OffLabel & PoliticalSpeak: changing off-label to unlicensed

HSCT-BMT had been added to our essential unlicensed treatments list #PoliticalSpeak #MSBlog #OffLabel

An important issue we, and others, have tried to address is the global access to effective DMTs for people with MS (pwMS) who live in resource-poor settings. On a personal level you need to ask yourself what it is must be like to be a pwMS living in a resource-poor country? This remains my primary motivator for this policy initiative. The good news is that this policy will hopefully be taken forward by new partners with political influence. 

What I have learnt is that awareness is simply not enough to change behaviour. To change behaviour you need policy, carrots and sticks (incentives and disincentives), and nudges to make it easier to change your behaviour that not to change your behaviour. In my next life I plan  to be come a behavioural psychologist and/or politician. In this week’s BMJ their is a great Editorial on ‘Redesigning healthcare to fit real people‘, which states that it take an average of 17 years for proven healthcare innovations to be adopted. If our Essential Barts-MS list of unlicensed therapies takes 17 years to be adopted then we will have failed the 100,000’s of MSers living in resource-poor settings. We can’t let this happen.

After some thought and discussion and exchange of emails with some patient advocates I have decided to add HSCT/BMT to the essential list. HSCT is not a ‘drug‘ therefore I have had to change the list title to ‘essential unlicensed treatments‘. The evidence-base that HSCT is an effective treatment is increasing and as it is available in many resource-poor settings, where it is cheaper than many of the imported high-cost innovator DMTs, I see no reason why it can’t be used. 


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

Please note that w e continue to have a lot of resistance to the adoption of our essential list from many ources; internally from within our University (Policy Unit), Pharma, colleagues (from both resource rich and poor countries) and even several of the MS Charities. The good news is that at least some of the charities now realise that this issue is at least worth discussing. 

Our QMUL policy unit feels that there is far too much inappropriate off-label prescribing in the world and that we should really stick to the policy of only prescribing on-label. Pharma clearly opposes it as it challenges their very lucrative business model. 

Colleagues in resource-rich countries feel the legal risks of prescribing off-label are an issue and they don’t like double-standards; why should they apply lower standards to treating pwMS in their countries? Why can’t pwMS in resource-poor countries have access to the same DMTs with appropriate risk:benefit assessments to those living in richer countries? Many people favour lobbying Pharma to make available licensed DMTs at a cheaper price to pwMS in resource poor countries. Although this option sounds easy it difficult to implement; the logistics of getting innovator products into these markets is not insignificant and once you do there are whole lot of local issues that need to be addressed. 

We have an open survey running to see how much support we have for the off-label prescribing component of our policy. Although over 80% of you are supportive of our efforts the response rate has been very low in terms of overall numbers, which indicates to me that this is not a major issue for readers of this blog. I find this depressing; surely this issue is worth fighting for?

CoI: multiple

6 thoughts on “OffLabel & PoliticalSpeak: changing off-label to unlicensed”

  1. Your audience, mostly from developed countries, have access to DMTs. We are selfish: we need better access, better care and a cure for MS in our well off countries… This is why you initiative for poor countries do not get much success here. It is not our focus.But you are right it is is worth fighting for all patients. First for ethical reasons. Plus, ultimately, these cheap drugs will help manage the prices of DMT everywhere. The best ones will get registered for MS or imposed off-label in our well-off countries to sustain the overall MS cost. And this will challenge pharma to invest in other molecules to get closer to a cure. (Don't claim this loudly, this is the worst pharma nightmare)It is a very nice battle to make the world better. But please don't get burned out/killed by this task and by its enemies, we need your research!

    1. Who knows?. We get page views but this is influenced by Bots, people will return less because we are answering comments more slowly.

  2. But you're not now advocating packing a suitcase and heading from the UK to Mexico or Russia for HSCT are you?

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