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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

#151

Post by ekianjo »

lotsofpulp wrote: post #23
ekianjo wrote: post #20

Earlier quoted context omitted.

You can't just reduce the patent period without reducing the regulatory process that is required to get stuff approved. It would not work otherwise.

I hope we're not on the path to reducing rigorous testing and vetting of medicines in exchange for cheaper prices. Medical science is extremely hard, and verifying cause and effect in a system as complex as the human body takes significant resources. People want cheap, people want safe, people want well qualified individuals pursuing these advances. What people don't want is higher taxes to enable government funding…

> I hope we're not on the path to reducing rigorous testing and vetting of medicines in exchange for cheaper prices

The FDA has extremely long review cycles. To say that this could be not be improved, while still keeping the same standards (or even better ones) is unlikely. Anyway, I don't think it's likely that we will see anyone touching the patent system anytime soon, so it's merely a thought exercise at this stage.

Re: How pharmaceutical industry financial modelers think about rare diseases

#152

Post by dcx »

rscho wrote: post #147
dcx wrote: post #146

Earlier quoted context omitted.

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 proced…

I understand what you mean by the typical American cancer story. But I do get the sense you have pattern-matched this story for what I shared and may have missed the value of it.

We're not talking about quack treatments and unnecessary procedures here. Being intelligent about diving into the medical literature for a specific cancer, and consulting with academics and professionals in the field, in order to correctly identify treatments that may be effective for this case at the cutting-edge years before they enter the mainstream - this seems like an eminently correct thing to do. A simple Google search of "glioblastoma ketogenic diet" should show you the growth of research and good results now emerging in this area. They were ten years ahead of the curve on this.

And it's a fact that some medical professionals are better than others and this makes a quantifiable difference. There are bad doctors. And this is reflected in the studies. Doctors fall behind on recent developments, or have different levels of experience with specialised procedures (say, due to being in lower-population rural areas, as in this case), or just plain have worse manual dexterity. [1] [2] The original hospital did a bunch of damage, and I believe they even tried to sweep things under the rug in some way (I don't remember the exact details unfortunately).

What I am describing is not something the average person can do. Which is why it is not a mainstream recommendation on how to interface with the medical system. The average person will probably end up suckered into magic cancer beads, quack doctors, and then into the story you describe. This takes intelligence, effort and time. And risk-reward calculations. My relatives are quantifiably in the top one percent on measures of intelligence and education. As mentioned I chose to share this here specifically because many of us here fit the same kind of demographic.

[1] https://well.blogs.nytimes.com/2013/10/31/a-vital-measure-yo...

[2] https://www.theatlantic.com/health/archive/2017/02/when-evid...

Re: How pharmaceutical industry financial modelers think about rare diseases

#153

Post by boyband6666 »

gdy wrote: post #144
boyband6666 wrote: post #46

Earlier quoted context omitted.

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.

That's actually a very interesting one, and has been written about at length by a number of medical stats people.

Hopefully I do them justice to their arguments by saying that the benefits from supplements (if any) are tiny - as in a hazard ratio of death is pretty much 1 unless you are actually deficient. Equally however the potential downside of them is pretty much zero - expensive wee is what they can mostly be summarised as. Whilst they may be money wasted, the're mostly benign, save the odd tragic case.

If supplements had the side effects of chemotherapies however, they'd be banned straight away - without evidence of benefit, the risks would be too high.

For a more authoratative take, one of my heroes: http://www.dcscience.net/2013/11/18/we-know-little-about-the...

Re: How pharmaceutical industry financial modelers think about rare diseases

#154

Post by boyband6666 »

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…

The studies performed are the same ones, but companies submit it to the FDA first, who are more likely to approve, and approve faster than European regulators (the European Medicines Agency, EMA). The FDA are the most permissive regulator is what has been seen in the literature - there was a really good study looking at the same drugs (TKIs) submitted to various agencies (it's not the only one, but is the cleanest):

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3769667/

As for thalidomide, yep the FDA had an amazing employee who saved a lot of damage being done, and shows exactly why regulators exist, and assess the safety and efficacy before you can sell things!

Re: How pharmaceutical industry financial modelers think about rare diseases

#155

Post by boyband6666 »

agumonkey wrote: post #135

Great article. Best of luck to them. Anybody knows about efforts to lower trial phases costs down ? (without lowering quality of course). Any out of the box idea ?

Collaboration - one control arm, 2+ active drugs from multiple companies (why not 6+?). Fewer patients exposed to placebo, less paperwork, same statistical power, more so if you randomise 2:1:1:1:1:1 rather than a standard trial with 1:1 or 2:1

Re: How pharmaceutical industry financial modelers think about rare diseases

#156

Post by gdy »

boyband6666 wrote: post #153
gdy wrote: post #144

Earlier quoted context omitted.

"you still need to know if it works" No, you do not. Just look at the market of dietary supplements.

That's actually a very interesting one, and has been written about at length by a number of medical stats people. Hopefully I do them justice to their arguments by saying that the benefits from supplements (if any) are tiny - as in a hazard ratio of death is pretty much 1 unless you are actually deficient. Equally however the potential downside of them is pretty much zero - expensive wee is what they can mostly be su…

Potential downside is pretty high if you consider that some people tend to take supplements and other sorts of snake oil instead of working medicine.

Re: How pharmaceutical industry financial modelers think about rare diseases

#157

Post by phkahler »

Balgair wrote: post #136
phkahler wrote: post #133

Earlier quoted context omitted.

But if you've been handed a death sentence, why NOT try a keto diet? That's a rhetorical question btw of course the is no reason not to try it.

Each cancer is unique to the organism. Not all cancers are death sentences. Nearly every sunburn you or I have had has been cancerous to some degree; the inflammation and reddening are your body fighting those cancers (among many other issues). Some cancers can be benign, some can be handled quite easily, some can be treated in other ways. Again, each is unique. The data on Ketogenic diets and cancer are showing a lo…

Palliative care is an admission that chemo won't help and pretty much all options have been tried. At that point one can sit back and die, or try something.

Re: How pharmaceutical industry financial modelers think about rare diseases

#158

Post by elliekelly »

refurb wrote: post #150
elliekelly wrote: post #148

Earlier quoted context omitted.

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”.

That’s fair and the struggle for all regulators I would imagine (though the stakes are higher with human health).

The crypto people drive me nuts the same way. One minute they’re extolling the virtue of markets “free” from regulatory interference but the second it turns out the market was a scam they want to know what the government plans to do to get their money back.

Re: How pharmaceutical industry financial modelers think about rare diseases

#159

Post by Balgair »

phkahler wrote: post #157
Balgair wrote: post #136

Earlier quoted context omitted.

Each cancer is unique to the organism. Not all cancers are death sentences. Nearly every sunburn you or I have had has been cancerous to some degree; the inflammation and reddening are your body fighting those cancers (among many other issues). Some cancers can be benign, some can be handled quite easily, some can be treated in other ways. Again, each is unique. The data on Ketogenic diets and cancer are showing a lo…

Palliative care is an admission that chemo won't help and pretty much all options have been tried. At that point one can sit back and die, or try something.

Again, it depends on the situation at hand. Though ketogenic diets are showing promise, it depends on the cancer, as such diets may aggravate the cancer. One should follow the advice of the oncologist/s as well as research on their own.
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