the new treatment receives (to get an idea of what this looks like in practice, read Chris Gibbon’s brilliant blog post on a highly cost-effective intervention named Neil). To counter this, the NHS uses NICE to review new drugs and decide if they are cost-effective. If not, they aren’t funded. In this case, though, we just don’t know yet, because that evaluation hasn’t been done.

Why don’t we know if CAR-T is cost-effective?
In practice, the decision making process involves a lot more nuance than just cost-effectiveness. People feel very strongly that new drugs should be made available, and patient groups have increasingly loud voices in the health policy arena. The people who could benefit from new drugs are aware they exist and can see exactly what they’re losing by not receiving them. Conversely, patients who will lose out through funding being redirected aren’t aware of it. Disinvestment is usually a quiet business - local authorities don’t tend to shout about services they are cutting. The political consequence of this is that there is a constant upward pressure on cost-effectiveness decisions, as decision makers face strong condemnation for rejecting drugs. While decision makers like NICE have a well-defined role that insulates them from the worst of the political pressure, politicians and governments are more exposed. Around the world, the pressure to approve and make new drugs available is mounting. In the US, for example, the FDA is introducing approval processes based on earlier and earlier trials. In the UK, this pressure manifested in the introduction of the Cancer Drugs Fund (CDF) in 2010.

The CDF provided an alternative way for cancer drugs to be made available on the NHS. Doctors could request drugs intended to extend the life of cancer patients, regardless of their cost-effectiveness. The consequences of this were rapid and unsurprising: the allocated budget ballooned, and by 2016 £1.3 billion of the NHS budget had been reallocated to the CDF, well beyond the original £50 million annual budget. More alarmingly, most drugs funded through the CDF didn’t do anything to extend patients’ lives at all. One review of CDF drugs found that only 18% had been shown to increase overall survival at all, and then by only 3 months on average.

In response to this, in 2016 the CDF was relaunched in a more constrained capacity. Now drugs had to demonstrate “plausible potential” to be cost-effective. Evidence on patient outcomes from their use in the NHS had to be collected, and the drugs would be re-evaluated after two years, when a final approval decision would be taken.

It remains to be seen how drugs in this new CDF will perform, but while there are constraints, that phrase “plausible potential” still leaves it pretty much a free-for-all. The bar is low and fuzzy and you only need one good (lucky) study to clear it. This is the standard that CAR-T has met so far.

The Fight
Back in April, the Chief Executive of the NHS, Simon Stevens, told the ABPI, the British Pharma industry body, that CAR-T could be available on the NHS by the end of the year. He qualified this by saying that Pharma needed to set “fair” prices for this to happen, but so far there is no evidence that this has happened. CAR-T is in the CDF, albeit for a very small population. If it turns out to be cost-effective, in two years time we will find that out and it will be funded routinely, to the benefit of these child cancer patients and their families. If it isn’t then the NHS will have wasted tens of millions of pounds and cost patients across the NHS their health. If that happens, the process should be to defund CAR-T. But will that happen? Patients will have had two years of access and Pharma will have spent two more years lobbying. Simon Stevens has already told industry he wants these drugs to be available, without knowing they work yet. My bet is that CAR-T won’t be cost-effective (so few of these drugs are) and that decision makers will have a fight on their hands to get that money allocated where it will do most good.

Let’s hope I’m wrong. See you back here in two years.


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