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GratefulOctopus t1_ixv04rl wrote

Reply to comment by Sariel007 in A novel medication for hemophilia B has just been approved by the Food and Drug Administration. The treatment is a form of gene therapy, intended to replace a dysfunctional gene that leaves people unable to control their bleeding. by Sariel007

Ok so it is mostly just to offset research cost? It's not how much it is to make/administer the drug?

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TheBearWillBeFine t1_ixw3f6f wrote

I work doing safety testing for drugs just like this. Setting aside all the money it takes to develop one of these viral gene therapy drugs, the cost to manufacture a single dose on a lot of them is somewhere around 50-90,000 dollars. The viruses need to be grown, then emptied of their regular genetic material, have the new, specially designed genetics put in, then replicated up to a level that can be infused into a person. Every single one of those steps requires highly skilled people to ensure it happens correctly. Growth of viruses need to happen in cell tissue culture, which also costs a lot for all the materials to do correctly, then all the supplies needed to purify out the virus from all the other crud in manufacturing. Then, once the product is made, it still has to be quality tested, to ensure things like dosage is correct, and that there isn’t anything left that’s contaminating. And all this doesn’t even count the years and years of research and development that goes into getting a virus with the correct components to be effective at treatment but otherwise non damaging to a patient. Drug product patents have to be filed pretty much as soon as a compound is discovered, so well before clinical trials even begin. They’re often only good for 20-25 years. So, if you figure 7-10 years of base research and development, another 3-5 years of clinical trials, getting FDA approval, etc, on average a company only has 3-5 years to make back ALL the money they put into a new drug, plus some profit to keep the doors open, lights on and investors interested in funding the next project.

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FUCKYOUINYOURFACE t1_ixv3d53 wrote

It’s also how many can benefit from the treatment. They have to charge more when the prevalence of the disease is lower.

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cygnoids t1_ixvgx98 wrote

This is the case but it’s also expensive to develop the cells used for AAV based drugs because the cells are killedwhen the virus is made. This, you can’t reuse the same set of cells throughout the process.

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itwasquiteawhileago t1_ixwemjx wrote

This is where orphan drugs come in. The government (US, anyway, but I'm assuming others have similar programs) can help provide some funding and allowances for private companies to work on drugs for conditions that have a low enough prevalence as to make it economically non-viable. Should someone that has a one in a million disease be left to suffer because Big Pharma can't turn a profit? This is a primary example of why and how governments should function: to ensure people aren't left behind. And, who knows what other things may spawn off such research, so it's not like everyone else might not benefit, too.

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Plastic-Bluebird-625 t1_ixv1486 wrote

Correct. It's not about the cost of making it. It's the years of research with tons of doctors is what makes the cost.

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KindlyContribution54 t1_ixvi25z wrote

I saw a bunch of scientists weighing in on this when it was posted elsewhere and it does actually have high ongoing costs custom tailoring it to each patient and expensive materials.

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picking_a_name_ t1_ixw8odn wrote

It's the cost of the research for this drug, plus paying for the fact that only 1 in a 1,000 drug potentials actually make it to market. So they recoup those costs too. Plus using the costs of other medicines you would have had to buy instead.

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Colonelfudgenustard t1_ixvgyb3 wrote

They've got to pay back all those volunteers in the studies. Wait a second . . . the volunteers don't get paid!

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