Tuesday 5 August 2014

Ebola prizes revisited

So it appears there's an experimental treatment for Ebola that's now shown to be effective in humans. But it's not going to hit the market any time soon. Why? Getting through the FDA's trials is way too expensive relative to the amount that a company could earn from a drug that helps very poor people.

From the Telegraph:
A source close to the Atlanta hospital, where Dr Brantly is being treated, told CNN: "Within an hour of receiving the medication, Brantly's condition was nearly reversed. His breathing improved; the rash over his trunk faded away."
One of his doctors reportedly described the events as "miraculous."
By the next morning, Dr Brantly was able to take a shower on his own before getting on a specially designed Gulfstream air ambulance jet to be evacuated to the United States.
Dr Writebol was also administrated with the drug, which was transported to Liberia in a special sub-zero container.
She showed a less remarkable recovery, but is hoped to travel to the US on Tuesday to continue her treatment.
According to CNN, the drug was developed by the biotech firm Mapp Biopharmaceutical, based in California. The patients were told that this treatment had never been tried before in a human being but had shown promise in small experiments with monkeys.
ZMapp has not been approved for human use, and has not even gone through the clinical trial process, which is standard to prove the safety and efficacy of a medication. The process by which the medication was made available to Dr Brantly and Dr Writebol is highly unusual.
So the drug looks promising. But...
The news comes after health workers said drugs that could fight Ebola are not particularly complicated but pharmaceutical firms see no economic reason to invest in making them because the virus’ few victims are poor Africans.
Outbreaks of the deadly haemorrhagic fever have so far been limited to remote corners of African countries, and have killed a little over 2,400 people since the first infections were identified in 1976 in what was then Zaire.
Developing, trialling and licensing vaccines or treatments would cost significant amounts of money, and drugs companies argue there is not enough demand to justify the outlay.
“These outbreaks affect the poorest communities on the planet. Although they do create incredible upheaval, they are relatively rare events,” said Daniel Bausch, a medical researcher in the US who works on Ebola and other infectious diseases.
If the FDA mandates very expensive processes for drug approvals, expect investment only in drugs that provide a sure-fire moderate return, or a chance at a really high return. Ebola treatment provides neither, so why would anybody throw piles of money away by running it through the FDA trials? There then are two solutions: ensure that there's a return at the end of it by providing a prize for a treatment that makes it through the trials, or ease up the FDA trials for drugs in this kind of category. Does it really make sense to mandate placebo trials for drugs hitting diseases with 60% fatality rates? We are condemning people to a very high risk of death for the sake of ensuring that there aren't drug side effects and that the drugs are more effective than placebos (pretty easy to tell quickly where the fatality rate is otherwise 60%!).

That doesn't stop public health folks for condemning businesses rather than the procedures they've demanded though. I cannot understand how these peoples' brains work:
A lot of that has to do with what Prof John Ashton, Britain’s leading public health doctor, termed the “moral bankruptcy” of profit-driven drugs developers.
“We must...tackle the scandal of the unwillingness of the pharmaceutical industry to invest in research to produce treatments and vaccines, something they refuse to do because the numbers involved are, in their terms, so small and don't justify the investment,” Dr Ashton told The Independent on Sunday.
“This is the moral bankruptcy of capitalism acting in the absence of an ethical and social framework.”
Yup. Demanding that businesses selflessly throw money away is way more effective than changing the regs to bring costs down or putting up public money so that the burden of providing the public good fall on the tax base instead of the pharmaceutical sector. Sadly, about par for the course in public health advocacy. Do these guys have to pass some kind of field exam in hating the market before they get their public health degree?

HT: @epicbeer


  1. Eric,

    There's no requirement for *placebo-controlled* trials. Drugs are routinely approved without them. The requirement is for evidence of effectiveness and safety, which takes more than one person. You could probably get fast-track approval based on a small dose-escalation study and a single-arm efficacy study, especially if the drug is as effective as is being claimed.

    Even if it is, based on the description the drug would only be useful in places with reasonable hospital facilities, since it needs to be given soon after infection and it probably requires continual refrigeration to remain active.

    On the funding issue I agree almost completely, with the same proviso as last time that there are many things more important than a cure for Ebola, even if you live in West Africa.

  2. If that's right, then either those costs remain above the likely earnings, or everything in the story's just a rent-seeking attempt to cause inframarginal payments to come into existence. I wonder what the costs would be of the cheapest possible route through the FDA....

    Agree that there are higher priorities, and that those too should see prizes instead of patents where appropriate.

  3. I wouldn't be surprised if the costs are still too high -- especially as the treatment sounds as if it might have fairly limited applicability.

    The real difficulty in running the efficacy trial would likely be lag. In monkeys, according to the stories, it was much more effective 24 hours after infection than 48 hours after infection, but most people aren't symptomatic even 48 hours after. To get people that early, you'd have to treat a lot of exposed people, many of whom wouldn't have been infected anyway. That means the expected death rate will be a lot lower than 60%, and pretty uncertain, so larger sample sizes would be needed to get convincing evidence.

    If the treatment is highly effective well into the disease, that would make it a lot less expensive to get evidence of efficacy.

  4. It will come to a point the government takes the property rights that it provides companies to incentivise innovation, and removes it in cases of extreme public interest. Aka.. they will appropriate the intectual property and perform the testing/production as a public health service.