How quickly could an effective drug treatment for ME/CFS be rolled out once approved?

[Sasha]

Still thinking about the cure! And with the UK as an example.

Suppose an effective drug treatment is discovered and gets approved for use in the UK tomorrow. We know we've got hundreds of thousands of PwME in the UK, some in utterly dire straits (including some of us on this forum), all of us desperate to get our lives back.

But there are only so many doctors, and we don't even have a medical speciality that looks after us. If the drug is something like daratumumab, our GPs won't be able to give it to us.

In my dreams, the NHS would mount some sort of emergency protocol, like it did to give the first Covid vaccine at speed, to get all PwME treated over the course of a few months, starting with the most severely ill.

In my nightmares, the NHS just sits there and does nothing.

What would actually happen? Is there anything we should be doing now to get the best possible outcome when the time comes? (I do now think it's 'when', not 'if'.)

What happened with rituximab, @Jonathan Edwards? And are there any lessons we should learn from that?

 

[Jonathan Edwards]

What happened with rituximab, @Jonathan Edwards? And are there any lessons we should learn from that?

Not really. We have already learnt the lesson - the UK is seriously under-resourced and wastes a good bit of what it has on a crazy 'internal market'. Everything is uphill. But sometimes you get there.

 

[Trish]

I think it entirely depends on the cost and availability of the drug. If it's expensive and/or requires treatment in hospital, rich people will buy it privately and the rest of us will get nothing. If it's cheap and readily available and GP's are allowed to prescribe it, it could be quick. If it's available over the counter without a prescription we'll all be buying it.

 

[hotblack]

What would happen? Meetings
How would it be rolled out? Unevenly

I don’t mean that as a pessimistic view but that is usually how things go, even in the best cases. The covid vaccine example you gave shows what is possible, but it involves bypassing a lot of things so is unlikely to happen. And even in that example there were issues.

The NHS won’t sot there and do nothing but there will be a great deal of variation depending upon local services, who and what people know, luck, etc. If NHS England decided to push things centrally it may help a bit? But equally that may ruffle feathers and be seen as unhelpful?

 

[Sasha]

I think it entirely depends on the cost and availability of the drug. If it's expensive and/or requires treatment in hospital, rich people will buy it privately and the rest of us will get nothing.

I can't see a scenario in which the rest of us get nothing. Non-rich people with MS don't get nothing. The country can't afford to have so many of us economically inactive. And our families want us back. All hell would break lose if PwME weren't treated if an effective treatment were available.

If it's cheap and readily available and GP's are allowed to prescribe it, it could be quick. If it's available over the counter without a prescription we'll all be buying it.

That's for sure, though!

 

[Trish]

Non-rich people with MS don't get nothing. The country can't afford to have so many of us economically inactive. And our families want us back. All hell would break lose if PwME weren't treated if an effective treatment were available.

I'm thinking of the limited roll out of the latest obesity drugs. Most people who are using them are paying privately. Only those on a very restricted list are getting them via the NHS. I suspect the same would happen for ME/CFS, with a very restricted list such as those with very severe ME/CFS getting expensive meds. Of course they should be first on the list, but if it's expensive, it's sure to be rationed.

 

[Sasha]

I'm thinking of the limited roll out of the latest obesity drugs. Most people who are using them are paying privately. Only those on a very restricted list are getting them via the NHS. I suspect the same would happen for ME/CFS, with a very restricted list such as those with very severe ME/CFS getting expensive meds. Of course they should be first on the list, but if it's expensive, it's sure to be rationed.

That's an interesting comparison. But I can't see this as equivalent, since obesity has other available treatments (principally dietary, though I realise that a good diet is not available to everyone) and is not as disabling as ME/CFS.

According to Google AI::

You can get Ozempic on the NHS only for the treatment of type 2 diabetes, not for weight loss. To qualify, you must have type 2 diabetes and have been unsuccessful with other treatments like metformin, or be unable to take them, and meet certain criteria such as having a BMI of 35 or more with obesity-related health issues, or if your job could be affected by low blood sugar from other medications. For weight loss, Wegovy is the semaglutide medication approved by the NHS, but access is restricted to specialist weight managent services and requires meeting specific criteria.​

And:

Approximately 220,000 people are expected to receive weight loss drugs like tirzepatide (Mounjaro) on the NHS in England over the next three years, with a phased rollout prioritising those with the highest need. In total, it is estimated that around 3.4 million people in England are eligible for the treatment based on NICE guidelines, which recommend tirzepatide for individuals with a BMI of 40 or more, or a BMI of 35 or more with at least one weight-related condition.​

​

Initial rollout: The first phase will provide the drug to around 220,000 people over three years.​

​

Eligibility: Initial eligibility is for those with the highest clinical need, including those with a BMI of 40 or more with at least four other specific weight-related conditions.​

​

Expanded eligibility: Eligibility is set to expand over time to include a wider range of people.​

​

Total eligible population: NICE estimates that around 3.4 million people in England will be eligible for treatment over the next 12 years.​

​

Private availability: While the NHS rollout is gradual, many people are already using these drugs privately. Over 1.4 million may be using GLP-1 drugs privately, far exceeding the number currently accessing them through the NHS.​

 

[V.R.T.]

I think three things would be on our side in this situation.

A) An effective treatment would legitimise our struggle in the eyes of doctors and the public, and the NHS will want to counter the seriously negative publicity as how they've treated us seeps into the wider public conciousness.

B) Those of us who were lucky enough to get it privately would be able to be tireless activists for NHS prescription of this drug in a way that the realities of even mild ME/CFS prevent currently.

C) Patients, family members and carers would now have something concrete and simple to demand.

 

[Kitty]

I can't see a scenario in which the rest of us get nothing.

No, me neither.

What may slow things down significantly is getting evidence for who benefits. The drugs may carry significant risks, so they'll want to be sure it's more likely people will benefit than come to harm. But if NICE recommends the treatment for people who fit a set of criteria, I think it will be rolled out.

I don't think the comparison with obesity drugs is necessarily a useful one, it's such a different situation. The drugs involved there seem likely to turn out to have major drawbacks, so it's not surprising a healthcare system with a pretty conservative approach is limiting them to folk with few or no other options. ME/CFS isn't like obesity—even the most mildly affected people don't have alternative (and less risky) approaches they could try first.

 

[Sasha]

ME/CFS isn't like obesity—even the most mildly affected people don't have alternative (and less risky) approaches they could try first.

Any effective treatment for PwME is basically going to be like bringing people back from the dead. I'd expect the first severely ill PwMEs to recover to be plastered all over the news and a huge wave of public pressure on the NHS to rescue the rest of us.

 

[BrightCandle]

Way too many vested interests who would want to stop the drugs approval and I think that would be a giant fight, it won't happen overnight. Will be years of fighting to get NICE to finally approve it. Then like we have seen with cancer drugs it will involve protests to the government to get it funded and rolled out. We will likely see a very slow change over years towards treatment, the rich will just get it privately and the rest will be fighting the system for years.

NG201 Nice guidance for ME still hasn't been rolled out 4 years later, there is no interest in the NHS for ME patients at all. I don't see a drug being any different,

The lack of diagnosis is going to get in the way, no one can identify the patients. Those of us with GPs who refuse to read NICE and diagnose anyone (the majority) are not going to get a drug just because it's approved, we are still just anxiety patients refusing anxiety pills.

There are just so many issues at a government, NHS and society level that need fixing to make it possible for a quick rollout that are unlikely to be fixable anytime soon.

 

[hotblack]

Will be years of fighting to get NICE to finally approve it.

Not necessarily so. If there is clear evidence then it would be a simple job for NICE. A lot pf the decisions we see protests about are because, to be blunt, the evidence is not clear. There are treatments which drug companies charge vast sums for but have negligible advantages over cheaper treatments.

 

[Kitty]

I agree it would take time to get NICE approval (that's normal), but not that it would be withheld.

If it works well enough to return people to health, politicians are likely to read "returns people to work". I can't see them having any objections to that.

 

[Yann04]

A lot of the discussion is assuming that the drug is basically a cure/remission thing.

What if it’s more marginal?
Say a drug that prevents new worsenings but doesn’t improve “baseline”.

Or a drug that maybe gains people 10-20% on the FUNCAP/SF-36. That’s still big, especially for severe people, it would def be a noticeable effect for a well designed trial.
But it wouldnt mean people are suddenly “back from the dead”, and able to protest like hell either…

 

[hotblack]

A few links which may be useful in helping understand some of the structures and responsibilities within the NHS. It’s obviously a topic with a lot of history and politics. But if we can try to avoid that and focus on understanding how things work and who we will need to persuade, convince or lobby that may be useful? For both treatments but also for services we need before those become available.

The King’s Fund is a great resource for this. Much of the info is from before NHS England was brought back inside the DHSC but AFAIK a lot is still relevant

Here’s a simplified but easily accessible video

https://www.kingsfund.org.uk/insight-and-analysis/animations/how-does-nhs-england-work

A longer read on the structures involved

https://www.kingsfund.org.uk/insight-and-analysis/long-reads/understanding-accountabilities-structures-health-care

And a more detailed article on commissioning

https://www.kingsfund.org.uk/insight-and-analysis/long-reads/what-commissioning-and-how-it-changing

 

[OrganicChilli]

All hell would break lose if PwME weren't treated if an effective treatment were available.

I don't think so. Even in other countries protests are relatively small as we have to rely on allies and carers, but there is some awareness. I have seen nothing of the sort in the UK.

Daratumumab (if it works) can only be prescribed by specialists and they'll tell you they will only prescribe it once an ME/CFS specialist has given the green light. So your GP will send you to your local fatigue clinic run by the BPS brigade and if you're very lucky they won't block the use of an effective drug if other "interventions" (CBT etc) have failed.

 

[Jaybee00]

I’d be a lot more concerned about how the treatment would be rolled out in poor developing countries than in the United Kingdom, which is a top 15 country in human development index. How does the treatment get “rolled out” in Sudan and the Central African Republic?

This is an additional concern given that the U.S. just cut a large amount of funding for international health programs.

 

[Yann04]

How does the treatment get “rolled out” in Sudan and the Central African Republic?

If it’s something as expensive as Dara, it won’t, unless you’re rich enough to go fancy private.

 

[Kitty]

But if we can try to avoid that and focus on understanding how things work and who we will need to persuade, convince or lobby that may be useful?

I'd say one important route is to make sure a charity has the resources and the membership numbers to do things that individuals can't. Action for ME seems the most obvious candidate, which I why I joined again recently.

 

[Sasha]

A lot of the discussion is assuming that the drug is basically a cure/remission thing.

What if it’s more marginal?

You're right, I've been assuming a 'daratumumab works' scenario in which there are full responders and non-responders, so you are basically 'back from the dead' or not. I think that's a good scenario to focus on for thinking purposes, though...