Open 2022 Pilot study in Norway - Daratumumab in ME/CFS

[Yann04]

We can’t gamble with dangerous drugs just because there is a chance that it might work.

Who should take responsibility if it goes wrong and people get worse or die?

Who should cover the resources for proven treatments that other sick people are deprived of because we used a limited healthcare budget on experimental and unproven treatments?

Everyone wants treatments as soon as possible. But there are many very good reasons that we do trials first.

maybe see it more as a cost benefit analysis. When does the probability and weight of risk outweigh that of reward. I've been in periods where I was thinking if I stay this bad for another 3 months I don't want yo live anymore. Imagine in a case like that I think it would be ethically dubious to withold a drug with stunning results in a Double Blinded Phase 2 RCT. Of course, bureaucratic and institutional rules mean that sort of sceenario is basically impossible unless you're rich and paying for a drug at a private clinic.

The most loogical thing of course (Imagining the data from this and the phase 2 RCT hold up) is to have an accelerated phase 3 trial with emergency drug approval for ME once the results come out if they are positive.

But of course, we are still far from any of this. in such a small sample, the HLA differences could be randomness. We haven't even had a Controlled and Blinded trial yet. etc

 

[V.R.T.]

You have not really addressed any of the points I highlighted, so I don’t really see any reason to go back and forth saying that we disagree with each other. There are other threads dedicated to the considerations with regards to trialing drugs, so we should try to avoid derailing this thread any further.

But you did not address any of my points. You acted like I was saying i want random untrialled drugs administered because i think a drug should be fast tracked for approval.

Who should take responsibility if it goes wrong and people get worse or die?

Was rolling out the covid vaccines fast a bad thing? People had bad side effects then. Sometimes a situation demands accelated approvals.

Who should cover the resources for proven treatments that other sick people are deprived of because we used a limited healthcare budget on experimental and unproven treatments?

This doesnt really deserve a response because its a ludicrous zero sum game fallacy. Again you keep talking about experimental and unproven treatments and not accelerated approval of a drug tested by a group widely agreed to be among the best ME researchers

 

[V.R.T.]

Nobody is advocating for random untested drugs to be given to patients.

 

[Utsikt]

You acted like I was saying i want random untrialled drugs administered because i think a drug should be fast tracked for approval.

I called it experimental and unproven. Not random.

 

[V.R.T.]

I really am puzzled by the response here sometimes.

I keep being told there's all this hope then in the same breath that treatments are a decade away and any hint of accelerated approval is tantamount to administering drugs to patients with no evidence.

 

[Utsikt]

Was rolling out the covid vaccines fast a bad thing? People had bad side effects then. Sometimes a situation demands accelated approvals.

A vaccine like that is far safer than Dara, and covid had far higher fatality rates than ME/CFS will ever get close to.

This doesnt really deserve a response because its a ludicrous zero sum game fallacy.

We do not have unlimited resources, so you have to always consider the alternative cost. That’s basic economics.

Again you keep talking about experimental and unproven treatments and not accelerated approval of a drug tested by a group widely agreed to be among the best ME researchers

It does not matter who did the testing if the data is unsufficient - something it very well might be after a phase 2 study.

 

[V.R.T.]

A vaccine like that is far safer than Dara, and covid had far higher fatality rates than ME/CFS will ever get close to.

If death was the only catastrophic negative outcome from illness worth considering, none of us would be here.

 

[V.R.T.]

It does not matter who did the testing if the data is unsufficient - something it very well might be after a phase 2 study.

If half the patients go into a complete lasting remission like in the pilot and cyclo studies how could that ever be insufficient?

 

[Utsikt]

If death was the only catastrophic negative outcome from illness worth considering, none of us would be here.

It was meant as an example, not an exhaustive list of negative consequences.

I’m taking a break now.

 

[Jaybee00]

Can go from phase 2 to approval


https://ascopubs.org/doi/10.14694/EdBook_AM.2012.32.114

 

[Jonathan Edwards]

If half the patients go into a complete lasting remission like in the pilot and cyclo studies how could that ever be insufficient?

A good number of people went into remission in the early rituximab trials but a formal controlled trial showed no effect. Life isn't that simple.

 

[V.R.T.]

A good number of people went into remission in the early rituximab trials but a formal controlled trial showed no effect. Life isn't that simple.

Didn't they cut the dose in half in the phase 3 that showed no effect? Or was that Ampligen?

But yes, unfortunately life isn't that simple because otherwise when your doctor tells you your symptoms are being caused by depression and you need to exercise you wouldn't end up with a severe form of one of the worst illnesses in the world.

Life is fiendishly complex but there is no excuse for making people as sick as me wait so unbearably long to try something that has a good chance of helping us.

 

[Jaybee00]

A good number of people went into remission in the early rituximab trials but a formal controlled trial showed no effect. Life isn't that simple.

OK but Fluge and his group are not stupid—they’ve learned from the ritux trial. E.g, they are measuring steps, which I don’t think they did in the ritux trial.

 

[EndME]

OK but Fluge and his group are not stupid—they’ve learned from the ritux trial. E.g, they are measuring steps, which I don’t think they did in the ritux trial.

It is precisely because they are smart that they will wait for larger controlled trials.

 

[Jonathan Edwards]

OK but Fluge and his group are not stupid

I know Oystein well and, no, he is no way stupid but by jingo he is likely to have the biggest placebo effect of any physician just because he is so committed and so human. Eyes need to be wide open

 

[V.R.T.]

Okay apologies all for crashing out the other day.

On reflection I think as @Yann04 says the most sensible/hopeful course of action is an accelerated phase 3 if phase 2 goes well.

Apologies @Utsikt I took our disagreement a bit too personally. It's a difficult time for me at the minute.

 

[Utsikt]

Okay apologies all for crashing out the other day.

On reflection I think as @Yann04 says the most sensible/hopeful course of action is an accelerated phase 3 if phase 2 goes well.

Apologies @Utsikt I took our disagreement a bit too personally. It's a difficult time for me at the minute.

No worries - and apologies back for being brash. We’re in terrible circumstances and I completely understand your desire for things to move as quickly as possible.