Friday, 4 April 2014

NICE approves alemtuzumab (Lemtrada)

"Alemtuzumab is recommended as an option, within its marketing authorisation, for treating adults with active relapsing remitting multiple sclerosis."

This is what NICE has decided in its final appraisal determination, available online today.
What does it mean 'within its marketing authorisation'?
 
This is the final breakthrough for alemtuzumab in England and Wales (NI hopefully to follow soon, and Scottish Medicinces Consortium due to publish their verdict in May), and fantastic news for people with relapsing MS as it means alemtuzumab will now indeed be available on the NHS.  A great achievement; many congratulations to all involved in the development of this drug, notably the MSers taking part in the trials!  What a difference compared to the situation in the US!  
It is now only a few months until the first people with relapsing MS will receive Lemtrada on the NHS, so here's a taster of how convenient treatment with alemtuzumab can be (5 infusions in the first, and 3 in the second year)...
...and the commitment it involves to monitor for side effects:


28 comments:

  1. This will turn out very badly. Seriously.

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    1. It is choice and it is risk. I would prefer to have a choice and then choose the risk I am willing to take if I am properly informed.

      Will you get the chance to take the risk, this may depend on your neuro, there will be the "Wait and See Brigade" and maybe some "Early and Aggressive" ZeToers then it will depend on what you think about "Time is Brain" and how good you think the alternatives are.

      The European Experiment has begun and the regulators from across the pond will be watching with interest. As numbers using the compound increase the risk:benefit profile will show itself. This is what happened with Nataluzimab. I hope a similar problem does not materialise.

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  2. This is good news so well done to everybody involved in particular the two As from Cambridge, ProfG can take a little credit too for in being so engaged in getting it through the regulatory maze. I wonder if the Brain Shredder posts had any influence in focussing the mind for the need for first line options.

    Now the battle will be ensuring that the trusts will pay.

    This good news.

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    1. I also point out that without the involvement of Genzyme Sanofi this day may not have come, because academics do not develop drugs.....pharma do

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    2. Yes MouseDoctor is correct; the focus on shredding and dementia is part of a campaign to remind people of how bad MS can be and to make regulators and payers realise that there is a narrow window of opportunity to make a real difference to the lives of MSers.

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  3. This is such good news. I read it whilst at work and I actually burst into tears.For the first time since my diagnosis a year ago, I have felt hope. Huge thanks to everyone involved.

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  4. MD, Is this one of the drugs that is Neuroprotective,therefore beneficial for PPMS and SPMS too?

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    1. There was a small study done many years ago suggesting that Alemtuzumab does not stop progression. However, the question remains will it change the slope of progression. Given the promising findings with Tysabri in PPMS, then there is every reason to hope that it could offer some benefit also. If it removes the inflammatory attack it will be neuroprotective. However believe we need something else as well

      Now that Sanofi are making money on their investment they may venture into studying progressive MS.

      Is it beneficial for PPMS and SPMS....answer is I don't know

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  5. But this drug is not guaranteed to be successful for all MSers, even some newbies, right? It's not a cure, right? It's pretty dangerous, too, right?

    Why is it being hyped so much when it doesn't guarantee a prevention of progression?

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  6. I am not sure if readers of this blog who live in the UK realise the enormity of the NICE appraisal: "Alemtuzumab has a UK marketing authorisation for treating adults with relapsing–remitting multiple sclerosis with active disease defined by clinical or imaging features". This will allow us to treat-2-target of NEDA more easily as this is the first drug that we can use based on either clinical or MRI criteria of disease activity. This has been a long time coming; hopefully NICE will eventually allow us to use MRI activity to define activity for the other DMTs.

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  7. This is great news. I was on one of the early trials. I had highly active MS with disabling relapses. 8 years on after my fist infusion I have had no relapses and stable on the EDSS. I work full time in a professional job. My deficits all relate to early relapses. I had an MRI (brain and spinal cord) and the excellent Dr Coles informed me that there was no sign of disease activity / no new lesions. The follow-up after treatment is not that onerous. I received excellent after care and monitoring. For those who, looking at the risks / benefits, think Alemtuzumab is a treatment option, my advice is to start as soon as possible after diagnosis (if your disease is active). A very good day for RRMSers.

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    1. Should have said the MRI was last month.

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  8. All of this seems like hot air. Genzyme's (a Sanofi company) shares are trading massively today. It's a good day for all with a vested interest in alemtuzumab right now. This drug is like the Emperor's New Clothes. It is a cash cow that we have to wait decades to see if it works. By then all guilty parties will have their golden pensions in tow.

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    1. Damn another miss opportunity.

      I suspect it is a good day for people without a vested interest also. It is more choice. You can take it or You can leave it

      You will know that it works...your relapses stop! You don't have to wait years for this.

      Is it a cure? That will take time to answer and will not be a straight answer I suspect.

      Based on animals studies and such an approach. Start early and the answer can be yes (not always) start to late and the answer maybe no (We know this last part is also the case in humans and published data), Don't start and the answer is no.

      So the question asked is how early is early.
      I don't know. Cambridge will have most insight on that one, but they may not know either but the earlier the better.

      This is true for all drugs I suspect

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  9. I think it is a good alternative to Tysabri for those with aggressive disease. I wonder if the FDA will change its stance.

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  10. I find it difficult to read some of these negative comments. The decision by NICE is life changing for MSers.Yes there are potential side effects but at least we now have a choice. MS is a hideous disease.It ravages the body and here we have a drug that can slow progression and possibly allow us to reach a good age without being bedridden.I will take my chances with Alemtuzumab. We are lucky to have it, to have the choice,finally.

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  11. Prof G,would you give this drug off label to people who are progressive?
    How do you treat progressive patients? Just looking at other Countries,it seems they are more likely to things that we are not.For example,Saud Sadiq treats PPMS patients with Intrathecal Methotrexate,as do some European countries. While success is not guaranteed,the outlook tends to be more risky and positive at the same time. There is also Ivig-I know of someone in the US who was on this,and it helped her. A lot.
    I do feel that in the UK the Neurology community are too conservative. I think progressive patients get it rough here. Can this not change,so that we don't have to wait ten years for something that might help us,if anyone can be persuaded to study it? Why not take a chance,with the consent of the patient,and feed date information in a central way,so that you can build a therapeutic picture?
    It doesn't really matter what drug,just something that has had some research done in it.

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    1. Re: "Prof G,would you give this drug off label to people who are progressive?"

      It all depends how you describe progressive MS. The pathological changes that drive progressive MS are there from the very beginning. They are already there in people with RIS (radiologically-isolated syndrome). Therefore when we treat MSers with RRMS we are already treating progressive MS.

      Would I give it someone who has already missed the boat and is the SPMS or PPMS phase? Possibly, provided they were still having superimposed relapses and evidence of focal enhancing lesions on their MRI. I don't buy into a pure clinically-based classification system of MS. I think we need to classify MS into those forms with focal inflammatory lesions and those without ongoing focal inflammatory events. In the latter case I would not give alemtuzumab; however, I would love to see a combination therapy trial done with say alemtuzumab plus laquinimod in this population. The combination of a potent anti-inflammatory therapy and a neuroprotective drug makes the most sense to me when tackling non-relapsing (MRI-ve) progressive MS.

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  12. A real dangerous drug

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    1. Re: "A real dangerous drug."

      I agree, but MS is a really dangerous disease. Alemtuzumab is associated with a large number of side effects and complications, which can be managed and treated. Once MS has shredded the brain there is little we can do to fix it. That is why early highly-effective treatment to prevent damage is the logical treatment strategy to tackle MS.

      The good thing about alemtuzumab is that it is now a 1st-line drug and it provides options. If you don't line the risks you don't have to have it. It is all about being empowered and being in a position to say Yes or No. It is worth celebrating that fact.

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    2. A real dangerous drug....I agree so it leaves the door open for someone else to fill that slot with a less dangerous drug. With £60,000 reward at least at the end of that rainbow. Will a less dangerous drug work as well....quite possibly

      There are already contenders knocking on the door. Will any company hold their nerve and go for an induction therapy with any of the anti-B cell therapies or will the skangos (New word for greedy executive see yesterdays comments) hold out for the repeated long-term £15,000p.a. treatment verses the the quick fix £60,000?

      As it is the Grand National Today we can say "Game On"

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  13. Prof G is a hero for being the man behind this.

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    1. Whilst profG played an unsung role that his modesty will keep quiet I am sure he will send his hero worship to cambridge and to the people and MSers.

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  14. I saw we need to wait for something better and safer. This drug is a potential killer.

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    1. I agree. The risks are big. This is not something to decide on the flip of a coin.

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    2. Some people think disability is a better option than death - I envy those who do especially if they have actual experience of disability to inform their opinion. I, however, know that I would likely choose death over unrelenting disability. People have a choice as to how they feel about these things, and in the same way I think people should have a choice of what treatments to take. Anyone who doesn't like the risk-benefit profile of any treatment will be at liberty not to take it. I would suggest that while the risks aren't negligible, they aren't as high as the flip of a coin.

      I for one would absolutely be willing to take the risk of alemtuzumab to try to slow things down, given the very considerable risk those with untreated MS endure. I would absolutely take the risk of some physical disease over the psychological and physical devastation of MS.

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