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Comment by thrance

1 year ago

Which is why there should be no patents in healthcare. Most research is already publicly-funded, production and distribution should be too. We could drown in cheap medicine, instead we artificially increase the prices by 5000% to fatten the pockets of already super-wealthy parasites.

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thrance

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tptacek  1 year ago

Are you thinking this through? It costs billions of dollars to bring a new drug to market --- before sales, admin, and marketing costs --- and most attempts to bring drugs to market fail. In the very best case for the public, the drugs are incredibly cheap to manufacture; that's an achievement for a pharma company. Do you see how the incentives work here?

  • A_D_E_P_T  1 year ago

    > It costs billions of dollars to bring a new drug to market

    Have you ever wondered why this is the case?

    In the "Golden Age" of pharmaceutical development -- from roughly 1930 through 1962 -- it cost orders of magnitude less, adjusted for inflation, was much faster, and outcomes were no worse.

    I'd argue that the new (post-1962) Phases II and III -- drug efficacy studies -- are wholly unnecessary and are indeed unethical.

    First, because you can't even presume to gauge a drug's "efficacy" without huge and exorbitantly expensive trials -- and, even then, trials can be inconclusive.

    Second, because the FDA selectively ignores efficacy data. See, e.g,. flibanserin and certain Alzheimer's drugs that have been approved recently. This was scandalous, but not nearly scandalous enough.

    Third, the statistically sound way to find both rare harms and true-to-life benefits is to watch how a drug behaves in millions of real patients, not in a few thousand volunteers. Modern electronic-health-record feeds, claims databases, and wearable data streams let regulators run near-real-time Bayesian safety signals across populations that dwarf any Phase III cohort; the surveillance network that caught rofecoxib’s cardiovascular signal in 2004 had orders-of-magnitude more patient exposure than the approving trials, and today’s infrastructure is far denser.

    It would literally be 10x faster, 100x cheaper, and 100x easier to grant conditional approval after Phase I and then run postmarketing surveillance. You'd better signal-to-noise on both efficacy and harm at a small fraction of the cost and time. It's downright perverse to outlaw early access to promising investigational drugs, forcing patients to wait 8-12 years, and then foot them with a multibillion-dollar bill for ritualized trials that are unnecessary -- for the same ends could have been attained in far better ways.

    • firejake308  1 year ago

      You have an interesting argument here. It sounds like you're saying that once you've proven safety in Phase I trials, and you can skip to the large, post-market Phase IV studies to do the dose-finding and efficacy-proving that would traditionally be done in smaller Phase II and III trials. As long as the drug is not dangerous, which can be proven by the Phase I trial, there is no need for the masses to wait for slow Phase II/III trials because in the worst-case scenario, the drug is safe but not effective, which is an acceptable outcome. So far, I find this pretty convincing.

      However, I would raise two objections.

      1. Without a Phase III trial that proves effectiveness, payers do not have sufficient motivation to purchase a drug that may or may not work. One solution would be to have the producer of the drug offer the drug for free/cheap until it demonstrates effectiveness, but that becomes a lot more expensive when you have a larger cohort. This is admittedly an implementation detail that could be sorted out, but it's a very important detail that would have to be addressed for this plan to work in the real world. The bigger problem is:

      2. The best way to prove that a drug works is a large, randomized controlled trial. Your proposal is definitely an improvement over current trials in terms of making them larger, which would help to improve statistical power (and also get potentially helpful drugs into the hands of patients, of course). However, without the "ritual" of a Phase III trial, you lose randomization and you lose the placebo control. These are big losses that make statistical interpretation of results very hard because they open up the door to many potential confounders. As you mentioned, Bayesian signals can try to control for some of these confounders, but you can never be confident that you've considered every possible variable, whereas randomized placebos can eliminate almost all confounders.

      So ultimately, I think that Phase III effectiveness trials are necessary in order to prove effectiveness because the statistics with post-market Phase IV trials will never be as "clean" as an RCT. And if we were to delete Phase III trials and skip straight to Phase IV, we would never again be able to confidently say whether or not a drug works. Thus, the ethical cost of making patients wait a few years for the Phase III trials to finish is outweighed by the benefit of being able to say with confidence that the drug works for the rest of eternity.

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  • thrance  1 year ago

    Are you arguing this can't be achieved by the public sector too? Or that pharma companies don't make obscene margins?