Orphan Drugs: Costs

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Orphan Drugs: Costs

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Күн бұрын

Last week, we argued that the benefits of the Orphan Drug Act are more elusive than commonly assumed. But what are its costs? That's the topic of this week's Healthcare Triage.
Sources can be found in the posts friend-of-the-show Nick Bagley has written on the subject: theincidentalec...
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Stan Muller -- Director, Producer
Aaron Carroll -- Writer
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Пікірлер: 30

@davcar94 7 жыл бұрын

Really enjoying the series on orphan drugs. Thank you Dr. Carroll + crew!

@kujmous 7 жыл бұрын

Why is this channel not mandatory viewing for Congress?

@RobertoGonzalez-gg3jc 7 жыл бұрын

Hi Dr. Carroll, this is certainly a naïve question, but isn't the very small size of the market already a big protection for the companies? I mean, for drugs aimed at rare diseases, how much competition will they face really? Congrats for the great show, I have shared lots of its contents with health policy researchers here in Brazil.

@rufioh 7 жыл бұрын

Why can a drug get orphan drug status even though they were already in production for decades without it, like 3,4-DAP that you mentioned?

@PissedOffGhost 7 жыл бұрын

it was prescribed off-label before. for example a drug gets approved by the fda to treat stomach pain, turns out that drug also treats headaches, so a doctor prescribes it for patients with headaches, even though the fda didn't approve it for headaches.

@bradwilliams7198 7 жыл бұрын

If you look up the history on Wikipedia, 3,4-DAP was never approved as a drug. It existed as a chemical compound (you can buy it, but it may not be sufficiently pure for human consumption). My guess is that if this situation persists, some future Breaking Bad chemists will figure out that it's much more profitable to make something like 3,4-DAP than meth.

@Innerparty101 7 жыл бұрын

3,4-DAP could have had activating or deactivating groups attached to it, increasing the potency of its effect(s). Drug manufacturers could use this to argue that they have the exclusive right to the new, altered form. For example, right now scientists have been taking Donepezil (an Acetylcholinesterase inhibitor) and adding groups such as nitrogen and chlorine to the chemical structure. Preliminary testing has shown that these changes have caused increased uptake/effectiveness of the drug, which is probably what's going on here.

@bradwilliams7198 7 жыл бұрын

Definitely, looking at structural analogs to find one that is more effective, has less side effects, or a more favorable metabolism/activity profile is a legitimate invention, as it yields something better than the original compound. But they didn't do that in this case. They just started producing a medical grade product, and went through the FDA paperwork...and for that they get to charge 1000x the price.

@Praisethesunson 7 жыл бұрын

It's clearly giving a massive windfall of profits of major pharmaceutical corporations and maybe not so clearly "helping" (assuming trading all their money for a little extra life is considered helpful) a small group of sick people.

@brendansully12 7 жыл бұрын

In the case where they only offer a marginal improvement over existing drugs, why can't the patients just take the existing drugs? I'm more than willing to learn about specific abuses and look forward to the upcoming episode, but I really feel you're only looking at one side of the story.

@elshaddai741 7 жыл бұрын

It would be interesting to know how much of the 1 billion dollars is reinvested into new drugs and recycled to make widespread drugs cheaper. 1 billion is without a doubt an unjustifiable price tag, but the optimist in me is hoping it might have enabled some good that otherwise might not have come about. I am, however, doubtful!

@CaptmagiKono 7 жыл бұрын

Yeah, I am more on the pessimistic side and assume any money that is left over is put directly into somebodies bank account.

@Edgewalker001 7 жыл бұрын

Every time I hear about things like this it just makes me more sad. The truth is that a lot of research that could be directed towards curing disease is instead directed towards long term treatments for rare diseases or giving people that were already going to die anyway a little more time, simply because it's more cost effective. *sigh*

@TheGuitarguy377 7 жыл бұрын

Well, everyone's already going to die. If treating a disease is more cost-effective than curing a disease in terms of the years gained, shouldn't we favor it?

@michellep1555 2 жыл бұрын

yep, they can cure it once. but to make more money, they just want people to use it for life. like soliris. they ask patient to forced their gov to pay half a million per year per patient. so wrong in so many leave

@kriss12loverap 7 жыл бұрын

No comments?

@cm3655 7 жыл бұрын

Anybody know if these rare diseases are mostly genetic diseases that can be tested from birth?

@vitamindubya 7 жыл бұрын

1:58 I don't believe you!!! EU is perfect and not evil America.

@katiefrank7351 7 жыл бұрын

the NHS was already crippled by the Tories, they just don't have the funding to spend such a rediculous amount on a drug when they'll get back less than £10 from the patient for each prescription.