OffLabel: Is the use of off-label DMTs justified in 2015?

Off-label prescribing. Is it justified? #ClinicSpeak #MSBlog #MSResearch #OffLabel

“After last week’s post on off-label prescribing, I contacted our health policy unit about formally addressing this issue in the UK with the aim of producing a policy document. Surprisingly, I have got a lot of push back from the unit. Their position is that there is too much inappropriate off-label prescribing in the world and that we should really stick to the policy of only prescribing on-label. If their position is correct then we should not be investigating and promoting off-label DMT usage for MSers in resource-poor healthcare environments. Do you agree?”

“I don’t agree. The incentives for licensing a drug from the Big Pharma perspective are financial. In comparison the incentives for academia are usually because of an unmet need in a particular patient group. The problem is that without a financial case academics, and the NHS for that matter, don’t have the resources for licensing a drug or to maintain a license once one is granted. This then creates a Catch-22. I personally think there is a massive unmet need for off-label prescribing in MS. This is particularly pertinent that we have now changed our therapeutic aim in MS to treat-2-target of NEDA. We now have something to aim for regardless of which DMT we prescribe. Clearly the issue of off-label prescribing is not relevant in developed markets where there are laws to protect Big Pharma investment in novel innovative therapies. What we are trying to address is the unmet needs in environments where MSers simply can’t afford innovator brands. Would you rather be on an off-label DMT or no DMT at all?”

Proposed essential off-label DMT list

About the author

Prof G

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


  • Yes you should be able to get off-lable DMT.
    If you are willing ot take the risks and if there is medical data to support off-lable use (which is true in MS for all these DMTs).

    I really wonder why Mitoxantrone is only off-label in the UK. In Germany it's a "regular" DMT within the so called "escalation process" (if MS is highly active or gets severe very fast, or other DMTs have proven not to work)

    There are a lot of orphan diseases and the off-label-restrictions should be lowered so these people may have a chance to survive!

  • seven ideas….seven battles to fight

    Based on recent studies azathioprine is as effective as beta interferon and as leflunomide is the precursor of abagio then they will be in the same league of low efficacy

    Rituximab will be effective but it maybe has a high cost. The drugs that are probably in the same league are mitoxantrone and cladribine (maybe cyclophophamide but at what dose) but will neuros risk using them. Cladribine in my opinion is probably as effective as any of the current active DMT, but in many cases probably safer.

    Cladribine is the only induction therapy and could cost $350 US price, funnily enough Chinese price would be about $10,000 and UK about $2000 at current generic prices. Maybe the World MS societies and neuros should club together to get the studies done properly (with one or possibly more of the above), maybe fight one battle well and hopefully win.

    Merck serono let the cladribine fish go, maybe out of altruism they could support the "MS drugs for all" to bring an MS drug to countries that can't afford MS drugs.

    Oops I have just woken up from my dream and and back in the real world. Maybe we just have to wait until 2019 for generic fingolimod to hit the streets 🙂

    • Wouldn't surprise me….to think that Copaxone patents are still alive when it was described about 40 years ago shows us how lawyers can spin a story and protect a market……

  • I am all in favor of off label use of DMTs – just because they have not been proven effective to be in treating SPMS and PPMS according to the rigorous study guidelines and outcomes, we have not proven that they do nothing beneficial at all and they don't cause harm. In other words, those DMTs may just be helping SPMS and PPMS anyway and every little bit of help people with those variations of MS receive, is important, right? I know that was a rambling way to say yes, I agree.

By Prof G



Recent Posts

Recent Comments