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Cystic fibrosis breakthrough has given patients a chance to live longer

theatlantic.com

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Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#131

Post by TechnicalVault »

ipaddr wrote: post #76
mason55 wrote: post #17

Earlier quoted context omitted.

> I would have imagined that most people with CF already had extreme anxiety - wondering when the infection that is going to end one's life will arrive. Is it really possible that Trikafta is causing noticeably worse anxiety? There's a discussion of this in the article. Essentially, a bunch of people have said that they started experiencing extreme anxiety when they started Trikafta, and that stopping or lowering the…

Clinicians are always a day late and a dollar short. The evidence comes from these people reporting. These clinicians need a 50 million dollar grant to get that proof.

If you've ever had to organise a scientific study you'll understand why. Privacy and ethics are important but they make recruitment of a large and representative sample for any medical study a nightmare. First you'll need to run your protocol past an ethics board and do back and forth until it's agreed. Once you have that done you'll send out an invite (often blind) in hopes of getting people who fit your study aims and you will get who you get. There are often costs associated with this.

So now you have some willing participants you have to screen your initial group to filter it down to people who actually meet your recruiting criteria and consent them for your study. Finally you actually get to gather your data, if you need people to come in for sampling and have to cover their expenses.

Next you'll look at your data and realise there's some confounding effect which reduces your powers to infer anything (e.g. somehow you overrecruited a particular group and they turn out to do something which correlates with the thing you're studying). You'll cry a little and realise you need to recruit more people to have any statistical power to draw a conclusion.

tldr; medical and scientific studies are hard if you want them to actually have any validity.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#132

Post by speedylight »

bane wrote: post #7

Oh wow. I remember a kid in elementary school with CF. I didn't understand at the time what he and his family was going through. I remember his personality and intelligence, his skill on the soccer team. He was unusually driven for an 8-9 year old. As I got older and into my 20s, I thought about him a lot -- it was understood that most CF patients don't make it out of their early 20s and I knew even if he were partic…

Have you tried looking him up? He could still be alive.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#133

Post by epivosism »

elliotto wrote: post #130
matthewtse wrote: post #126

Earlier quoted context omitted.

I love this reply. It's a particularly touchy subject because it involves people's health and the ability/inability to pay for it. But as a sufferer of a debilitating disease with a miracle drug that costs an arm and a leg, I've become a convert to the US pharma-industrial complex. These drugs take billions of dollars and decades to research--that funding/development isn't going to happen without venture capitalists…

It feels like there's a lack of imagination here. Could you conceive of a world that was successful in its research of drugs, but was propped up by an economic model other than pharma venture capitalism?

Freedom of choice means you have the right to try to create such a system. As other people can try what they want, VC funding, astrology, etc. It's all legal and competes.

But, if someone is selling a system which doesn't exist yet, but is supposed to be better, but that will only work if we also shut off the one working system first, before knowing... well, I'm skeptical.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#134

Post by actuallyalys »

derivative7 wrote: post #117
kelnos wrote: post #85

Earlier quoted context omitted.

What about direct government funding? Isn't that a reasonable alternative? Maybe not a politically-popular one, but I wouldn't be surprised if health care overall would be cheaper under a system where pharma research is done with public money, and then isn't allowed to be patented. Might even in save enough money to avoid much in the way of extra taxes to fund it. Hell, a lot of medical research is funded (in part) b…

Most taxpayers don't have cystic fibrosis and hence don't want to pay to have it cured. Not feasible. This hypothetical is kind of lame.

The U.S. government already funds a lot of research including some on conditions (or non-medical problems) that don’t affect the majority so that doesn’t seem to be an actual barrier. Polls tend to show Americans support existing funding [0].

Obviously there is a limit to how much spending the public will support, but I don’t think it’s a complete nonstarter.

[0]: https://www.pewresearch.org/short-reads/2018/07/03/americans...

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#135

Post by pas »

TechnicalVault wrote: post #131
ipaddr wrote: post #76

Earlier quoted context omitted.

Clinicians are always a day late and a dollar short. The evidence comes from these people reporting. These clinicians need a 50 million dollar grant to get that proof.

If you've ever had to organise a scientific study you'll understand why. Privacy and ethics are important but they make recruitment of a large and representative sample for any medical study a nightmare. First you'll need to run your protocol past an ethics board and do back and forth until it's agreed. Once you have that done you'll send out an invite (often blind) in hopes of getting people who fit your study aims…

wouldn't this side-effect become visible in Phase4 data? (and there should be at least some hints of this in the previous ones)

so, what I'm trying to say is that for this particular drug we "just need to wait", no?

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#136

Post by TotalCrackpot »

fnordpiglet wrote: post #120
TotalCrackpot wrote: post #112

Earlier quoted context omitted.

How do you practice for becoming a quant outside of the academic system?

You do things because you love it and couldn’t do anything other than that. You build and study not because you want a job but because when you wake up in the morning everything else you have to do is a chore and the reason you live is to learn and see things work. If that’s who you are, you will struggle with the chores of life like shelter and food for a while but you’ll learn and do so much of value people can’t i…

Ok, so how do you practice in practice? What specific actions do you do to make yourself more attractive to prop shops?

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#137

Post by sunnybeetroot »

bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

Assuming your anxiety medication is an SSRI or similar, your weight gain could be attributed to this as it’s been observed in people who take these medications.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#138

Post by ETH_start »

mmustapic wrote: post #128
ETH_start wrote: post #106

If I can be allowed a bit of sophistry: It's a beautiful thing for treatments to be devised for rare conditions, to give people decades more life. It would be more wonderful still if we devised treatments for aging, the universal condition, to grant everyone decades more life. Hardly anyone deserves to die. The vast majority of people are singularly incredible and worthy of life. They deserve to be equally safe from…

Many incurable diseases come with an awful quality of life, like CF. Even cancer, treated or not, is a terrible experience. Just aging, what you call “a disease”, is nothing like that.

Aging comes with a terrible quality of life. By the end, the person is severely debilitated.

Osteoporosis can make it so that a fall can lead to major fractures, which leads to a severe reduction in a person's independence. Macular degeneration can make an elderly person blind.

Neurological deterioration can take the form of conditions like Alzheimer's, where a person loses their memory of everyone they love.

The body, as it ages, becomes much more susceptible to other, terrible, diseases as well, like cancer, heart disease, and diabetes, each carrying its own set of debilitating effects that degrade quality of life.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#139

Post by AdamN »

TechnicalVault wrote: post #131
ipaddr wrote: post #76

Earlier quoted context omitted.

Clinicians are always a day late and a dollar short. The evidence comes from these people reporting. These clinicians need a 50 million dollar grant to get that proof.

If you've ever had to organise a scientific study you'll understand why. Privacy and ethics are important but they make recruitment of a large and representative sample for any medical study a nightmare. First you'll need to run your protocol past an ethics board and do back and forth until it's agreed. Once you have that done you'll send out an invite (often blind) in hopes of getting people who fit your study aims…

It seems like we really need to move forward to 'indefinite/active studies'. I believe some researchers are trying to get onto this pattern but of course there are major privacy/quality of care concerns.

By 'indefinite/active studies' I mean that the studies never stop - data just keeps going back into the system as a flywheel of the drug distribution process. I take a second-generation drug for CML (leukemia) and none of my health data goes back into research unless my doctor decides to elevate my data into a paper or something (which I don't think has happened yet).

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#140

Post by dennis_jeeves2 »

qgin wrote: post #96
forgetfreeman wrote: post #3

I had a coworker with CF that started this treatment. Over the course of their first year of treatment, as it became plain to them just how effective it was and what the implications for their long-term survival might be, their behavior changed drastically. Within 18 months of starting treatment they were showing worrying indications of both budding mental health issues and heavy substance abuse. Their marriage, whic…

Imagine if tomorrow, it was announced there had been a discovery that doubled average human life expectancy to 150. There would be happiness, but there would also be chaos. How many people would quit all the things they'd told themselves they were too old to change? The job they were grinding out until retirement. The marriage they'd resigned themselves to? Imagine billions of people getting pulled backwards back acr…

>There would be happiness, but there would also be chaos

Which is good in the short term, correct? Long term people hopefully would be wise to settle for stable relationships, jobs etc...

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