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Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

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121–130 of 137 posts

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#121
post #100

Earlier quoted context omitted.

It's easy. The UK can say "no" to paying for certain drugs. They take some political flak for it, but overall they've been able to keep spending down. Even mention not paying for something in the US and holy hell happens. Even Medicare and Medicaid can't say no for the most part. If American's want cheaper healthcare they are going to have to get used to having less of it and not get all the fancy new technology righ…

>It's easy. No, clearly it's not. I work for a startup that's delivering health services. Insurance companies have a very high bar for what they cover. They are very conservative about covering new therapies. There's a lot of snake oil in medicine (acupuncture, homeopathy) and they don't want to pay for what doesn't keep their members healthy.

It's not easy to execute, but it's easy to figure out one of the key drivers of increased costs in the US.

Compare drug coverage in the US vs. Canada or the UK. New drugs are often covered immediately post-FDA approval in the US. Heck, look at Exondys 51 for Duchenne's. The FDA barely approved it and pretty much every US insurer is paying for it. That doesn't happen in other countries.

A great example is the diabetes drug called Jardiance. It has better efficacy than older drugs and every US insurer covers it. It's not covered in many Canadian provinces.

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#122
post #99

Earlier quoted context omitted.

Yes, but sales and marketing has a positive ROI. If you spend $100M on sales and marketing, you'll get $150M back (for example). Otherwise, why would you do it? So if pharma companies cut out all of their sales and marketing, they'd have even less revenue than they do today. And that would lower R&D spend.

Why do drugs need marketing?

Because it's one of the key ways that physicians get new information about drugs. Not every doctor has the time or the proclivity to do research on every newly launched drug. Having a rep come in and answer questions goes a long way to getting doctors up to speed on the latest information.

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#123
post #59

Earlier quoted context omitted.

You'd want to eliminate as many potential confounding variables as possible. If they took the control group from another hospital, maybe that other hospital does something that improves or hurts their chances. Even if you pull from the same hospital, just being in the study might change the treatment they get in subtle ways. There's also the issue of blinding. If the control group is some outside group, then you know…

With something like cancer which has a wide geographical coverage, and more or less standardised treatments, couldn't you get a good result from including everyone in hospital X in a treatment and comparing to the general public?

You'd also have a host of confounding factors outside the treatment. Trace minerals in local water, different pollution, different seasons, etc...

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#124
post #58

> “We estimate the cost to manufacture a dose would be about $4,000,” she says. That’s compared to a cost of around $50,000 to alter a patient’s cells and return them. > Either type of treatment is likely to cost insurers half a million dollars or more if they reach the market. Can someone more familiar with the pharmaceutical industry explain this?

Because it's not about how expensive it is to actually make the drug. To use a software analogy, Oracle doesn't price their database software by what it costs in bandwidth for you to come to their site and download the latest version. Or in the days of shrink-wrapped software, the cost of software was far from the cost of the materials (cardboard and discs). Books similarly aren't priced based on the cost of printing…

In some cases (and you left out marketing, which often exceeds the R&D costs). In other cases, the cost is directly related to 10% less than other treatments. Or to whatever the CEO thinks he can screw out of patients. See: Shkreli; Epipen.

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#125
post #59

Earlier quoted context omitted.

You'd want to eliminate as many potential confounding variables as possible. If they took the control group from another hospital, maybe that other hospital does something that improves or hurts their chances. Even if you pull from the same hospital, just being in the study might change the treatment they get in subtle ways. There's also the issue of blinding. If the control group is some outside group, then you know…

With something like cancer which has a wide geographical coverage, and more or less standardised treatments, couldn't you get a good result from including everyone in hospital X in a treatment and comparing to the general public?

You can get A result, but not a good one.

You want to eliminate anything and everything that can (in)directly effect the outcome of treatment in a study. Ideally, the only difference at all would be the variable you are testing, but real life doesn't make things that easy.

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#126
post #119

Earlier quoted context omitted.

Speaking from very recent experience, some things are obviously standard, like "take these pills twice a day" but cancer often comes with lots of ancillary issues, and most treatments come with side effects. There was a lot of variance in how doctors and nurses treated the side effects, how they spoke to the patient, what other resources they provided, the quality of bedside manner, their attentiveness to lab results…

Sure. Anyone with cancer should go to a high volume academic and designated cancer center. The hospital should also rate high in us news and reports and score top marks in nurses, safety and cleanliness. But you can't deviate from FDA guidelines except in very exceptional cases.

What guidelines?

Doctors have tremendous latitude in how they treat patients and, as far as I know, there are no "official" guidelines for most diseases (very communicable diseases might be one major exception).

There are guidelines for developing, manufacturing, advertising, and selling drugs. However, doctors can use them off-label. From the FDA (here: http://www.fda.gov/ForPatients/Other/OffLabel/default.htm ) "From the FDA perspective, once the FDA approves a drug, healthcare providers generally may prescribe the drug for an unapproved use when they judge that it is medically appropriate for their patient. "

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#127
post #58

Earlier quoted context omitted.

Because it's not about how expensive it is to actually make the drug. To use a software analogy, Oracle doesn't price their database software by what it costs in bandwidth for you to come to their site and download the latest version. Or in the days of shrink-wrapped software, the cost of software was far from the cost of the materials (cardboard and discs). Books similarly aren't priced based on the cost of printing…

In some cases (and you left out marketing, which often exceeds the R&D costs). In other cases, the cost is directly related to 10% less than other treatments. Or to whatever the CEO thinks he can screw out of patients. See: Shkreli; Epipen.

I disagree with your statement "in some cases". It's "most cases" and where it is not, those are the ones that get the attention and screaming headlines. Shrekli is in the news precisely because this behavior is not normal. But generally, much like other goods, drugs are priced based on how much the consumer values them. I will also add, that for really big ticket things like oncology drugs, I believe every company offers payment assistance programs which often have very high income cutoffs that provide co-pay help or outright free drugs when insurance doesn't cover it.

Marketing is a red-herring. Sales and marketing should bring in more money than you spend on them or they're not worth doing. They exist to drive money This makes the company more profitable in the near term than R&D expenditure which can take decades to materialize.

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#128
post #119

Earlier quoted context omitted.

Speaking from very recent experience, some things are obviously standard, like "take these pills twice a day" but cancer often comes with lots of ancillary issues, and most treatments come with side effects. There was a lot of variance in how doctors and nurses treated the side effects, how they spoke to the patient, what other resources they provided, the quality of bedside manner, their attentiveness to lab results…

Sure. Anyone with cancer should go to a high volume academic and designated cancer center. The hospital should also rate high in us news and reports and score top marks in nurses, safety and cleanliness. But you can't deviate from FDA guidelines except in very exceptional cases.

That was my experience of variability for a family member treated within a particular high volume academic hospital and designated cancer center.

But my point is in response to the original parent comment. If you give treatment Y at one top hospital and your control group is everyone getting treatment X at all the other top hospitals, you still have basically anecdotal data.

If I'm very sick and the standard treatments aren't working, would I want to try a protocol that was anecdotally very successful in some clinic? Absolutely. Just because it's not scientifically proven to work doesn't mean it doesn't work.

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#129
There is some scrutiny.

Abstract: The London team also gave the children standard chemotherapy, they failed to show the cell treatment actually cured the kids. “There is a hint of efficacy but no proof,” says Stephan Grupp, director of cancer immunotherapy at the Children’s Hospital of Philadelphia, who collaborates with Novartis. “It would be great if it works, but that just hasn’t been shown yet.”

Re: Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish

#130

There is some scrutiny. Abstract: The London team also gave the children standard chemotherapy, they failed to show the cell treatment actually cured the kids. “There is a hint of efficacy but no proof,” says Stephan Grupp, director of cancer immunotherapy at the Children’s Hospital of Philadelphia, who collaborates with Novartis. “It would be great if it works, but that just hasn’t been shown yet.”

The odd thing: does standard chemo ever totally cure anybody? If not, then what did cure them, if not the new treatment?
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