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How pharmaceutical industry financial modelers think about rare diseases

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Re: How pharmaceutical industry financial modelers think about rare diseases

#141

Post by DoreenMichele »

iandanforth wrote: post #140
DoreenMichele wrote: post #127

Earlier quoted context omitted.

Ordinary joes are already finding loopholes in the medical system, such as this article from yesterday about the popularity of old insulin pumps with an exploitable security flaw: https://news.ycombinator.com/item?id=19781118 Eventually one or more of the ultra wealthy is going to survive a disease that government sanctioned healthcare says is incurable. I knew a woman who used chelation to resolve issues in a child…

You may be right but now I want to hear your story!

- once upon a time, roughly 18 years ago -

They found a better name for my condition than "hypochondria." They renamed it atypical cystic fibrosis.

It's a variation on a dread disease with a short life expectancy.

I began getting healthier. My doctor expressed zero curiosity and scheduled me fewer appointments.

Fast forward 18 years, the internet likes to claim I have Munchausen. In other words, I've come full circle from "crazy" to "legitimate medical condition" to another variation of "crazy" for the crime of rudely getting healthier when the world says "You can't do that!"

- the end -

Re: How pharmaceutical industry financial modelers think about rare diseases

#142

Post by dickless-a-hole »

burtonator wrote: post #122
dcx wrote: 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…

> I have relatives who managed to convert their dad's brain cancer outcomes from a couple of months in palliative care (which the doctors were recommending) to five more mostly-good years That's literally NOT how this works and this line of thought is VERY dangerous. You have an N=1 sample size here. Doctors use probability distributions when making prognosis and recommendations for treatment. It's far more plausible…

Hey shit for brains, did you know that many experimental treatments for cancer are randomized double blinds that result in a total lack of treatment.

Doctors frequently sign their patients up to roll the dice and get nothing.

By all means shout down anyone you want but you have NO idea which cancer patients are actually being saved by their medication.

Treatment != care.

Re: How pharmaceutical industry financial modelers think about rare diseases

#143

Post by lysium »

DoreenMichele wrote: post #130
FabHK wrote: post #118

Earlier quoted context omitted.

> One thing I've learned from smart family and friends about medical care is that you can sometimes improve outcomes quite a lot if you apply brains and effort. I'd be very careful with this sentiment though, because it is easy to turn it into the toxic notion that if a loved one dies from some disease (which is pretty inevitable), you didn't put enough brains or effort in it.

Unfortunately, every medical "support group" I have ever belonged to bent over so far backwards to not "blame the victim" that you absolutely couldn't talk about what worked because, inevitably, the sickest people with the worst war stories were the same ones who smoked, actively dismissed the idea that replacing carpeting with wood and tile floors might make a difference, etc. No, it absolutely wasn't socially accep…

Off topic: why would replacing carpeting with wood and tile floors make a difference?

Re: How pharmaceutical industry financial modelers think about rare diseases

#144

Post by gdy »

boyband6666 wrote: post #46
cbhl wrote: 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…

Simple answer is probably not; even a lowering of standards you still need to know if it works - so still doing studies. You'd also then get more drugs like Lily's recently that in post-marketing turns out not to work, but ate of half a billion in sales, and a lot of patients who got not benefit. There is no simple easy answer, it's a massively hard problem. The regulators have to balance benefit and risk, and for th…

"you still need to know if it works"

No, you do not. Just look at the market of dietary supplements.

Re: How pharmaceutical industry financial modelers think about rare diseases

#145

Post by DoreenMichele »

lysium wrote: post #143
DoreenMichele wrote: post #130

Earlier quoted context omitted.

Unfortunately, every medical "support group" I have ever belonged to bent over so far backwards to not "blame the victim" that you absolutely couldn't talk about what worked because, inevitably, the sickest people with the worst war stories were the same ones who smoked, actively dismissed the idea that replacing carpeting with wood and tile floors might make a difference, etc. No, it absolutely wasn't socially accep…

Off topic: why would replacing carpeting with wood and tile floors make a difference?

It's cleaner.

I'm referencing discussions about lung issues in specific, if that helps you make sense of things.

Re: How pharmaceutical industry financial modelers think about rare diseases

#146

Post by dcx »

burtonator wrote: post #122
dcx wrote: 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…

> I have relatives who managed to convert their dad's brain cancer outcomes from a couple of months in palliative care (which the doctors were recommending) to five more mostly-good years That's literally NOT how this works and this line of thought is VERY dangerous. You have an N=1 sample size here. Doctors use probability distributions when making prognosis and recommendations for treatment. It's far more plausible…

This was far more than just random chance. It's clear as day that quality of medical care makes a difference in outcomes, right? I have sent this discussion to the relatives in question so forgive me if I get the exact details wrong. But the median survival time for glioblastoma is fourteen months, and they were quite a while into that. Their original doctors were recommending palliative care (giving up on further medical treatment) with a couple of months expected survival.

My relatives live in a small city not known for its quality of medical care. They found the very best neurosurgeon in the country for glioblastoma and transferred him over with some difficulty. They learned that the initial surgeries were not done to a high quality or possibly even slightly botched - and the new surgeon was able to perform additional surgeries that were able to remove a significant amount of the tumour (and did follow-up surgeries later on). Of course this would extend a patient's lifespan!

The ketogenic diet now seems to be becoming a standard recommendation for glioblastoma treatment. There are TON of studies now. Here are a few more more. [1] [2] [3] Note the dates on these studies (2017-2018). Back when they looked at this, this was absolutely bleeding-edge stuff. Of course going to the cutting edge of the research, speaking to professors and using your brain to figure out what might help in your case will improve outcomes!

Regarding clinical trials, one can read up on / sign up for trials of treatments at different stages of the drug research pipeline. As the modelling spreadsheet in the link above shows, between 1-in-10 and 1-in-2 of these trials will result in FDA-approved treatments that will hit the market between 2 and 10 years from now. If your current expected survival time is in months, it is statistically worth implementing as many of these as possible, because at least one of them will probably work, and you don't have the time to wait for the FDA! You don't really care which one as, like you say, your sample size is N=1.

I shared this in the hope that some of the smart people on this forum would read this and know that this is something you may be able to do. We are engineers and scientists. We can read studies and think critically. We can spend a ton of time absorbing specialised information about the N=1 case that matters to us. Death comes for everyone and the above may not make a difference in many cases. But it turns out that sometimes it does! Don't treat medical care like a black box.

[1] https://www.practiceupdate.com/content/sno-2018-clinical-tri...

[2] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5834833/

[3] https://www.sciencedirect.com/science/article/pii/S104084281...

Re: How pharmaceutical industry financial modelers think about rare diseases

#147

Post by rscho »

dcx wrote: post #146
burtonator wrote: post #122

Earlier quoted context omitted.

> I have relatives who managed to convert their dad's brain cancer outcomes from a couple of months in palliative care (which the doctors were recommending) to five more mostly-good years That's literally NOT how this works and this line of thought is VERY dangerous. You have an N=1 sample size here. Doctors use probability distributions when making prognosis and recommendations for treatment. It's far more plausible…

This was far more than just random chance. It's clear as day that quality of medical care makes a difference in outcomes, right? I have sent this discussion to the relatives in question so forgive me if I get the exact details wrong. But the median survival time for glioblastoma is fourteen months, and they were quite a while into that. Their original doctors were recommending palliative care (giving up on further me…

I get that people would try anything to help their loved ones. This in itself is admirable and goes to show the guy was truly loved.

We are beyond discussing the scientific value of your approach, so I'll try to communicate what you say conveys for a professional.

This is the typical american cancer story. The guy did not want to die, and his family and himself forced him through loads of invasive/experimental procedures because the success of treatment was viewed as a meritocratic achievement, which it is within the bounds of established treatment. I've seen this too many times to count and it saddens me greatly that already sick people get a substantially added dose of suffering on the basis of wishful thinking.

If the guy wanted to help OTHERS through his participation in trials, then that's really nice. His family, although very nice people I am sure, should not have had anything to do with his decision.

PS: the "best" surgeon wanting to operate on someone most of his colleagues would not touch with a 10-foot pole should have you start running.

Re: How pharmaceutical industry financial modelers think about rare diseases

#148

Post by elliekelly »

refurb wrote: post #138
elliekelly wrote: post #126

Earlier quoted context omitted.

Your comment reminded me of the (ridiculous but sadly necessary) "Sunscreen Innovation Act" passed a few years ago.[1] U.S. financial regulators often "vet" their equivalent foreign agencies and whitelist those whose processes are sufficiently analogous and in some cases allow US entities to rely upon the work done in those whitelisted jurisdictions. I've always wondered why the FDA can't work out a similar system. I…

I've always wondered why the FDA can't work out a similar system. If the EU has approved a drug and done all of the studies and asked all of the questions surely we don't need to do all of the same studies and ask all of the same questions just so we can approve the drug a decade later. The reason is thalidomide. The US was one of the few countries that chose not to approve it despite other countries doing so. Saved…

Ah yea, thalidomide. That drug is often used to justify poor policy decisions at the FDA. It's the reason the FDA banned women of child bearing age from clinical research.[1] In the late 80's there was even a study conducted to determine the relationship between obesity and risk for breast and uterine cancer. Guess how many women were enrolled? Zero. Thanks to the FDA's misguided knee jerk policy response to thalidomide.

It seems to me the FDA's process might "protect" a small number of people but that comes at the expense of many thousands of lives.

[1]https://www.drugwatch.com/news/2018/07/25/excluding-women-fr...

Re: How pharmaceutical industry financial modelers think about rare diseases

#149

Post by pmart123 »

burtonator wrote: post #122
dcx wrote: 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…

> I have relatives who managed to convert their dad's brain cancer outcomes from a couple of months in palliative care (which the doctors were recommending) to five more mostly-good years That's literally NOT how this works and this line of thought is VERY dangerous. You have an N=1 sample size here. Doctors use probability distributions when making prognosis and recommendations for treatment. It's far more plausible…

I would echo similar experiences. Healthcare is extremely bureaucratic, and there is an art to navigating it. While I have more than a sample size of one throughout my extended family, I can share my own experience when I had appendicitis right before my sister's wedding in my early 20's.

My initial action after I started having symptoms was to go to the ER. I waited five hours in the waiting room, and then decided to go home due to my pain and fever condition being intolerable. I then learned that I could have my primary care doctor call the ER to fast pass me from having to wait in feverish pain. Then, once my diagnosis was official, the resident on call recommended that I elect him to perform the surgery overnight. Since it was during my sister's wedding, my parents were able to come by after the rehearsal dinner, and insist that I wait until the morning for the doctor to perform the surgery. The doctor, who had a national reputation, came in at 6 AM, and with the confidence of Steph Curry lining up for a three point shot, changed the operating procedure due to specific circumstances. As a naive 20 year old, I would have just elected to do what the resident was recommending, and it probably would have been ok. Yet, I think I ended up better off with having family present, pressing back on the recommendation, and waiting for the doctor to arrive.

Re: How pharmaceutical industry financial modelers think about rare diseases

#150

Post by refurb »

elliekelly wrote: post #148
refurb wrote: post #138

Earlier quoted context omitted.

I've always wondered why the FDA can't work out a similar system. If the EU has approved a drug and done all of the studies and asked all of the questions surely we don't need to do all of the same studies and ask all of the same questions just so we can approve the drug a decade later. The reason is thalidomide. The US was one of the few countries that chose not to approve it despite other countries doing so. Saved…

Ah yea, thalidomide. That drug is often used to justify poor policy decisions at the FDA. It's the reason the FDA banned women of child bearing age from clinical research.[1] In the late 80's there was even a study conducted to determine the relationship between obesity and risk for breast and uterine cancer. Guess how many women were enrolled? Zero. Thanks to the FDA's misguided knee jerk policy response to thalidom…

Hey, I don’t disagree that that FDA is very conservative. My comment was more around the politics of it.

Look back at the Vioxx hearings. People crapped all over the FDA because they didn’t catch an increased risk of CV issues until post-approval.

If you’re the FDA, it’s “damned if you do, damned if you don’t”.

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