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How pharmaceutical industry financial modelers think about rare diseases

cureffi.org

81–90 of 159 posts

Re: How pharmaceutical industry financial modelers think about rare diseases

#81
post #42

This is an impressive article and an impressive couple. From the site's about page: My name is Eric Vallabh Minikel and I’m on a lifelong quest to develop a treatment or cure for human prion diseases. I originally trained as a city planner at M.I.T. and was working as a software engineer and data analyst in the transportation sector when, in December 2011, I got some bad news. My wife and the love of my life, Sonia V…

>For example they switched him to a ketogenic diet back when this was just starting to show results for cancer

I'm curious as to how this would have an impact. Is there any research demonstrating that keto slows the development of cancer? Or hypotheses on why that would be the case?

Re: How pharmaceutical industry financial modelers think about rare diseases

#82
post #81
post #42

This is an impressive article and an impressive couple. From the site's about page: My name is Eric Vallabh Minikel and I’m on a lifelong quest to develop a treatment or cure for human prion diseases. I originally trained as a city planner at M.I.T. and was working as a software engineer and data analyst in the transportation sector when, in December 2011, I got some bad news. My wife and the love of my life, Sonia V…

>For example they switched him to a ketogenic diet back when this was just starting to show results for cancer I'm curious as to how this would have an impact. Is there any research demonstrating that keto slows the development of cancer? Or hypotheses on why that would be the case?

Partially related to insulin and blood sugar levels largely.

IGF.

Depends on the cancer though, etc.

Re: How pharmaceutical industry financial modelers think about rare diseases

#83
post #49

Earlier quoted context omitted.

You see people do this a lot with housing. They make $100k after 5 years and pat themselves on the back. They ignore that if they had put their down payment in the market, they would have made $110k.

The difference is that they can buy homes on margin, whereas no one would lend to them to invest in the market on margin. Return has to account for only putting 5% to 20% down. Not that I think homes are a good investment outside of the few burgeoning cities in the US, compared to index funds, time/labor costs included.

They must be a shockingly good investment in my city from all the junk mail I get advertising shared equity contracts.

Re: How pharmaceutical industry financial modelers think about rare diseases

#84
post #81
post #42

This is an impressive article and an impressive couple. From the site's about page: My name is Eric Vallabh Minikel and I’m on a lifelong quest to develop a treatment or cure for human prion diseases. I originally trained as a city planner at M.I.T. and was working as a software engineer and data analyst in the transportation sector when, in December 2011, I got some bad news. My wife and the love of my life, Sonia V…

>For example they switched him to a ketogenic diet back when this was just starting to show results for cancer I'm curious as to how this would have an impact. Is there any research demonstrating that keto slows the development of cancer? Or hypotheses on why that would be the case?

If you google for studies, at this point you'll see an abundance of research on this with positive results, which apparently was just getting started when they dug into it and perhaps not well known to the average doctor. My memory plus a quick look into one [1] suggests it's because brain tumor cells largely feed on glucose, whereas normal brain cells can also metabolise ketone bodies for energy. So you're starving the cancer of resources.

[1] https://nutritionandmetabolism.biomedcentral.com/articles/10...

Re: How pharmaceutical industry financial modelers think about rare diseases

#85
post #49

Earlier quoted context omitted.

You see people do this a lot with housing. They make $100k after 5 years and pat themselves on the back. They ignore that if they had put their down payment in the market, they would have made $110k.

The difference is that they can buy homes on margin, whereas no one would lend to them to invest in the market on margin. Return has to account for only putting 5% to 20% down. Not that I think homes are a good investment outside of the few burgeoning cities in the US, compared to index funds, time/labor costs included.

Most equities are already leveraged.

Re: How pharmaceutical industry financial modelers think about rare diseases

#86
post #49

Earlier quoted context omitted.

You see people do this a lot with housing. They make $100k after 5 years and pat themselves on the back. They ignore that if they had put their down payment in the market, they would have made $110k.

The difference is that they can buy homes on margin, whereas no one would lend to them to invest in the market on margin. Return has to account for only putting 5% to 20% down. Not that I think homes are a good investment outside of the few burgeoning cities in the US, compared to index funds, time/labor costs included.

My comment ignores the leverage and just looks at the downpayment alone.

To your point, their $100k gain at 5x leverage kind sucks compared to a $110k gain at zero leverage.

Re: How pharmaceutical industry financial modelers think about rare diseases

#87
post #79
post #34

Earlier quoted context omitted.

There is much wrong with the pharmaceutical industry including pay-to-delay generic I disagree on this one. Pay-to-delay is just the branded drug manufacturer and the generic company splitting the difference through negotiation. What you end up with something in-between the two potential outcomes. Situation: branded drug has 2 more years of patent life, but generic company wants to challenge it; neither party is sure…

If the patent is invalidated, then what provides the generic company with exclusivity for two years-- I thought the regulatory exclusivity provided to first-in generics was only 6 months?

It was just an example. You are correct that the first generic entry gets just 6 months of exclusivity.

Re: How pharmaceutical industry financial modelers think about rare diseases

#88
post #73
post #34

Earlier quoted context omitted.

There is much wrong with the pharmaceutical industry including pay-to-delay generic I disagree on this one. Pay-to-delay is just the branded drug manufacturer and the generic company splitting the difference through negotiation. What you end up with something in-between the two potential outcomes. Situation: branded drug has 2 more years of patent life, but generic company wants to challenge it; neither party is sure…

> Everybody gets something from the deal Everyone except the patient, who continues to pay the exclusivity premium but would (assuming option 2) have paid less.

But you don't know option 2 would have been the outcome. Rather than both companies duking it out in court for a few years, the patient benefits when the generic entry happens quicker than if the patent held.

Re: How pharmaceutical industry financial modelers think about rare diseases

#89
post #40

Looking at these numbers, I wonder whether the FDA process is a bit too conservative with respect to safety. There were the articles yesterday about diabetics yesterday creating closed-loop feedback systems out of old insecure parts, because a FDA-certified alternative is decades out. I've heard also of folks joining studies for newer-and-better IUDs that are approved by European regulators, but need to be re-certifi…

The thing is, the big established pharma companies don't necessarily mind the difficulty in bringing a drug or device to market since it makes competition more difficult. And it's a politically difficult move to support loosening regulations and restrictions here, because some people will inevitably die due to the lower standards, and everyone will remember those dozens/hundreds of people rather than the hundreds of thousands who are able to get new treatments faster

Re: How pharmaceutical industry financial modelers think about rare diseases

#90
post #84
post #81

Earlier quoted context omitted.

>For example they switched him to a ketogenic diet back when this was just starting to show results for cancer I'm curious as to how this would have an impact. Is there any research demonstrating that keto slows the development of cancer? Or hypotheses on why that would be the case?

If you google for studies, at this point you'll see an abundance of research on this with positive results, which apparently was just getting started when they dug into it and perhaps not well known to the average doctor. My memory plus a quick look into one [1] suggests it's because brain tumor cells largely feed on glucose, whereas normal brain cells can also metabolise ketone bodies for energy. So you're starving…

[1] was a mouse study. Do you have any examples of human studies? I have this picture in my mind of someone showing up to a doctors office with a thumb drive of 30 PDFs of nonclinical studies they found through a Google search and demanding treatment x, diet y, and so on.
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