Rare disease dilemma
The Herald has a story about a new treatment for a very rare blood disorder, and the fact that Pharmac isn’t funding it.
The drug, eculizumab (brand name Soliris), is currently the world’s most expensive, at about NZ$500 000 per year. It’s also very effective. There’s starting to be a lot of this: we now have the technology to develop specific treatments for a wider range of rare diseases, and most of the rest of that ‘most expensive’ list are replacement enzymes for rare deficiency disorders. Another recent example is ivacaftor (brand name Kalydeco), which, in about 5% of cases of cystic fibrosis allows the defective chloride transporter protein to work normally. The result appears to be complete control of the disease, but at a cost of US$300 000 per year. Similar drugs for other variants of cystic fibrosis are being tested.
Funding any one of these drugs would be a minor total cost for Pharmac, because each rare disease is rare. There are only about eight people in New Zealand who would take eculizumab, which would cost only 0.5% of Pharmac’s budget; there would be about 25 who could take ivacaftor, adding up to a percent or two of the budget. The difficulty is that rare diseases collectively are not rare — the European Organization for Rare Diseases estimates that 6-8% of the European Union population have a rare disease and applying that figure to the NZ population still gives 270 000 people. At $500 000 per person, Pharmac’s total budget would pay for 1500 people to get this sort of very expensive treatment. At the moment there probably aren’t 1500 people in NZ whose rare diseases are expensively treatable, but there are a lot more than eight.
The patient support group for people with this rare blood disorder obviously think the treatment should be funded
The group’s founder, Auckland artist Daniel Webby, 32 – who almost died from PNH complications – said the funding process did not recognise the rights of rare-disease sufferers.
“They need to recognise that for rare diseases, [drug] development costs are higher per patient. They need to put that into their budget and make sure people get … life-saving treatments when they are available.”
I’m sure Pharmac does recognise this, but changing the national approach to subsidy of health care to give priority to ‘miracle’ treatments for rare diseases is not the sort of decision Pharmac should be making on its own, and the money shouldn’t be taken out of the current Pharmac budget (which is already on the low side). Kiwis need to decide whether a miracle drug fund is something we want to support and are willing to pay for.
[Update: The Herald has an editorial weighing in strongly against expensive drugs even if effective. I basically agree, but it’s a pity they don’t have the same attitude to miracle treatments that don’t work]
Thomas Lumley (@tslumley) is Professor of Biostatistics at the University of Auckland. His research interests include semiparametric models, survey sampling, statistical computing, foundations of statistics, and whatever methodological problems his medical collaborators come up with. He also blogs at Biased and Inefficient See all posts by Thomas Lumley »
Let me be really heartless here:
Spending that kind of public money on drugs isn’t defensible. Public money is fungible — we can spend it on road safety, swimming lessons, breakfast in schools, house insulation, OR drugs. I realise this is someone’s life we are talking about, but it is often someone’s life we are talking about. This person has a name and a face. The alternative use of the funds would just help a statistical person. I would suggest that we need to be moved by the statistics, not the faces and names.
12 years ago
The costs are fungible; it’s the benefits that aren’t.
In order to make funding decisions you have to reduce the benefits to some common scale. That’s unavoidably true. But the choice of the common scale is neither unique nor morally neutral. A simple cost-effectiveness calculation will equate, say, an extra year of healthy life for one individual with a 10% chance of an extra 0.1 year of healthy life for 100 people. But these don’t have to be treated equally, and many people wouldn’t want to treat them equally.
Personally, I do tend towards the utilitarian risk-neutral cost-effectiveness viewpoint. Also, these drugs are so expensive that I suspect the public consensus after thinking carefully about the issue would be not to fund them. But that’s a choice, not a mathematical deduction.
12 years ago
Wow! That linked table was an eye opener. Sure the R&D and clean protein production is expensive, but hundreds of thousands per year for treatment; Really? How are these prices set? I thought Herceptin was pricy, but compared with these it’s a steal.
Homegrown antibody therapy and novel pharmaceuticals are probably going to be out of reach for the foreseeable future, but I couldn’t help noticing that many of the drugs on that list are purified recombinant enzymes, e.g., #2 Elaprase (Iduronate 2-sulfatase) for Hunter’s @ US$375k; which from the patents has full sequence info available, and appears to be perfectly capable of being expressed in yeast.
With the porous views to IP and HSNO that a life-threatening disease would bring, I have to wonder how long it’ll be before we hear of the first DIYbio/Biopunk efforts.
12 years ago
If you think about the probability of success in developing one of these, and the returns that speculative venture capital investors demand, it’s possible that these drugs wouldn’t be developed if they couldn’t overcharge for them.
The optimistic view is that we’re at a threshold, that development costs (in specific types of simple genetic disease) will keep falling, and that it will eventually be more profitable to charge only $50000 per year and get more customers. And at least for the small-molecule drugs, eventually they will come off patent and the Indians (or whoever it is by then) will come out with generics and knock four zeros off the price.
The pessimistic view is that a lot of new treatments are antibodies for which ‘generic’ (biosimilar) versions will be very hard to define, so each new competitor will need to do new trials at least to Phase II and the price can be kept high indefinitely, even after the patent runs out.
I do like the possibility of bootleg biopunk generics for the enzymes. Where’s Cory Doctorow when you need him?
12 years ago