#OffLabel

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I am speaking at the Multiple Sclerosis International Federation (MSIF) Access to Treatment and Healthcare meeting next week in London. My talk is on “Off-label opportunities, barriers and risks in availability and affordability”.

My journey to this point goes back 5-years and started when I was on sabbatical and was visiting countries all over the world and seeing how MS was managed. I soon realised that MSers living in resource-poor environments had poor access to MS disease-modifying therapies and other MS services. This led us to formulate an Essential Off-Label DMT list and to start a social media campaign using the hashtag #OffLabel to get people to adopt off-label prescribing of DMTs. We also developed a protocol for off-label cladribine use and have distributed it widely. 

These activities and other factors nudged the MSIF to make ‘access to treatment’ one of their priorities and led to the MSIF submitting an application to the WHO to get three DMTs, albeit licensed DMTs, onto the essential medicines list (EML). If successful (hopefully we will hear next week – fingers-crossed) we will be able to use this as a springboard to raise awareness of untreated or under-treated MS across the world. It will also raise awareness about MS in low prevalence areas and challenge the prevailing dogma that MS is a rich-world problem. 

Please note our essential off-label DMT list is ‘evidence-based’ or at least anchored to licensed DMTs. The following is a new version of the list.

  1. Azathioprine*
  2. Dimethyl fumarate (generic, licensed for psoriasis)
  3. Cladribine
  4. Cyclophosphamide*
  5. Fludarabine*
  6. Leflunomide
  7. Methotrexate*
  8. Mitoxantrone
  9. Rituximab* 
  10. HSCT

*drugs that are on the 19th WHO Model List of Essential Medicines (April 2015)

I am particularly interested to hear stories about off-label treatments in your countries and if any of you are receiving off-label treatments.

Apart from rituximab use, particularly in Sweden, off-label prescribing is simply not being adopted. Why? I think it takes more than a few people standing on a soap-box to make it happen; what is required is a systems change and a whole lot of nudges to get HCPs to start doing it. This is why I have become so interested in behavioural psychology and behavioural economics, which teaches us why information is simply not enough to change HCP behaviour.

CoI: multiple

About the author

Gavin

Professor of Neurology, Barts & The London. MS & Preventive Neurology thinker, blogger, runner, vegetable gardener, husband, father, cook and wine & food lover.

20 comments

  • Go tell it to policymakers that can actually facilitate the changes you’re seeking, not publishing it here to an audience of no-ones. You’re just using this blog as an ego-trip.

    • Pardon… I am reading this, and I am not a “no-one.”

      There should definitely be more therapies available, off-label or not, across the board. Since important, profound voices are summarily quashed by big money and bigger government, perhaps a movement of culture jamming aimed at the ‘true cost’ of drugs and government stonewalling may elicit serious consideration if disruptive enough.

      We have a peculiar culture of off-label use here in the U.S. The list would be too voluminous to post here. Also I am not proficient enough to compile a useful list for answering your query, Professor.

    • It’s this type of mentality that is hindering the advancement of MS treatment. PATIENTS have the loudest voice but only in numbers. Start talking to your neurologist about exploring off license treatment options, join a lobby group. Make YOUR voice heard.

      • I’ve tried making my voice heard to my Neurologist in XXXXXXXX. He is not interested so I’m coming to see Prof Gavin as I want to hear what is available for me. Thank you. x

  • I was once prescribed Melatonin as an off-label medication, before it was licensed. But then, even once it was licensed, I couldn’t get it, because I never had a doctor who was that bothered about me. I have it now, but only because I went private and now get to see neurologists.

    I wonder if Yorkshire counts as a ‘resource-poor environment’ or if that has nothing to do with why the NHS wouldn’t let me see a neurologist for decades, get tested, get an MS diagnosis, and get treatment?

    Anyway, I want Lemtrada or HSCT, I’m stuck on beta interferon which makes me more ill in itself, and I can’t go on like this.

    So I hope some doctor cares enough to take the risk of giving me something better, because I don’t have anything left to lose.

    • Resource poor…depends which part of “Gods own” your happen to be talking about

      If your disease is not contrlled you should have the discussion on escaltion

    • I get Melatonin for migraine. It’s a relative new idea, treatment for this. My GP was not able to prescribe due to good old Nice. So the Neurologist was happy to give a first prescription and if it helps my GP will continue. I have failed other migraine treatments.

  • Prof G can you please update me with regards to rituximab being offered as a as an off-label drug which I believe was potentially happening soon

  • Comments from our Troll from Leicester removed.

    We are doing trials of off-label agents notably in progressive MS. E.g. STAT2, lipoic acid etc Do you think neurologists will start prescribing if these trials are positive?

          • Hard to say, but i wonder if biotin is symptomatic? They claim rapid effects neuroprotection will take time to show itself

        • MouseDoctor
          November 30, 2018 at 9:45 pm

          There is not point in imagining..pharma wont touch this with a bargepole

          Now to your wish about “neuros and the MS Societies” I can’t say anything but you would be wrong to suggest that they are not talking about lipoic acid.

  • MD where are things at with regards to biotin and lipoic acid? For example I thought biotin had been shelved finally

    • Biotin…..I dont know. They withdrew their licencing application to allow them to get more data. I am not sure if they have given up.

      ProfG/DrK may know as they are more involved. I have not paid alot of attention to it…I wasn’t very impressed when the story was presented. I think this is probably another example of trial kills drug. It was never really clear what it was doing and it looked like a symptomatic/remyelination drug not a neuroprotective drug, yet they did neuroprotective trials….drug down toilet again.

      Lipoic acid. I guess the Oregon group will be having another go…All I can say is watch this space

      • If on high dose biotin, remember to stop taking it 3-5 days before your bloods are taken. Really important if you’ve received alemtuzumab. It interferes with the assay used to measure thyroid function (and a bunch of other tests). Just a heads up to those experimenting with this supplement.

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