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

[A.B.]

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.

Maybe the people making the decisions are not aware of the magnitude of the problem.

Maybe they're not convinced that this is a disease and not just human nature (laziness).

The message should be that it's a real disease and a big problem. Coincidentally this is pretty much the message of the IOM report.

 

[Jonathan Edwards]

How would we get that to happen?

We keep powder dry until an opportunity arises, in my experience.

 

[Nielk]

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.

The greatest cost to the US government and the private insurance companies from ME/CFS is long term disability. Very few disease render a patient disabled for decades. Most very serious diseases will kill the patient in a short time, ME/CFS can render severe patients totally disabled for 30-40 years. This can total to over a million dollars per patient in disability payments.

This is exactly what the government and insurance companies are trying to avoid.....by marginalizing and minimizing the true disability of this disease.

 

[medfeb]

[QUOTE="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."[/QUOTE]

Not sure how that would work as long as patients are paying for their own drug, which they do in the current cost-recovery protocol, it seems like it would hard to do a double-blind study - they'd know by the size of the check they write.

The following quotes from FDA meetings might be useful/relevant to this discussion
At the 2013 FDA ME and CFS drug development workshop...
- Dr. Woodcock, Director of the FDA, said that drug development efforts were challenged by the lack of clarity on endpoints and on patient selection.
- Ms. Roth, Director of Regulatory Affairs at Lily, said more clarity was needed on a) the criteria used to describe the drug indication (the specific disease condition for which a drug is approved), b) the endpoints to be used for registration and c) what threshold is needed to demonstrate the “substantial evidence” of drug effectiveness required for drug approval

At a separate meeting, another FDA official said that she had heard from Pharma companies that they were concerned that they would "have difficulty getting reimbursement for approved products because the definitions are so wishy-washy that insurers may not be willing to pay for a product."

Ms. Roth's comments make me think about the challenges that pharma would have in developing a drug label when the disease is defined by Fukuda. The definition is so broad and encompassing, how would a clinician know what portion of that patient population a given drug would help.

 

[SOC]

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.

No. The company that spent all the money on the research is allowed to have a number of years of exclusive production to make up the cost of the research. Once that time period is over, the cost is reduced because the development costs are deemed to have been recovered (although I'm sure the pharma companies wouldn't agree with that). At that point it becomes legal for companies who didn't invest in research to simply copy the drug and manufacture it at lower cost.

We would never get a pharmaceuticals company to do the necessary research to develop a drug if we allowed other companies to immediately copy the drug and sell it at a lower cost. The development research has to be paid for somehow. That's just common sense.

 

[SOC]

To the best of my knowledge the US government does not fund drug development. That's done by private industry. Does the UK government fund drug development? If so, which drugs were developed by the UK government and how was the pricing determined? Are those drugs much cheaper, both in the beginning and in the long run, than drugs developed by private industry?

 

[NK17]

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.

I wonder if @Jonathan Edwards can talk about Ampligen with Dr. Peterson since he will present and attend this year 10th Invest in ME conference in London.

 

[BobM]

Once again, thanks for engaging in this kind of discussion! We need these things thought through critically.



That's just it - my understanding is that people can't get Ampligen unless they're in some weird scheme. Bob Miller was getting it because his doctor was in that special scheme with Hemisperx - he had to move himself and his whole family so he could be near to that doctor to get the infusions administered. I'm not sure if that scheme was stopped - if my memory serves, the clinicians were getting Ampligen under some special arrangement to do with research. I wonder if it was to provide safety data or something.



Bob (and Cort, I think) claim that the FDA is approving drugs with a similar safety/efficacy state of evidence as Ampligen for other conditions; there certainly seems to be a lot of weirdness about how the FDA do things and my understanding is that the FDA is seen by many in the US as being in the pharma industry's pocket.



I'm not sure about your cost calculations on this. An estimated 90% of US PWME don't have a diagnosis, so we're only talking about 100,000 patients at this point (though that could change if the IoM recommendations are rolled out); not all doctors are going to prescribe it; and it may become apparent quite quickly what subset of patients benefit. So the real figure could be more like $50/year maximum; and one would hope that some patients would do so well on Ampligen that they wouldn't need to stay on it.

In terms of whether I'd be happy to pay $X (whatever X is) for drugs of questionable efficacy for other common conditions, I think that would depend on whether those conditions already have any kind of treatment available: ours doesn't.



People were disappointed, I think, at the time about how Hemispherx presented their data. If they presented it the way you've been suggesting (scatterplots of individual patient data) that would be useful.



I think I'm sounding like some sort of major apologist for this campaign because I posted it and then bumped it so that the Ampligen afficionadoes here on PR would be aware of the campaign. But I'm aware that the data as presented by Hemispherx aren't as strong as they might be and I agree that in a sensible world, the next thing to happen should be further, high-quality trialling of Ampligen and the dose-assessment that you're suggesting.

But that doesn't seem to be an option: the US govt agencies are legally barred from trialling Ampligen (as I understand it) and Hemispherx are too small a company to be able to afford to do it (article on that here by Cort - who is very pro-Ampligen).

That leaves people like Bob Miller with only one option, it seems to me: to campaign for the FDA to approve Ampligen as is.

If you were Bob, what would you do?

How else can he move the Ampligen issue forward?

Sasha, this thread has just been brought to my attention, I am happy to address the posts here, but not tonight. I have a doctor appointment in the morning, so sometime in the later day... The "scheme" you are referring to is set by the FDA, it's called the compassionate care cost recovery trial. You can have the treatment if you pay for it. Those thinking does the drug work ? I and many other patients have been on Ampligen for years, for me personally it's been 15 years, I am not wealthy, so having paid $24,000 a year for 15 years should tell patients something. I have the best clinician in this field, so that should tell patients I have ME/CFS, my labs and scans prove it. As for the cost, it is like any other product, making large batches will reduce the cost. Making batches for less than 100 patients is very costly. Also compare Rituxan in cost 2 treatments are around $70,000. As for why.do I push for approval, I have seen many patients go from bed bound & home bound to working again. Two are clinicians. Some no longer require treatments, some like me do. We are all different. Ampligen is an immmune modulator, it's does much more than boost NKC. As for me, if I had to I could access the treatment without trying to help others get it. I hope this helps until tomorrow.
Thank you,
Bob Miller[/QUOTE]

 

[BobM]

Sasha if you would flag these posts, so I can further address the issues I would appreciate it. I gave my reply, then it was gone, I have found it after 20 mins. But would like to finish my response tomorrow.

Thanks Again,
Bob Miller

 

[oceiv]

@BobM Looking forward to your posts tomorrow or as you're up to it. I just started reading about your and your wife's impressive ME/CFS advocacy. I knew only bits of the story before @Sasha (thanks) pointed me to your FB page.

Here are my questions:

• Is there an online timeline of your advocacy efforts somewhere besides Facebook? (For people who don't like/use FB)

• Who is the FDA Action Team?

• You said on HR that your email campaign targets staff of congress members. Which congressional members? Why were they chosen as the ones to target?

monica.volante@mail.house.gov; sarah.curtis@mail.house.gov; Elizabeth.brown@mail.house.gov; dennis.sills@mail.house.gov; mark.ratner@mail.house.gov; james.paluskiewicz@mail.house.gov; scott.dziengelski@mail.house.gov; thomas.power@mail.house.gov ; janet.woodcock@fda.hhs.gov; Nancy.Lee@hhs.gov

• Would you talk about the governmental bias against approving drugs for ME/ CFS vs. approving experimental treatments for other illnesses.

Thanks very much for your advocacy.

 

[oceiv]

Here is an explanation from the FDA of how the drug approval process works in the U.S..


It's more complicated than presented, because politics is involved. Both FDA politics as well as congressional politics. Others probably have more details. Also, the current system is set up to benefit larger drug companies. Smaller companies like Hemispherx are at a disadvantage because the system requires large upfront costs

@jimells IMO, the people, in the relevant government agencies, who have pushed ME/CFS as an imaginary illness or as a psychological illness (still real, although these people don't see it that way) stand to benefit most from the status quo. Also, psychiatrists and psychologists in the field who claim to treat ME/CFS.

@Sasha The wider implications of Ampligen being approved by the FDA are that other U.S. drug companies would see profit in developing other drugs for the illness and would then be more likely to do so. Insurance companies here don't necessarily push medication prices down. The drug may be less expensive for only people who have that particular insurance company's coverage. Hospitals have had some successes in pushing several drug's prices down, but not across the board for all drugs.

@catly Insurance companies would at the very least have a choice as to whether to cover it, but if Ampligen is approved, coverage could still vary from one insurance company to the next.

@snowathlete There is currently a cost to all insured for many expensive drugs for many different illnesses and procedures. Insurance companies benefit because they get more money too. There is little incentive for them to reduce insurance coverage prices. There is now a new requirement that insurance companies spend a certain percentage of premiums on medical costs. If this requirement is kept and not loosened, we will likely see a cumulative effect. The lack of incentive to keep insurance premiums down is one reason why many Americans have been priced-out of being able to have insurance. The cost of the uninsured is then shifted onto the federal government (medicaid, etc), states and to the remaining insured people.

Thank you @Jonathan Edwards for your insights and advice.

 

[Sasha]

Sasha, this thread has just been brought to my attention, I am happy to address the posts here, but not tonight. I have a doctor appointment in the morning, so sometime in the later day... The "scheme" you are referring to is set by the FDA, it's called the compassionate care cost recovery trial. You can have the treatment if you pay for it. Those thinking does the drug work ? I and many other patients have been on Ampligen for years, for me personally it's been 15 years, I am not wealthy, so having paid $24,000 a year for 15 years should tell patients something. I have the best clinician in this field, so that should tell patients I have ME/CFS, my labs and scans prove it. As for the cost, it is like any other product, making large batches will reduce the cost. Making batches for less than 100 patients is very costly. Also compare Rituxan in cost 2 treatments are around $70,000. As for why.do I push for approval, I have seen many patients go from bed bound & home bound to working again. Two are clinicians. Some no longer require treatments, some like me do. We are all different. Ampligen is an immmune modulator, it's does much more than boost NKC. As for me, if I had to I could access the treatment without trying to help others get it. I hope this helps until tomorrow.
Thank you,
Bob Miller

Welcome back to the forums, Bob, and many thanks for your helpful comments. We're a bit short on detail on the background to Ampligen here and my memory of the issues is clearly very fuzzy.

We're very fortunate to have Prof. Jonathan Edwards posting here, who is the advisor to the Invest in ME UK Rituximab trial and was the researcher who came up with the idea of B-cell depletion using Rituximab as a therapy for rheumatoid arthritist (in which it's now the standard therapy, as I understand it) and is therefore very familiar with issues of drug development and approval. He has raised some very interesting points - I'm looking forward to your discussing them, if your energy allows. We're all interested in there being a good way forward.

Sasha if you would flag these posts, so I can further address the issues I would appreciate it. I gave my reply, then it was gone, I have found it after 20 mins. But would like to finish my response tomorrow.

Sorry, Bob, I don't understand what it is you want me to do!

 

[Sasha]

A new development:

http://forums.phoenixrising.me/inde...hile-seeking-formal-regulatory-approva.36090/

 

[snowathlete]

The greatest cost to the US government and the private insurance companies from ME/CFS is long term disability. Very few disease render a patient disabled for decades. Most very serious diseases will kill the patient in a short time, ME/CFS can render severe patients totally disabled for 30-40 years. This can total to over a million dollars per patient in disability payments.

This is exactly what the government and insurance companies are trying to avoid.....by marginalizing and minimizing the true disability of this disease.

I agree. It's much cheaper to deny us support (something governments are well practiced at) than it is to research and treat the disease. And that's been the case for so long that the cost there is from that doesn't get taken into account much - it still doesn't feel like a cost.

Anything that changes that position will be beneficial to us I think, whether ampligen or something else.

 

[jimells]

Sorry, Bob, I don't understand what it is you want me to do!

Maybe a tag to @BobM would help him find this thread?

 

[jimells]

IMO, the people, in the relevant government agencies, who have pushed ME/CFS as an imaginary illness or as a psychological illness (still real, although these people don't see it that way) stand to benefit most from the status quo. Also, psychiatrists and psychologists in the field who claim to treat ME/CFS.

As we have seen in the UK, the pschobabblers are closely connected to, and paid by, disability insurance companies. Since at least one major UK insurer, UNUM, is also very big in the US (with the same shameful history of denying benefits, including my former partner), it seems likely to me that insurers in the US are fully behind HHS/NIH/CDC policies towards this illness.

It would be interesting to know just which former government officials are now, or will soon be, working for these insurers...

 

[Anne]

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?

Not to speak to.

You'll get a chance at the conference and colloquium in London in May!

 

[Jonathan Edwards]

No. The company that spent all the money on the research is allowed to have a number of years of exclusive production to make up the cost of the research. Once that time period is over, the cost is reduced because the development costs are deemed to have been recovered (although I'm sure the pharma companies wouldn't agree with that). At that point it becomes legal for companies who didn't invest in research to simply copy the drug and manufacture it at lower cost.

We would never get a pharmaceuticals company to do the necessary research to develop a drug if we allowed other companies to immediately copy the drug and sell it at a lower cost. The development research has to be paid for somehow. That's just common sense.

Actually, I was talking about a competitor drug - like abatacept coming along to compete with anti-TNF in RA or a different type of anti-TNF. Once health care systems can choose between two competing drug classes with the same indication then a price war starts. Clearly the patent situation applies if there are no competitor drugs. But if there are then prices start sliding once one company sees its market being nibbled by another. The list prices may stay unchanged but price-cutting goes on in the system. This is something relatively new that has occurred with these high cost biologics and injectables.

 

[Jonathan Edwards]

[QUOTE="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."

Not sure how that would work as long as patients are paying for their own drug, which they do in the current cost-recovery protocol, it seems like it would hard to do a double-blind study - they'd know by the size of the check they write.

I think that is easy enough to deal with. The physician and the patient agree that the patient will receive the same total amount of drug as they are used to, over a one or two year period perhaps. During that period they will sometimes receive half dose and sometimes one and a half dose - maybe for periods of three months. If patients cannot detect a difference in effect then the dose can be reduced (and increased) further until the patient can tell the difference. Patients might be concerned about the effects of dose reduction but if so the protocol could be offered only to those for whom a trial of weaning off was considered anyway. It would need some careful thought but if patients are still receiving five times as much drug as they need (which would seem possible) then it would be in everyone's interest to find that out.

 

[medfeb]

I think that is easy enough to deal with. The physician and the patient agree that the patient will receive the same total amount of drug as they are used to, over a one or two year period perhaps. During that period they will sometimes receive half dose and sometimes one and a half dose - maybe for periods of three months. If patients cannot detect a difference in effect then the dose can be reduced (and increased) further until the patient can tell the difference. Patients might be concerned about the effects of dose reduction but if so the protocol could be offered only to those for whom a trial of weaning off was considered anyway. It would need some careful thought but if patients are still receiving five times as much drug as they need (which would seem possible) then it would be in everyone's interest to find that out.

Thanks for the reply. Interesting idea.

I guess the question would be whether the effect of lowered dose could be felt within the allotted time window. I think I remember one patient saying that she was off Ampligen for a year before she relapsed. Of course, with effective monitoring as part of a study, the differences felt as a result of a lowered dose might have been detected in a shorter window