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Rw005g

Rw005g
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Constantinople, Byzantine Empire
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December 31
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Strategos
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Varangian Guard
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Defender of the Old Republic, and member of the 99%

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FEBRUARY 10, 2012 12:37PM

Orphan Diseases and Capitalism

Rate: 22 Flag

I think its sad and unfortunate that corporate America and the United States government wholly assign medical research grants and funding, according to a disease's prevalence.

Congress feels that we should only fund medical research that benefits the greatest number of people. Corporate America feels the same way. When you invest money, you want the "biggest bang for your buck." Why invest money in researching diseases that very few people get? Why should a company spend millions of dollars developing drugs and treatments for such rare diseases, when the likely profits from the selling of said drugs and treatments are likely to be very small?

It seems that the rules of Capitalism, here, are similar to the basic rules of base, majoritarian democracy.  Here, the needs or desires of the many outweigh the needs of the few or the one. Democracies throughout the world, though, have learned throughout the course of time that this is a bad policy. The rights of small religious, political, ethnic and/or religious groups are just as important as those of the majority, and when the majority wants to pass legislation in the interest of the whole, we need to be careful that we don't deny the benefits of the "Social Contract" to other, more vulnerable members of our society. 

For example, when a country is at war, it often enacts a draft. That said, there are many people who would be opposed to military service, either for religious reasons or matters of personal conscience and conviction. Hence the ability of a specific class of people to be exempt from conscription. Also, when a government (often acting on the desires of a majority) wishes to seize private property for public use, the law dictates that the owner of said land be compensated according to "fair market value." We seem to have developed, as a democracy, general political and legal rules for protecting the minority or the individual from the abuses inflicted by the majoritarian will.  

 That said, we have no such rules in the economic sphere. The economic demands of the majority may run counter to the economic requirements of the few. For example, the sicknesses that are most likely to affect Americans are cancer, heart disease, etc...As such, we invest, as a society, hundreds of millions of dollars on trying to find cures and/or treatments for these diseases. The "majority will" can often be gauged, in some ways, through market mechanisms, however imperfectly. Of course, CEOs, politicians and shareholders are imperfect beings and they interpret this "majority will" differently and imperfectly. 

That said, there are hundreds of diseases out there, called "orphan diseases" that are totally ignored by Academia, the government and corporate America, because the profits that could be gained from their exploration are marginal. Our refusal to research cures and treatment often has nothing to do with our likelihood of finding a cure or successful treatment. Indeed, sometimes the treatment can be very easily produced, but the companies don't want to pay for it, because the diseases are either rare, or because those who are afflicted with it lack the money to pay.

My wife is an academic in the sciences and she says that many scientists are fascinated by "orphan diseases" because they give us unique insights into how different diseases work, spread, operate and the like. And by researching and knowing them more in-depth, we can actually gain a greater understanding of more widespread diseases like cancer, epilepsy, ALS and the like. 

I only bring this up because I found out that one of my good friends from high school, Heather A., died this morning. She had an "orphan disease," named chiari, and had to undergo massive brain surgery to fix it. Only a few have ever been performed. At first it looked like she would do well, but she went into a coma and was declared brain dead two days ago. She passed away this morning, but not before engaging in the incredibly heroic act of donating her organs to desperately sick people in need. 

Chiari, and hundreds of diseases like it, are not getting the funding and research dollars that perhaps they should be getting. Many diseases that can be treated and possibly even cured are being ignored, simply because corporations and their government enablers don't think its profitable to do so. 

Heather A. was one of the most cheerful,wonderful people I have ever met and the world was a better place for her being here. I had many wonderful political, social and economic discussions with her, ever since we were in high school, and I shall miss her and these talks greatly. She was one of my biggest supporters, in terms of encouraging me to write and start a blog, and she was one of my blog's biggest fans. I shall miss her very, very much. 

If we can support the "will of the majority" in politics and law, while also protecting the "interests of the minority," then can we not also pursue such a policy when it comes to biomedical research and healthcare?

I think that we can. We owe it to ourselves, our friends and our families. I for one, am now wholly resolved to pursue the cause of greater research equity. It is a cause I will not give up. 

 

Heather A--rest in peace. You will not be forgotten.

 

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This is a caring, timely post. I am sorry for you loss. As to health care, s many of us have argued, employment-linked care is a dreadfully stupid.

r.
Thanks, Jon. She was a great person. We need to invest $ not only in disease treatments and cures that will be profitable and help the many, but also in those treatments and cures that are more expensive and will help the few. All people are important and we must develop a new way of funding research, targeting disease, and managing our nation's healthcare.

A pure market-driven approach is not the answer.
I am truly, truly sorry about your friend RW.
There was a "Quincy" episode about that topic that did some good in the 1980s, as to the Orphan Drug Law or whatever it was called, with Tourette's syndrome I believe triggering that effort on the episode. Maybe that will be true here too, if maybe in some weird unknowable way.
Even if its not markets, there are economies of scale issues as to research in a certain sense, and also a lot of... herd behavior effects among scientists too as to "What problem are we supposed to solve?" in Academia in general. You have to stay "on the reservation" generally speaking in any system to thrive, if, that often creates blind spots too. People in those systems also understand that too.
Given the inevitable dependence of science on grants in some version.... that's hard to get away from completely, although, as a counter-example, I met a German once, Herman the German, of course.. :), who did work on a granted only a semi-orphaned disease: cystic fibrosis. The reason he did that was that there was some billionaire whose child had the disease. Of course, statistically speaking, that type of personal motive would still tend to replicate the effort towards that of the disease in the general distribution in the population, which would leave orphans, and would probably under-provide research too if only generated by a private motive due to non-excludability of benefits in that field, unless there were more effective efforts to treat classes of illnesses, whose tie-correlation might not be obvious. For "orphans," that's probably the best hope, as to finding better classifications within the set of orphaned diseases that then yield treatment mechanisms that can be produced to get the scale economies of more common diseases, scale economies in an ultimate sense really being in terms of a version of the Law of Large Numbers: if a lot of people with a lot of resources try to solve a problem that has a solution, it will be solved over time almost surely.
But again, more than anything, I am really sorry for the loss of your friend RW.
Its .. there's never anything really right to say, other than that you offered a very loving tribute to her, which demonstrates that she was certainly not alone as to having a good friend in this world. And of course, there is the example of the Quincy episode, and ponds and stones. Ripple effects can be powerful, and when Klugman got, as my grandmother always complained, on that particular "soapbox" it had a lot bigger effect than just that effort per se, if maybe you don't get to know that for sure, but just believe maybe that was the case or will be. Take care RW, but know that you were obviously a good friend, as valuable thing as we do.
It is hard to believe that we treat orphan deseases like we have long treated orphans. Everyone is precious and needs to be treated like they are a patient of "House." Excellent post
rated with love
An interesting and well-represented viewpoint. Excellent post.
I don't have a quick answer about how we should decide which medications should be developed but I agree that the only criteria shouldn't be what can save the most; however that would actually be better than the real criteria for the corporations which is how to make the most money.

In many cases they get funding subsidies from the government then they still keep the patents and gouge the consumers.

The most blatant example of this is the African AIDS scandal.

This is blatant corporate welfare with lives on the line.
I watched in the 80's while AIDS became more and more prevalent and nothing was done because it afflicted a despised population, people that the establishment at the time, and the fucker in the White House at the time, didn't give a damn about. It's inconsistent. If the population afflicted is public enough, or vocal enough, or well like enough by those in power, then something is done. If not, you're out of luck...

Until we realize that there are areas best not left to the market, we'll have this sort of inhumanity. I'm sorry about your friend. It's tragic.

RATED.
Manhattankid: you are 100% correct. What happened to those with AIDS in the 1980s is very similar to what I discuss above. Healthcare and research funding, capital, was directed and funneled away from a disease because of the bigoted pressure of the majority and their agents in Washington, DC and corporate America.

I suppose AIDS funding and research only got off the ground once (a) the stigma evaporated and (b) corporate America realized how much money they could make from treating afflicted Westerners.

That said, many in Africa and India, who lack money, are not getting the treatment they need. And this isn't because of their sexuality, but because of their poverty.

But again, the same principle is at work. "Need" isn't playing a part, only the ability of (a) an improper majoritarian impulse or (b) a bigoted minoritarian panel to (c) direct and channel needed capital for research and treatment.
Heather A. was the one who gave me the name for my blog. She was fluent in Spanish and found me the Spanish subtitle for the banner above. She will be supremely missed.
RW - First, my sincerest sympathy for your loss. I'm sure she would have liked your tribute.

I have personally suffered from "orphan disease" syndrom. In 1975 our infant son was diagnosed with Globoid Leukodystrophy, also known as Krabbe's disease. He wasn't expected to live past two - he lived to be almost ten.

There was no treatment or research until the son of Buffalo Bill's quarter Jim Kelly was diagnosed and died from the disease. Kelly is the primary advocate for research funding. It is still basically an orphan disease.

Great post which should be read by more.

r
Rated.

Someday maybe it will all be figured out to benefit EVERYONE including those who are sick....
RW, really sorry to hear about your friend. I've lost too many to too many different things not to feel that loss.

You know, it's odd that in science, it falls to the corporations who decide to invest money in universities and research that promotes their interests at making a profit. I get it. It's even understandable, because if they only did humanitarian stuff that made no money, we'd have to have a society that no longer used money.

If only...

In the meantime, these companies, in their focused drive to invest in potential "Blockbuster" products based on the research forget that there's a veritable pharmacopia in nature that is vastly unexplored, unexamined and unprotected from loss. In their drive to target specific diseases and specific course of pathology to success, they ignore many other potentially rewarding avenues of research as well.

It's like you said, your wife said the study of these rare and infrequent diseases could be the keys that unlock how to reverse aging, stop the spread of infection, regenerat cells, prevent cancer cells from even forming, etc. But, because, someone behind a desk realizes that getting people to support Big Ticket causes brings in money in some fashion -- if only to defray the costs of their research in the hopes that they can patent a drug to treat it, they readily perpetuate the glaucoma of viewpoints when it comes to what's the best way to deal with these larger, more prevalent diseases.

It could be that they are reducing avenues of potentially rewarding research and thought by excluding these things. I believe the potential to invigorate our knowledge of health, disease curing, treatments and prevention lie in the fields less examined. The reason for my belief in this is that, the larger diseases have already yielded all their "low hanging fruit" in understanding them. By the same token, many of those who, say, become oncologists, focus on one specific type of cancer, get trained in the latest treatments and don't look beyond their own narrow field of interest to explore techniques, options, methodologies and new information that might provide them key insights into breaking past the most recent barrier to progress with the "brute force" method of thousands all trained to respond in the same way to the same diseases, based on established history of the research so far.

When it comes to funding, I think these corporations would be much better served if they expressly devoted their funding to universities with the admonition that half goes to the prevalent big time diseases and the other half goes to funding the research of the rare, the infrequent, the mysterious diseases and the study of natural remedies, homeopathic treatments and their integration into mainstream medicine.

Imagine a world where medicine has once again regained its primary focus of attention: to ease suffering, improve quality of life and do no harm. A world where doctors aren't told, "You can't study that, there's no money in it," or where research and study between disparate fields actually cooperate, communicate and collaborate on curing and understanding everything they can about the human body, because that's what's important.

A lot of things are going to have to change, but I think it can be done, even in a system that values making a profit. It's just that we, as a society, have to come to demand of our companies, that they are going to have to behave responsibly according to the dictates of providing a lasting and valued service to humanity if they're going to study medicine.

That means, like in your post, the minority diseases require the same level of consideration, study and attention as do the majority diseases, simply because it's part of how we pursue knowledge. The only way to do that is to change the perception of how effective the current methods are to increasing our understanding of disease, the body's response and preconditions to it, and how to use cross-discipline knowledge to increase our chances for success in these efforts.

Once again, really sorry to hear about your friend.

--r--
My previous post, from last night, may not be an orphan disease but maybe it is. My friend is about to die from a form of lymphoma so rarely seen (in the US anyhow) that the top doctors at three major hospitals misdiagnosed her, repeatedly. And there is no chemo that works, it turns out. I am thinking, maybe this is the frontier, where finding out if this acts more like a virus than cancer (what she repeatedly got misdiagnosed for), or auto immune than infection, or incapacitated inflammation, might be the key to opening up other treatments. We know the basic medicine for most of the major diseases not only doesn't cure them, but often causes more problems (like statins causing muscle damage, liver damage, heart failure and diabetes but only prevents heart attacks in people who have already had them). Statins are a huge financial boon, but have little to do with restoring health. For most people, consuming less commercially prepared food and beverage will do more for them, but that is a money loser and will never be supported by the FDA. Keep in mind, the FDA is only minor oversight to keep killing drugs off the market, and they are funded by the major players. They don't investigate medicine, they investigate research, which we know is not only flawed, but often faked altogether (Vioxx anyone?).
Sadly, there needs to be more research done in general, but we aren't a country of scientists, we are a country of capitalists, some of whom fund scientists, who also want to make a living. Socialist countries do no better for taking care of their orphan disease patients, but they may be more likely to fund non-partisan research. Death panels are a joke, because they don't decide who will die. They decide who they will pay for to extend life. And that is always determined by the financial bottom line. (see previous post on life, liberty and abortion).

Good stuff, Rw. Sorry about your friend. It's been a crying day here too.
RW...first of all, my condolences on the loss of your friend.

The notion of capitalism dictating what "can" and "cannot" be done in the area of medicine and medical treatment has got to end. This problem you presented here is horrendous, but the overall problem is widespread, pervasive...and completely unnecessary.

We can do so much more...were it not for the constraints of "will it put bucks in the right pockets."

With your permission, I would like to post a comment I just posted in another thread on the general issue. Please let me know if it is okay with you.
Frank, Go right ahead
Thanks. Here it is:

Fifteen years ago I went through cancer treatment at a VA hospital in East Orange, New Jersey. I was fairly new to the VA…having come to it mostly because I had no health insurance.

I had many people tell me the VA gave lousy medical service—but for the most part, who really cares how good the service is when you are going for a checkup; to have a troubled ear cleaned; or to investigate some shortness of breath.

Then cancer raised its ugly head. A particularly invasive form of non-Hodgkin;s lymphoma.

Well, I guess if the truth were told, some of the doctors and nurses seemed liked rejects—and many of the staff were less than professional. There was lots of inappropriate chit chat…and more than a fair share of rude responses to questions and requests.

But there were several doctors and nurses who were as good as any I had seen on the “outside”…and the bottom line is that they got the job done. The chemo and radiation worked. As I said, it was 15 years ago and I am still alive.

Some of the other medical treatment was not up to what I thought it should be…but it was adequate. Adequate!

What I am saying here is, socialized medicine (which is what the VA is) may not be five star…but it beats the shit out of not having any treatment from here to Hell and back. Adequate treatment may be second to exceptional treatment…but it is way, way better than no treatment at all.

We NEED a socialized medicine scheme in place in this country…yesterday. It can exist alongside a capitalistic, non-socialized system…and the people who can afford the “better” system can use it. But the ones who need medical attention and cannot afford the “better” system will still get adequate medical care under a socialized program.

Probably the treatment and care will be a lot better than just adequate.

We can train doctors and nurses…give ‘em a free education and have them make their repayment by serving in this socialized system for a set number of years. We can even pay them a very decent salary while they are serving. And at the end of the term, if they choose, they can go onto a private practice…or find positions in the non-socialized systems.

We can build, outfit, and staff medical facilities…for use by the socialized system.

WE CAN DO IT…AND WE GODDAM WELL OUT TO BE DOING IT.
Frank: I totally agree.

I see health care as being like an airplane service.

Some riders take coach, while others can take first class.

That said, all of them get to the same location. If you want to pay the minimum, you can get the basic service (which still gets you where you want to go).

If you want a giant seat, lobster, lots of liquor, free movies galore, awesome supermodel stewardesses, etc... then ride first class.

Same should apply to health care. Adequate and sufficient service should be available and affordable by all.
I am sorry for your loss.
Social goods for social need, I say. Selling medicine and treatment isn't like selling cars or toothpaste or breakfast cereal. There's a lot of black holes in the market when it comes to taking care of people, we don't break down in completely predictable ways and we're not too profitable as an investment in the long run.

England had it right with their National Healthcare laws, but now they're undoing all of that. Reeks of socialist havoc, they say, and they unleash more havoc as a result. There is a counter-revolution going on under the cover of this collapse. Fight back, organize, fight for your lives!
rate
Very important post. Unfortunately lots of non-orphan diseases - for example, Crohn's Disease - don't get treated in the US because Food Inc is suppressing the link between Crohn's and Johne's Disease in dairy herds. In Europe and Australia, people with Crohn's disease are treated (and cured) with antituberculosis drugs and a vaccine against mycobacterium avium paratuberculsosis, the organism that causes it.
Sorry for your loss Rw. Excellent post.
Boy I never thought it would end up like this myself. I share your grief and concern.
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I'm sorry, Rw, for your loss of such a wonderful and close friend. I am sure that you will do much to advance this cause and it is very sad that compassion is not a form of currency in this culture. You have done well to bring my attention to this matter and there are many who, though they may be the majority, need an advocate such as you.
I send my sincere condolences, Rw. I had never heard of chiari, so I imagine it is REALLY rare. I will be looking into it further.

Lezlie
Well, as we're dealing with lung cancer, I can assure you that diseases with lots of "parents" aren't doing all that well either.
My condolences to Heather A, and my empathy for your grief. Your wife sounds like a really smart woman, RW, I guess that’s why you married her. Ordinarily I would say that it that it made sense to concentrate limited resources to benefiting the most possible people but her point about developing a deeper understanding of disease as a whole by also funding research into some of the more obscure diseases by scientists who are inclined to do so has me no longer feeling ordinarily. She makes a great point.
My condolences for your loss.

This country has to start looking at health care as a right, not a making-making opportunity for corporations.
Thank you all for your condolences. Today was Heather's memorial service. Although I couldn't attend, because it was more than 1,500 miles away, my thoughts and prayers are with her family today. May God be with her and may she have peace and freedom from the pain that her affliction caused her here on earth.
My condolences on Heather's death.

And now comes the question:
What do you think is the best way to encourage orphan disease research?

And now a worse question:
Forgetting profits for a minute: What is not moral about trying to save the greatest number of people?

I'm not talking about the AIDS case here because that was driven by factors other than numbers - that involved the sacrifice of a given population.

I suppose one could make the case that we should, on moral grounds, concentrate on diseases/conditions where the victim isn't complicit in contracting it. That could conceivably be a good moral case but, of course, a terrible economic case and a terrible practical case.

I don't know. Regardless, today's important question is the first, not the second. I ask the second because you'll have to address it, when you're ready, in order to be effective at addressing the first, and it is by addressing the first that you will honor Heather's memory most. To save others from the grief you're going through at the moment is one great way to derive something positive from the immense negative that is your friend's death.
I am very sorry for your loss, please accept my sincerest condolences. I am frustrated continually with our health care system and how we imagine that we are actually doing constructive medical research when we realize that everything associated with the medical field is incredibly profit driven or driven on the on the business paradigm. People have to beg for money for research, and again, like health care, we should have it funded by tax dollars and managed by those with no profit driven motives. The answer is becomes obtuse in how we are conducting our American formula these days. We are driven by all the wrong incentives with respect to healthcare and research.
Sal:

I don't quite understand what you're trying to say here.
My question has to do with what happens if we have finite resources to use for research. Assuming that, we have to make choices as to allocations. Using what standards? Or do we assume that the reason we might have finite resources is illegitimate in the first place and involves spending on lesser priorities?
Sal: When it comes to biomedical research, of course, the "needs of the many should outweigh the needs of the few." That said, this should not imply "to the total exclusion of the few" or the "wholesale ignoring of the few."

While we gear the vast majority of our funding for those diseases that impact the vast majority of people, we should also study other, more rare diseases, to see if they can be easily treated or cured. They may not be common, but if a disease can be easily treated or cured, I don't see any reason to wholly ignore it. I understand why 80-90% of funding should go toward the most common diseases.

That said, many scientists think that the NIH should have at least 1 research team per rare disease cluster, so that we can investigate all of these diseases and make consistent progress in these fields of disease research and not ignore them. Indeed, the insights we find in regard to rare diseases can also have benefits in terms of the other diseases.

Targeted research isn't always the best kind of research.

For example, who would've thought that Radiation Treatment for Cancer would have been a side effect of the Manhattan Project?

Oftentimes, knowledge for knowledge's sake is important in its own right. And if it can be done while at the same time, saving lives, all the better.

Hypothetically, many scientists say that 10% of the NIH budget should be allocated toward rare disease and that each rare disease cluster should get its own "research team" consisting of at least 10 scientists.

This way, we aren't left in the dark whole of total ignorance when similar, more widespread diseases following similar patterns, pathways and the like emerge.

There are a whole gamut of reasons why we shouldn't ignore orphan diseases.

That said, I agree with you 100% that the majority of funding should go to the most common illnesses.

I just think that there should be no academic, governmental or corporate PROFIT-BASED PROHIBITIONS on promising research regarding other diseases, if they hold out a strong potential for a cure or life saving, prolonging treatment.

A subtle difference.
The fact is, according to my wife, who is a cancer research scientist at a top research institution, there is totally enough $ for orphan diseases, but the funding scheme is skewed and funding for many of them is downright prohibited, due to the cult of profit, that totally permeates the industry.

an 80/20 mix is frequently mentioned by scientists.

Let's not forget, many diseases, like AIDS, are not being treated, simply because its not profitable, even though solid treatments are available. While treatments are available, now, in the West, we need to remember that the vast majority of the people on earth who have HIV/AIDS aren't in the United States, Europe or the West. They are in the developing world. And they don't get the drugs they need because they are poor.