The calculus for determining whether an indication is worth pursuing is probably determined more by 1) potential pricing and 2) translati...
The calculus for determining whether an indication is worth pursuing is probably determined more by 1) potential pricing and 2) translational risk than by total number of patients, tax incentives or added exclusivity, although shorter clin development requirements are hugely important If the translational risk is high (i.e. what works in mice / other disease models prob won't work in humans), the probability of success approaches zero. So the expected value approaches zero. It doesn't matter how many patients there are, the magnitude of the unmet need or how nice the molecule is if it isn't effective. With a high enough price, you can get a billion-dollar drug from a few hundred patients. Avexis, which was acquired by Novartis for $8.7B, had a lead drug targeting Type 1 SMA, which has only a few hundred patients worldwide. If they charge $2M / patient (which they might) it only takes 500 patients for $1B annual revenue. Of course they were pursuing other indications, had other development projects and had valuable manufacturing assets so the $9B wasn't all for that one indication, but a lot of it was And while $2M is prob high, the reason they can get away with even tossing out prices like that is that their drug is a potential cure for a disease that kills 90% of affected infants before 2 years of age. potential cure in that it has only been studied in a few dozen patients (thanks to ORphan drug act shorter cook studies) and that patients haven't been followed more than a few years, so who knows how durable the treatment is Is a medicine that adds 70 years to someone's life worth $2M? What about 40 years? 10? 5? Hard to know.
Posted by aaavl2821 on Apr 30, 2019, 4:07 PM
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pricing (Healthcare)
People hate it when faceless bureaucrats decide that some health care expense is too expensive for the large faceless bureaucracy to cove...
> I will never understand why people think ACA was so great.
Turning oneself in doesn't absolve one of a crime.
All that we know is that the doctor carefully guided the patient out of the intervention, flashing a value that nobody had introduced bef...
> Is that what we want?
If we're gonna stick with private insurance in the US, we should detach it from employment.
>Putting the entire population on vitamin D supplements would be too expensive for the country’s national health service, he told me.
> At a checkup a few years ago, a doctor told me I was deficient in vitamin D.
The calculus for determining whether an indication is worth pursuing is probably determined more by 1) potential pricing and 2) translati...
The calculus for determining whether an indication is worth pursuing is probably determined more by 1) potential pricing and 2) translational risk than by total number of patients, tax incentives or added exclusivity, although shorter clin development requirements are hugely important If the translational risk is high (i.e. what works in mice / other disease models prob won't work in humans), the probability of success approaches zero. So the expected value approaches zero. It doesn't matter how many patients there are, the magnitude of the unmet need or how nice the molecule is if it isn't effective. With a high enough price, you can get a billion-dollar drug from a few hundred patients. Avexis, which was acquired by Novartis for $8.7B, had a lead drug targeting Type 1 SMA, which has only a few hundred patients worldwide. If they charge $2M / patient (which they might) it only takes 500 patients for $1B annual revenue. Of course they were pursuing other indications, had other development projects and had valuable manufacturing assets so the $9B wasn't all for that one indication, but a lot of it was And while $2M is prob high, the reason they can get away with even tossing out prices like that is that their drug is a potential cure for a disease that kills 90% of affected infants before 2 years of age. potential cure in that it has only been studied in a few dozen patients (thanks to ORphan drug act shorter cook studies) and that patients haven't been followed more than a few years, so who knows how durable the treatment is Is a medicine that adds 70 years to someone's life worth $2M? What about 40 years? 10? 5? Hard to know.
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