NHS Turns Down Free Lifesaving Drug

A cystic fibrosis patient has been denied access to a compassionate use program because it's temporary. Why?

Cystic Fibrosis, a genetic malady, dooms its sufferers to a short and burdened life. It's longer than it used to be--CF sufferers used to expect to die in their teens, but now the average life expectancy is late thirties. Unfortunately, that's still tragically too short.

But now there's some hope, at least for sufferers with a version known as a G551D mutation--about 5% of CF sufferers. The mutation causes the body to produce a malfunctioning version of a protein that transports salt and fluids in and out of lung cells, so that the lungs become clogged and eventually damaged by thick, sticky mucus.

A new drug called Kalydeco can actually help that protein do it's work; it's the first treatment for CF that actually addresses the underlying problem, rather than focusing on therapies that fight infection and help patients break up and expel that sludge-like mucus. (For more on CF treatment, I recommend this Atul Gawande piece from the New Yorker.)

It's already been approved in the US. It's also been approved in Britain--but only by the drug regulators, not by the NHS, which means that unless you have private health insurance, you have no way to pay the six-figure price tag. NICE, the agency that does cost-benefit analysis for the NHS is still considering its verdict. The maker of the drug has offered to make it available for free on a temporary compassionate basis, until the NHS decides whether or not to pay for it. But at least one local health authority is turning down their offer.

Caroline’s father, Neil, 72, said: "Caroline has struggled for 29 years and it has been horrendous at times, but the last year has been the worst.

"She has always been a good girl, but she is very low now. She knows that she's dying.

"She has been given the golden crown, then had it snatched from her.

"The NHS Trust is sitting by while we are watching Caroline's health deteriorate at an alarming rate. How much longer can this go on?

"To have this one chance of life taken away after a lifetime of suffering is cruel beyond belief."

Jessica Nickless, vice-chairman the Ivacaftor Patients Interest Group, said: "If I stood by and witnessed someone being murdered I would be complicit to that murder, yet doctors can watch someone die. They are condemning Caroline to a slow, lingering death.

"For Caroline, it is now or never. We have almost exhausted every avenue."

A spokesman for Heartlands Hospital said: "We fully support our patients and are doing everything in our power to facilitate a fair solution.

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"The trust decided not to obtain Kalydeco on a compassionate basis because the drug company scheme is temporary and the trust would face withdrawing the medication when the scheme stops."

Sarah Palin took a lot of chaff for her silly "Death panel" remark, but in the case of small diseases like this, that's what these sorts of cost-benefit decisions effectively become. There are only 270 patients in Britain with this CF variant; the NHS knows all the names of the patients who will be sentenced to death if they withhold this treatment.

And at first blush, this particular decision seems incomprehensible. Why would you refuse to administer a free drug that could save this woman's life? Yes, there's probably some cost to administer it, but I doubt that cost exceeds that of traditional treatments.

But that last sentence explains it. The drug costs hundreds of thousands of dollars a year. The health authority thinks there's a good chance that after NICE hands down its verdict, they will decide that the cost is not worth it.

Indeed, it seems to me quite probable that they will say no. NICE uses something called a "quality adjusted life year" (QALY) to measure whether a treatment is worth the cost. Their general rule, as I understand it, is to approve any treatment that costs less than $50,000-$65,000 per QALY. Since this drug has to be administered for the rest of the patient's life, it's hard to see how it's going to pass cost-benefit analysis, even if you assume that the price will eventually fall. (Usual disclaimer: I'm not an expert on the British health care system, and there may be complicating factors I don't know about. But the health authority's behavior suggests that they agree with me.)

The moral calculus initially seems straightforward: give her some extra time now, even if the drug has to be discontinued later. But that's not how human psychology works. Right now, a health authority is telling a very sick patient that a treatment they would like is simply not on the formulary. That's bad--but it's not as bad as telling a healthy patient that they are going to discontinue a drug that's working, and let them sicken and die. One is passive, the other feels more active. If they allowed this woman to start the drug, it seems highly probable that they would never be able to stop--cost/benefit be damned. And given how tight NHS budgets are, that means some other patient would go without.

This is a phenomenon that psychologists call "loss aversion": we react more strongly to losing something we had than we do to never getting it in the first place. And that may well be why the drug's manufacturer is offering to make this very expensive treatment available for free; presumably they know how hard it would be for the hospital to stop treatment once it starts.

The health authority's reasoning, by the way, points out why it is so unendurably inane to say that we need ObamaCare because after all, other countries spend so much less than we do. The implication is that ObamaCare will somehow deliver those lower prices. It won't, and this is why: it is difficult to deny treatment, but nearly impossible to discontinue it. As Noah Millman pointed out a while back, American health care costs are not exploding now--at least, not relative to other countries. They exploded in the 1980s. Our current rate of health care cost growth is about average. But we're growing off a much higher base.

In my humble opinion, any attempt to roll back that level--or even ratchet down the rate of growth so that we are eventually even with other countries--is doomed to fail in the near-to-mid term. Had we never given Americans endless access to specialists, high-paying jobs in the health care system, private hospital rooms, and so forth, the health care sytem could deny those things. But now that we have them, there is no way to force specialists to become primary care physicians, slash nursing salaries by a third, and stick the patients back on open wards. The doctors have no experience as PCPs, the nurses have mortgages pegged to their current earning power, and the hospitals have been built around private rooms. Even if you wanted to restore the open wards (which are much cheaper to operate not only because of space, but also because you can monitor more patients with fewer nurses), doing so would cost a fortune and disrupt the operation of the hospital. In some layouts, I'm told that it would simply be functionally impossible.

We may be able to actually cut spending if there's a crisis. But short of that, I doubt we will. Some of the decisions--like the wards--are simply impossible. The rest are so politically unpalatable that we will find it functionally impossible, short of a crisis in which there's no choice. Had we behaved more like the NHS, we might be able to deny the amenities and treatments that American patients have gotten used to. But even the NHS knows that once you've given in, it's terribly hard to take them away.

Update: and not so easy to deny them, either; after newspaper story, the NHS caved.