Thank you: EMA MS Workshop

Are you for patient choice? The EMA is not; what should we do about it? #MSBlog #MSResearch #ClinicSpeak

“I would like to thank you all for completing the survey I posted last week to get some data for my presentation to the EMA on a MSers perspective in relation to the staggered 2-step approach. Whilst preparing my talk, which I did at the last minute, I realised that I made a mistake in accepting an invitation from the EMSP (European Multiple Sclerosis Platform) to speak on behalf of MSers. The EMSP should have taken the opportunity to have an MSer speak. I suspect the EMA did not agree – they don’t like being reminded that their decisions impact on peoples’ lives – hence my invitation.”

“I tried to focus my talk on the early hidden disabilities that MSers suffer from, early in the course of  the disease, that indicates how serious MS is as a disease; i.e. employment, quality of life, anxiety, depression, fatigue, divorce, suicide, etc. If we apply  the staggered 2-stage approach to treatment, using the EDSS, as our yardstick by the time MSers start becoming disabled we have missed the boat. People with MS need the option of early highly-effective treatments and it should be up to them and their neurologists to decide. Our survey results are clear on this.”

“More worrying is drug development using the staggered -2-step approach. The EMA want Pharma to develop drugs in series; i.e first do a trial in MSers who are more advanced and have or are failing current DMTs. Once your drug has been shown to be effective and safe in this population to only then start a trial in MSers who are naive to treatment. The serial, rather than parallel, approach will add an extra 4-5 years onto the time it takes to develop DMTs. This is unfair to MSers and Pharma. Why delay access to an effective treatment? The 4-5 year delay reduces the patent life of the drug and hence it will push up the price of drugs substantially. Pharma need to make money; if the can’t see a finish line why take the risk?”

“I was criticised by one of my colleagues for using case scenarios to make a point. He said I sounded like a surgeon describing my last surgical success. It is difficult to give a patient perspective without giving real-life examples. The two cases I presented illustrate what could happen if you delay access to highly-effective therapies; you run the risk of acquiring fixed disability and never catch-up to those who access the treatment from the outset. Unfortunately, our predictors of who is going to do well or poorly on the staged approach are to unreliable to use clinically. The point I was making is that it is up to you as people with the disease to discuss  things with your neurologist, make and informed decision and choose one or other option. It is horses for courses.”

CoI: multiple

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.


  • If what you are proposing is a substantial change in how neurologists approach and think about MS and its treatment, then I'm not surprised you're receiving criticism. It takes a generation to change a deeply embedded paradigm. You might get more results more quickly by approaching young neurologists, or medical students.

    Actually, teaching medical students that it is important to treat early would do a tremendous service. Part of the problem is the length of time it can take a GP to recognize and refer for early MS symptoms.

    Thank you for your efforts. The fact that you are getting pushback suggests that you are being heard.

  • Re – “ This is unfair to MSers and Pharma. Why delay access to an effective treatment? The 4-5 year delay reduces the patent life of the drug and hence it will push up the price of drugs substantially. Pharma need to make money; if the [sic] can't see a finish line why take the risk?"

    Erm… Don Giovannoni, I’m pretty sure that even if we were to extend a drug’s patent life these big pharmaceutical giants will still charge the same amount of money. It will just give them more of a timeframe to maximise profits. You can’t be seriously so patronising as to suggest that Big Pharma will under price novel DMTs if we elongate their patented property, can you? Have we really become as feeble in your eyes as for you to think we will swallow such pathetic reasoning?

    Blimey, things must be far worse than I thought. It seems like Team G have become a very expensive ship without a rudder. No wonder these MSers feel so let down and misdirected.

    • Dr Dre, selective reading again. Prof G is talking about the shortening of the patent life not extending it. You really need to get down to spec savers and buy yourself a new set of glasses? Or has MS reduced your cognition to such a degree you can't read plain english?

    • Yes it is a case of will they do the studies? If it is going to take so long to do them and when they finish someone else can make a generic drug as the patent life will of expired. Where is the incentive to do the study?

    • I am not calling for a patent extension just some pragmatic thinking in relation to the development time of a drug. Extending that by 4-5 years due to potential or unknown safety concerns increases the risks of investing in the field. Alemtuzumab went into an MSer in 1992 and got a license in 2013; 21 years later. Increasing that development time to 25 or 26 years would have been ridiculous. In fact I doubt Genzyme would have done the studies.

    • 21 years is ridiculous.
      Wasn't there a plan awhile back to use spinal taps as an outcome measure to shorten the time that trials take and to test drugs on progressive patients? I thought that was smart.

  • "Do a trial in MSers who are more advanced or who have or are failing therapy". This concept is unclear; if patients are more advanced and no longer respond to immune modulating therapies are they not considered SPMS? If they are initially progressing without remission are they not PPMS? Failing DMTs would imply non-RRMS. Use of therapies in non-RRMS seems to be analogous to treating viral infections with anti-biotics only benefitting drug companies.

    • Are they considered SPMS…not necessarily you can have someone on say beta interferon with RRMS and their disease begins to breakthrough say because they have developed neutralising antibodies to the interferon you may then switch treatment to say tysabri or gilenya. These people still have RRMS.

      Say you have a new great drug you want to get it to people as soon after diagnosis as possible but under this two stage approach you would first have to try beta interferon before getting the option of have to test the new one. This will serve to keep the injectables going for some time further. I wonder if this would be supported by some pharma who have got nothing in their pipelines.

      P.S. You should not think about treating viral infection with an anti-biotic, it won't work because these are designed to get rid of bacteria

  • I kind of agree with Dr Dre on this one. Never thought that he and I could ever see eye to eye, but on this we do.

  • Sounds like an excerpt from a 19th century Academy of Science meeting….

    "I was criticised by one of my colleagues for using case scenarios to make a point. "

    Ignore the reality. Nothing to be seen here. Move, move…thanks, for your cooperation.

    • Alemtuzumab was developed using a parallel approach; i.e two phase 3 studies recruiting together. If it was developed using the 2-step approach we would only have it licensed in 4-5 years time. A delay of 4-5 years may be a problem for some MSers and would be a problem for Genzyme who need to make money out of their investment in the MS field. Demanding a 2-step approach will simply scare away Pharma investment from the field at the detriment to MSers.

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