Action alert: Bob Miller calls for email campaign to request hearing on Ampligen

[user9876]

I guess that shows just how dumb capitalism is?

I think in the EU any government sponsored trials may fall foul of state subsidy rules which are intended to prevent one state subsidising their industries at the expense of others. But government paying for industrial research is allowed under certain conditions.

 

[Nielk]

I take that point. But my understanding is that several thousand doses have been given - so quite a lot of people have got it. So there is another possible argument - that people should be able to buy ampligen treatment if they can afford it. But how many PWME can afford $24,000 p.a. indefinitely? And should they if we do not know it works - or even that a quarter dose might be as good?

The patients who are currently receiving Ampligen from one of the few experts in the U.S. are paying out of pocket. they are doing this at a great cost and bother of going for twice weekly infusions because it is a life saver for them. I know a few of these patients and they would be bed bound without the Ampligen. with it, they can lead an almost normal life.

By the way, the cost for example at Dr. Peterson's who is currently giving half the dose which he found works as well, is $8,000 a year. I don't think that this is an outrageous amount for a drug given as a twice weekly infusion.

I don't think the FDA is going to respond - it would be opening the floodgates to hundreds of billions of increase in insurance premiums. I think the law needs changing. Governments support vaccine programs - I bet the law as it stands could be interpreted so that a trial could be done. I think it is the NIH that needs lobbying.

The FDA is a regulatory agency who decides the efficacy and safety of drugs. There are many drugs in use right now that are more expensive and do not give the same huge relief as Ampligen does for a subset of ME patients.

The US government does not fund drug trials. It is the private drug companies who have to spend all the money on trials to satisfy FDA approval.

The efficacy approval rate for a new drug that is an orphan drug is pretty ow. I believe around 20%.

I don't think that the problem with Ampligen getting approval by the FDA has to do with safety or efficacy problem.
The issue was with hemispherx, the drug maker of Ampligen. It is a very small drug company with little revenue. They tried to cut corners and did not properly administer previous trials. Hopefully that is being remedied in their current ongoing trials.

 

[user9876]

Dear Sasha,


The proposer says that approval would bring 'insurance coverage for all'. I found a blog that said that the cost of ampligen at their clinic was $2000 per month. So if a million Americans took ampligen that would cost $24 billion straight off. Is this the best way to spend the notional cost (almost to the penny) of ME? There would be nothing left for research.

Surely an argument should be made about economies of scale and so costs for a drug currently made as part of a research programme should dramatically fall when mass produced. Or doesn't that work with the drugs industry? I guess patents mean that they can value price due to no competition.

 

[user9876]

I had a close look at the study the ampligen people quote a while back. My impression was that the results are presented in a form that is hard to interpret. They say there is a 21% improvement in activity but the bare figures suggest it is 10% and that does not seem very much. To my mind this sort of average figure is not what is wanted. We want some sort of figure indicating how many people were usefully better in each group - a bit like the Haukeland study. I fully appreciate the problem of outcome assessment for trials but if a study is properly blinded I think there are ways of assessing useful improvement that ought to stand up.

I wondered if this was the reason that the FDA don't like ampligen. i.e. that the results and safety information seem to be presented in a challenging manner. At one point they asked for new trials but I think they got a new presentation of the original data.

The other thing that surprised me about the ampligen presentations to the FDA was they didn't seem to have done any work to look at why it might work or any sub-typing. For example, if they claim it works via NK cells I seem to remember (but could be wrong) that they didn't have any data around changes before or after the drug.

I seem to remember one interesting thing from their data which was that the subjective scores were worse than their walking test scores. Kind of the opposite from PACE. I wonder if that says something about trial design in more general terms.

 

[Sasha]

I don't think that the problem with Ampligen getting approval by the FDA has to do with safety or efficacy problem.
The issue was with hemispherx, the drug maker of Ampligen. It is a very small drug company with little revenue. They tried to cut corners and did not properly administer previous trials. Hopefully that is being remedied in their current ongoing trials.

Thanks for the info. I wasn't aware that there was an ongoing trial. Do you have any details? Presumably there's a protocol registered somewhere?

 

[Denise]

Thanks for the info. I wasn't aware that there was an ongoing trial. Do you have any details? Presumably there's a protocol registered somewhere?

The current study is a open label study of Ampligen in cfs meaning:

“Patients enrolled in the study are responsible for costs related to the therapy, e.g., drug cost, infusion cost, cost of supplies, diagnostic and other laboratory testing.”

https://clinicaltrials.gov/ct2/show/NCT00215813?term=Ampligen&rank=5

 

[jimells]

Then maybe the campaign should be to make laws on testing drugs more in the interest of the consumers and less in the interest of venture capital people.

That's not going to happen. The demands of the "Captains of Finance" trump everything and everybody.

It seems to me the people who at fault here are to some extent the American public who have voted for politicians who think the current laws are ethical.

In the US voting is irrelevant. There is really only one "party", the Business Party. Repugnants and Dummycrats are two branches of the same party. Many Americans now understand this, and refuse to vote. Thus we have declining participation, which tends to de-legitimize the system, and worries Our Dear Leaders.

I don't buy the bit about Hemispherx being too small. When I have come across situations like this before with drugs in the pipeline, if the data in house look promising to independent scientific advisors like me then the company usually has trouble fighting off the overtures from big companies to buy them out. The people in the small company sell out and live the life of Riley.

Yes, I do wonder why Giant Pharma fails to see profit potential here. Isn't the patent about to run out? That would have a very negative impact on future returns on investment. Drug ineffectiveness seems to have little to do with drug approvals and sales; SSRI's and statins come to mind.

There is plenty of evidence of regulatory capture of the FDA. The list of drug company malfeasance aided or ignored by FDA is longer than my arm. It doesn't exist to serve patients, but to serve Pharma. When patients are helped, that is more of a fortuitous coincidence than by design.

 

[Jonathan Edwards]

Surely an argument should be made about economies of scale and so costs for a drug currently made as part of a research programme should dramatically fall when mass produced. Or doesn't that work with the drugs industry? I guess patents mean that they can value price due to no competition.

That's another problem with the system - the price stays the same until a competitor comes along and then hey presto it seems to be possible to reduce it.

 

[jimells]

It's at this kind of point that I hit a bit of a brick wall, I must admit. There are clearly issues to be discussed if it's the NIH that should be targeted, but this is a US problem and needs US advocates to take the lead on first having a discussion, and then leading a campaign on it.

I think that campaigns that target specific drugs or disease definitions are bound to fail in the U.S. The issues involved are too complicated to explain in 60 seconds. The focus needs to be on increasing research dollars. The other side issues are just too distracting. I agree NIH, the source of research funding, is the target, but only indirectly.

Who benefits from HHS, NIH, CDC intransigence? That needs to be the target. The private disability insurance companies like UNUM seem to benefit the most. They will only respond when the costs of "business as usual" increases. So far our feeble efforts at raising those costs (they are not just financial) have been too small to have an impact. We're like a flea trying to annoy an elephant - it doesn't even notice. I'm not optimistic we will be able to have an effect any time soon...

 

[Sasha]

I think that campaigns that target specific drugs or disease definitions are bound to fail in the U.S. The issues involved are too complicated to explain in 60 seconds. The focus needs to be on increasing research dollars. The other side issues are just too distracting. I agree NIH, the source of research funding, is the target, but only indirectly.

Who benefits from HHS, NIH, CDC intransigence? That needs to be the target. The private disability insurance companies like UNUM seem to benefit the most. They will only respond when the costs of "business as usual" increases. So far our feeble efforts at raising those costs (they are not just financial) have been too small to have an impact. We're like a flea trying to annoy an elephant - it doesn't even notice. I'm not optimistic we will be able to have an effect any time soon...

This raises a point about the wider implications of a campaign like this as a strategic move in a wider battle. If Ampligen was approved (the argument goes), other pharma companies would see the advantages in running trials for ME/CFS.

Perhaps the implications of what you're saying are that if an expensive drug was approved for ME/CFS, then the insurance companies would be motivated to apply pressure for cheaper treatments. Not sure how they'd do that, though. Just because they may benefit from the NIH not holding their end up, it doesn't mean that they have the power to influence thm.

 

[Sasha]

...but for me, the campaign I'm really waiting for is the one where we target the NIH for more funding $$$ overall. I'm hoping that's coming down the pike soon.

 

[jimells]

Surely an argument should be made about economies of scale and so costs for a drug currently made as part of a research programme should dramatically fall when mass produced. Or doesn't that work with the drugs industry? I guess patents mean that they can value price due to no competition.

"Economies of scale" don't seem to work in any part of the medical industry. And drug companies are constantly developing new schemes to keep generic prices high (I forget how they work) as well as bogus patents that extend patent protection. Imitrex, a migraine medication, is a prime example of both. Even though it has been available as a generic in the US for six years, the Rite Aid "usual and customary" (whatever that means) price is still $20 a pill, for a widely-prescribed medication.

The Treximet story is particularly galling. GSK came up with the brilliant and brandy-new idea of having patients treat migraines with both Sumatriptan (Imitrex) and naproxen. (I have been doing this for years, with limited improvement over just Imitrex.) Next they outdid themselves by putting both medications in one pill. I mean, whoever heard of doing that before?

This "new" treatment is so revolutionary and miraculous that the current price of $72 per pill is, of course, actually a bargain! I'd be more than happy to pay a $50 premium per pill in order to take just one pill, as opposed to the burden of swallowing Imitrex and naproxen separately!

Of course, there are always some people who just don't like drug companies in spite of all they have done for us:

Sumatriptan is also available in the United States as an inexpensive [well, not exactly] generic prescription medicine, while naproxen is also inexpensive and over the counter. Many doctors and pharmacists saw the addition of a simple over the counter pain reliever to sumatriptan, and the subsequent marketing of Treximet as a "new" product, with a price of over $200 for 9 tablets [now $650], as a cynical ploy by GlaxoSmithKline to continue making brand-name profits when its patent for sumatriptan expired.

I find it very hard to think the FDA is "on our side" when they approve these kind of schemes

 

[catly]

I haven't read everything in this thread-- but wonder, if the FDA approved Ampligen- would insurance cover it? For example, the FDA approved the new sleep medication, Belsomra, but my insurance won't cover it because there are "other" approved medications for sleep.

Unless they would have to cover it if it was they only "approved" medication for MECFS/SEID??

 

[Jonathan Edwards]

The patients who are currently receiving Ampligen from one of the few experts in the U.S. are paying out of pocket. they are doing this at a great cost and bother of going for twice weekly infusions because it is a life saver for them. I know a few of these patients and they would be bed bound without the Ampligen. with it, they can lead an almost normal life.

By the way, the cost for example at Dr. Peterson's who is currently giving half the dose which he found works as well, is $8,000 a year. I don't think that this is an outrageous amount for a drug given as a twice weekly infusion.

That is interesting. I think it raises a possibility for solving the problem. This sounds like a very useful group of patients. Presumably they are all responders and found to require continuing usage of ampligen. It seems that Dr Peterson has tried reducing dose to a half and found no difference. That emphasises the importance of dose ranging. What I would suggest is that a small number of these patients are enrolled into a dose variation study. The dose can be varied on a double blind basis and the patients can be asked to indicate whether or not they think their current dose is optimum. Scoring systems could be personalised or based on a standard global fatigue score or whatever people think is most relevant. You then look to see what dose variation the patients can identify better than chance. If the patients are paying the total dose over a time could be kept the same (same price) but varied over relevant periods. If patients felt they were less good there could be a clause for returning to baseline dose.

A study of this sort ought to provide evidence for minimum effective dose and in doing so robust evidence for efficacy per se (dose responses are always more robust than just placebo control). Since dosages could be varied longitudinally over several phases and all patients are known responders it should be possible to get statistically significant data with a remarkably small number of patients. In theory one could even do it with one case. If there is someone out there who can tell what dose of ampligen they are getting for their ME then ampligen can work for ME - Q.E.D. But we have to absolutely sure about the double blinding.

I am not up on the history of this but it looks to me as if it ought to be possible to sort the answer out without resorting to another large company run trial. That might not suit the FDA but it might produce data that are so scientifically convincing that some investors might wake up.

 

[Sasha]

Sounds brilliant to me, @Jonathan Edwards!

The trial that @Nielk describes doesn't have a control condition and is unblinded so I don't know what the FDA's attitude to it would be, even when it's complete (and I've no idea when that would be). Whereas in what you're suggesting, it could be double-blind and patients would be their own controls.

It would be good if there could be some concurrent research (in the same patients) to determine what makes someone a responder. I would expect that there must be some patients who are in the early stages of getting Ampligen and don't know whether they're responders or not yet.

 

[Sasha]

I think it's Dr Daniel Peterson who is big on Ampligen, @Jonathan Edwards. I have a feeling he might have attended one of the IiME conferences. Have you met him?

 

[Jonathan Edwards]

I think it's Dr Daniel Peterson who is big on Ampligen, @Jonathan Edwards. I have a feeling he might have attended one of the IiME conferences. Have you met him?

Not to speak to.

 

[snowathlete]

That's another problem with the system - the price stays the same until a competitor comes along and then hey presto it seems to be possible to reduce it.

I agree with a lot of your analysis on this (though I only skimmed the posts). But may be if there was a cost incurred by the average American via insurance premiums then there would be more incentive (not less) for government to spend money on research - to find something that is cheaper, or at least which works better. At the moment, part of the difficulty I see is that ME/CFS patients are a cheap problem. Yes there is a high economic loss through people not working but that has always been the same, so it doesn't feel like a cost. I wonder if something to change that might be a good thing even if it was Ampligen and it turned out not to be amazing. It would also be a major stimulator for drugs companies I think, because right now the message is that ME/CFS is a bad investment. You not only have to find something that works somehow, but you may have to overcome hurdles regarding how the disease is perceived too. As soon as the door is opened to one drug I think it will be seen as lower risk.

But regardless, I'm not sure the FDA are going to change their minds unless there is some new data.

 

[Jonathan Edwards]

I agree with a lot of your analysis on this (though I only skimmed the posts). But may be if there was a cost incurred by the average American via insurance premiums then there would be more incentive (not less) for government to spend money on research - to find something that is cheaper, or at least which works better. At the moment, part of the difficulty I see is that ME/CFS patients are a cheap problem. Yes there is a high economic loss through people not working but that has always been the same, so it doesn't feel like a cost. I wonder if something to change that might be a good thing even if it was Ampligen and it turned out not to be amazing. It would also be a major stimulator for drugs companies I think, because right now the message is that ME/CFS is a bad investment. You not only have to find something that works somehow, but you may have to overcome hurdles regarding how the disease is perceived too. As soon as the door is opened to one drug I think it will be seen as lower risk.

Now that is a sharp Machievellian analysis. I cannot fault it. But as you end up saying...
So maybe we need to make sure there are (to be pedantic) some new data.

 

[Sasha]

Now that is a sharp Machievellian analysis. I cannot fault it. But as you end up saying...
So maybe we need to make sure there are (to be pedantic) some new data.

How would we get that to happen?