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Harvard Medical School MED-ED: Medicine's Missing Measure (video)

Waverunner

Senior Member
Messages
1,079
This is one of the best talks I have seen in a while. Juan Enriquez talks about our current problems in healthcare. Drugs get more and more expensive but we cure nothing. He gives a perfect overview of why we don't progress in healthcare. [When I tell you, that there was one FDA employee who not only caused a big traffic jam every morning he drove to work but also disapproved every single drug that was shown to him (so nothing could happen to him), including renal drugs and if I then tell you, that this special employee died of renal failure, you wouldn't believe me. This talk is very exciting and eye opening.

A talk by Juan Enriquez, managing director, Excel Venture Management; chair, HMS Genetics Advisory Council. Harvard Medical School MED-EDs are a series of short, thought-provoking presentations by renowned HMS faculty, alumni, and leadership volunteers built to share and inspire new ideas.

 

Sparrowhawk

Senior Member
Messages
514
Location
West Coast USA
Not that I'm advocating the wild west, but where else in the world might have a rigorous approach to medicine but a not quite so fossilized regulatory and approval process as the US?

We patients are literally being asked by scientists (current Lipkin thread) to donate our own money to finance their research but if there's little chance of approval for therapy on the other end of that research, well I don't even want to paint that picture.

Several people on this board have mentioned that we need an equivalent effort in terms of PR and lobbying as was mobilized for HIV/AIDS funding. Many differences in circumstances between the two diseases of course but I imagine some barriers are lifted in bureacratic circles if Congresspeople are putting the pressure on to fast track drug approvals.

But back to the idea of a better location for R&D and drug approvals. For a few decades I recall Switzerland being seen as the place for cutting edge medicine for those who could afford it -- I've never heard that from anyone with CFS so I'm not sure if that only applies to cancer. Does it matter to a multinational where its drug is actually licensed? The US is a big market but it is certainly not the only market. How long before the drug companies decide to offshore their R&D like the tech companies did, with cheaper costs, fewer limitations on trials? Maybe they have already and I am insufficiently informed.

I mean, we're willing to try just about anything that can be shown to halfway work, viz the thread about people heading South to slather tree frog excretions on themselves. If we had at least some assurance that our advocacy and dollars would lead to a useful drug approval I've no doubt we'd pull out the stops. But if we are limited to the current regulatory environment here, stateside...

I say all this as a person of two minds, because we have also seen really dangerous drugs become approved even in the highly regulated environment in the US and that's the last thing I would wish on any patient population. Less regulation may not be the answer. But lower-time-and-cost-to-trials would be nice.
 

Waverunner

Senior Member
Messages
1,079
Sparrowhawk: You made some very nice points. Let me throw in my two cents. Fast track drug approval is not what most people think it is. Fast track means, that you cut down the FDA approval time by about a year (from two to one year). Fast track does not mean, that you require less clinical trials or studies. So for a normal drug it takes about 11 years to get approved. With fast track it takes about 10 years because most of the time is consumed by the R&D efforts and FDA requirements (phase 1-3), not by the time the FDA reviews the results before approval.

Even if companies go abroad e.g. Switzerland, they have no chance of selling these drugs in Europe or the US unless they come up with the required studies.

Safety is a good thing and nobody wants to be harmed by drugs. The question is when safety becomes an innovation-inhibitor and the benefits of safety do not offset the benefits from otherwise approved innovative drugs. Our current approval system is much too old and much too rigid. The world changes every day. Our knowledge changes, our technology changes and we change. The same is true for our understanding of drugs. Today we know much better, what might harm us. However, the FDA approval system doesn't account for the progress we make. In my eyes it causes the mess we currently see. Small companies or clever scientists cannot enter the pharma market because it is nearly impossible for them to raise billions of dollars and spend ten years conducting trials for the FDA before their drug could get approved. So who is left? Well, the big pharma companies are left and as Juan Enriquez said, they spend double the amount for sales and marketing than for R&D.

In my eyes there are only two possible solutions. One would be to get rid of Phase 2+3 and maybe some other requirements. Instead you could require the CEO's of a pharma company to take the drugs they produce themselves, in order for them to make sure, that the drugs are safe (no company has the interest to harm people, it would ruin itself). This would cut down approval time by half and reduce cost by about 50%. In this case the doctors have to stop blindly following what FDA approved. They and their patients have to start thinking for themselves. This may sound difficult but actually it's just a matter of habit. The other solution would be for government to start a second moon mission, this time in the pharma field. Government wastes so much money everywhere. This money could be used for drug development. If government said, that it wanted to cure cancer, IBD, HIV etc. within 10 years, they could do it in my eyes. They just need to formulate a clear mission to scientists and how they want to get there. I can give you a good example for IBD and what we would need to do:

1) Start sequencing microbiomes for every patient with gut problems (cost for 16S rRNA sequencing: 10-20 dollars per patient and sample). As soon as the results of tens of thousands of people are in, we analyse them, we see correlation and we would know what role the microbiome plays for disease. The next step would be to order labs to produce probiotic strains. The current strains, which are sold in the super market are mostly useless. We need the 500 other strains, living in our guts, which are not sold. Start treating patients, sequence again, see if the microbiome changes. If the microbiome changes and the patient is healthy we know it was the microbiome, if the patient is still ill we might try other bacteria, if this doesn't help, the reason has to be somewhere else.

2) There was a study about viral infections (enterovirus) in Crohn's patients. They were all positive in a study, while only 1 of 15 controls was. Start checking for viruses in gut samples of IBD patients. Check the tens of thousands of results. If a viral infection seems to be there, start developing an antiviral drug that works. Stop suppressing inflammation (this is what ALL current IBD drugs do) and start treating patients. Are they healthy? Yes. Case closed. Are some still ill, go on.

3) We know that genes are implicated in IBD. Ask the best scientists all over the world to develop a working gene therapy scheme (they already have excellent ideas with high safety). Replace the causative mutations. Case closed.

Only government has the resources to do so but instead of producing innovation, government inhibits it. Imagine if government did this for IBD. Within ten years these illnesses would be gone. Government could sell the drugs 100 times cheaper to the patients because it is no company. Currently the IBD patients produce billions of dollars of direct and indirect cost every year. These costs would be gone. Not to mention all the other illnesses, that would benefit from this research (obesity, IBS, colon cancer etc.).

The HIV/AIDS protestors were a great thing. The problem is that they were dying, they had a clear cause and they were a homogeneous group. They had all the requirements for a group to be successful. People with CFS are very heterogeneous, we don't know the cause and many people don't take PWCs for real. But I still hope, that things change now. It can't be, that we have to pay for our own trials. Governments wasted trillions of dollars in Afghanistan, Iraq etc.. Instead of bombing people and starting wars, we should be curing patients!
 

Sparrowhawk

Senior Member
Messages
514
Location
West Coast USA
Thanks for the excellent, thorough response, Waverunner.

I'm a bit tuckered out today so I'll need to keep this brief -- please understand it's in no way a reflection on how thought provoking and on point I found your above.

1. Recognizing that the big Pharma companies may not be motivated to do drug research outside the US or EU due to the deisre to sell in both countries, at some point I still think some bright boy/girl will have a light bulb go off: "there's X billion people in India/China/rest-of-world, who gives a damn about these developed highly regulated markets, let's go for sales there. And thus will be born the kind of really fast trials that may be dangerously lax but may also allow drugs stuck in the pipeline here to get to other markets first. Then we will be in the status you describe of Caveat Emptor, and patients will need to order drugs from India (like they do now with LDN), but the'yll do it if they have enough evidence from other users that it works. Whether developed countries will allow that to be legal...I couldn't say. You have seniors continuing to order meds from Canada due to price difference, and I don't see anyone stopping them.

2. On the IBD -- the way folks are bypassing all of the funding and time you are talking about is admittedly not without risks, but they are homebrewing their own fecal transplants after vetting potential donor(s). This has been touched on in a few threads here on the board already. The reporting from several of the related web sites I read is that it is at lease WAY more effective a therapy for Crohn's and Ulcerative Colitis than anything else available. Big Pharma may want to patent bug cocktails, but until then, the full gut biodiversity needed to heal may be as close as the nearest next healthy human. At that point the main barrier is just our cultural squeamishness about the whole thing. I'm thinking it's still easier than flying to South America to swab tree frog sweat on my skin...
 

Waverunner

Senior Member
Messages
1,079
Thanks for the excellent, thorough response, Waverunner.

I'm a bit tuckered out today so I'll need to keep this brief -- please understand it's in no way a reflection on how thought provoking and on point I found your above.

1. Recognizing that the big Pharma companies may not be motivated to do drug research outside the US or EU due to the deisre to sell in both countries, at some point I still think some bright boy/girl will have a light bulb go off: "there's X billion people in India/China/rest-of-world, who gives a damn about these developed highly regulated markets, let's go for sales there. And thus will be born the kind of really fast trials that may be dangerously lax but may also allow drugs stuck in the pipeline here to get to other markets first. Then we will be in the status you describe of Caveat Emptor, and patients will need to order drugs from India (like they do now with LDN), but the'yll do it if they have enough evidence from other users that it works. Whether developed countries will allow that to be legal...I couldn't say. You have seniors continuing to order meds from Canada due to price difference, and I don't see anyone stopping them.

2. On the IBD -- the way folks are bypassing all of the funding and time you are talking about is admittedly not without risks, but they are homebrewing their own fecal transplants after vetting potential donor(s). This has been touched on in a few threads here on the board already. The reporting from several of the related web sites I read is that it is at lease WAY more effective a therapy for Crohn's and Ulcerative Colitis than anything else available. Big Pharma may want to patent bug cocktails, but until then, the full gut biodiversity needed to heal may be as close as the nearest next healthy human. At that point the main barrier is just our cultural squeamishness about the whole thing. I'm thinking it's still easier than flying to South America to swab tree frog sweat on my skin...

Sorry for my late answer. While I also hope that China or India play an important role in the developing of drugs, I still have my doubts. The first problem is, that these countries also require nearly the same tests and trials as we do (http://blogs.nature.com/tradesecret...nderstanding-the-chinese-ind-approval-process). I'm an absolute layman but if I remember correctly you sometimes even need longer trials in China, than you do here. Moreover you are forced to share some of your knowledge about your drug, which some companies refuse to do because they fear that intellectual property is stolen. I have been thinking about other countries over and over again but drug development requires certain skills and infrastructure. If you go abroad you lack the infrastructure and if you stay here, you suffer from over-regulation. Even if you develop a drug abroad, how do you want to market it? You won't get any high rep journals to publish your results, so you are stuck with all the thousands of snake oil seller on the www. Even if your product is great, it will be hard to compete with them. Currently you can buy a lot of cheap drugs from India but these drugs were mainly developed in the first world. India is no drug developer, it's more a drug copier and seller.

As you said, many fecal transplants are done at home with no surveillance. This is a very good example of what our healthcare system is doing wrong. Instead of helping people it prevents people with IBD or IBS from reaching working treatments or even a cure. All they can do currently is to get some anti-inflammatories from their doctors. Instead of finding out why fecal transplants work, government does nothing but moreover prevents scientists to produce probiotics because they lack a billion of dollars for approval. Now you are stuck with people doing microbiome transplants at home. Either government gets out of the way or it starts looking for cures and treatments. Otherwise we will be stuck with the current system, where no innovative treatments are developed because they actually could cure someone.
 

Sparrowhawk

Senior Member
Messages
514
Location
West Coast USA
OK I did not know that about China regulatory environment.

I'm coming at this from kind of a "invisible hand of the market"/post-National commerce standpoint, where the self-interest of multinationals eventually entails their moving their R&D to the least regulatory environment they can find, and selling to those who can afford the therapy, regardless of where they site production, or where the patient population may be. Cutting out all the middle men, if you will. Dystopian vision, to be sure.
 

Waverunner

Senior Member
Messages
1,079
OK I did not know that about China regulatory environment.

I'm coming at this from kind of a "invisible hand of the market"/post-National commerce standpoint, where the self-interest of multinationals eventually entails their moving their R&D to the least regulatory environment they can find, and selling to those who can afford the therapy, regardless of where they site production, or where the patient population may be. Cutting out all the middle men, if you will. Dystopian vision, to be sure.


The invisible hand of the market could play a very important role in the future. As soon as drug development gets better and easier (gene therapy would be a perfect example) and as soon as people find out, that it actually works and is relative safe (you wait a few years till enough fast adapters have tried it) country borders will stop medical cures the same way as they stop cocaine and other drugs from flowing into the country right now. With this I mean, that they can't stop it. They don't stand a chance. Demand and supply drive the market, not government intervention.