Help: EMA MS Workshop

H
I need your help with my talk at the European Medicine Agency MS Worksop. #MSBlog #MSResearch


“I have been invited to speak at the European Medicine Agency’s workshop on MS this coming Thursday. I am representing the European Multiple Sclerosis Platform (EMSP), a European MSer lobby, on the staggered ‘two-step approach’ for treatments with ‘profound’ effect on immunity.Christoph Thalheim approached me to speak on behalf of MSers after my talk at the TIME MATTERS satellite presentation at the European Neurological Society meeting.”



The EMA has decided to provide an opportunity for the different stakeholders to come together and discuss the key scientific issues in the field of MS. The stated goal of the workshop is to make sure that in the revision of the MS guideline EMA can take into consideration the most up-to-date, state of the art scientific developments in MS, as well as the positions of the experts in the field on the main topics in the guideline, which are:

  1. New outcome measures in MS: possible better ways to assess disability that overcome limitations of the EDSS.
  2. New perception of disability – including cognition, fatigue, pain and other symptoms specifically related to MS.
  3. The staggered “two-step approach” for treatments with significant effect on immunity:
    How to address safety concerns in new drugs with significant effect on immunity?
    What data on safety should be presented at initial MAA?
  4. Changing MS population, definition of “insufficient treatment response”, and their impact on the benefit-risk assessment of new drugs.
  5. The need for placebo in controlled trials in multiple sclerosis and how to design them.
  6. Patient reported outcomes, biomarkers and novel methodologies, and their role in the development of new MS drugs.



“I could simply talk about my treatment philosophy, i.e. early effective treatment, but I am not sure this is what you would want me to do on your behalf. Another strategy is to imagine myself as someone with MS who is as an MS activist; what messages would I want to get across? I only have 20 minutes. Should I present my campaign to rebrand MS a preventable dementia? Should I focus on MSer rights and access to active monitoring? If you have any suggestions please let me know; I need your help.”


“May be I should present my MS clinic list from last week? It was full of patients with moderate to severe disability who are now in the secondary progressive phase of their disease. I also saw two new patients with PPMS; a couldn’t offer them any DMTs. What would the same clinic look like in 20 years time when the next generation of MSers have had access to early highly-effective therapies?” 



“I have uploaded the EMA programme for you.”

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.

11 comments

  • I think we should have access to early effective treatment after CIS to prevent disability & other problems. Also more basic & clinical research funds.

  • I think you should make a case for early effective treatment with DMTs and if poss also the idea of rebranding as a dementia.

  • If you believe in neuroprotection and believe neuroprotective DMT's shall be first line therapy, since they will probably benefit all kinds of MS ( PPMS, RRMS and SPMS ) and sequentially the course of the disease, I believe this is what you shall focus on in your presentation.

  • I also agree that a case should be made for access to early highly-effective therapies. There needs to be an emphasis on 'the window of opportunity' and the requirement for more active monitoring.

  • Thank you for asking. I like almost all of your suggestions. I think early effective treatment, preventable dementia and mser preferences and activism would all be useful. I think one patient story, or one for each topic would be enough.

    For something you haven't mentioned, I'm not sure neurologists fully recognize just how demoralizing the slow inexorable course of this disease is. Something quickly fatal but with a chance of cure would be in many ways preferable. Waiting, knowing things will get worse but not when is not easy at all. I'm too unwilling to hurt my family, but the statistics on suicide match the felt experience of the disease. This ties into decisions whether to take risks on Stem cells or alemtuzumab. It should definitely go into the calculus re making drugs available and which ones.

    The standard drugs have been around long enough for patients to see first hand or through meeting msers who have taken them that they don't solve the problem. Whether they noticeably mitigate the problem is a perspective that neurologists may have but we don't. No research study is going to be as convincing as first hand or immediate second hand experience. Some may be more patient and optimistic or have obligations to small children and prefer a lower level of risk. You can't make the risk tolerance decisions for us. In an ideal world approved drugs at every level of risk would be available for us to try on our timetable, not one prescribed by authorities.

    Perhaps something else to study would be ways to assess and find the few of us who may be cognitively impaired enough to really not appreciate risk. But being an intelligent person, writing those words about cognitive impairment and imagining that that could be me in a few years really goes to the horror of this disease. I understand why you pulled back the curtain. I hope you convince the right people.

  • I'm an MSer since 4yrs. If I would participate I would lobby for

    (1) basic research for finding the MS cause (e.g. by creating a fund of 3% of marketing expenses of big pharma), and
    (2) long term studies of therapies showing risks/benefits in the long term* (e.g. by creating a fund of 2% of marketing expenses of big pharma).
    (3) changing the panelists and speakers since important stake holders are missing, i.e. there are only money "receivers", no MSers

    * Currently, the long term evidence of benefits/risks is not sufficient for me as MSer.

  • Should I focus on MSer rights and access to active monitoring?
    Yes!
    and on
    Patient reported outcomes!

    For example I now have a device (I won't mention the name out of fear the mouse doctor will delete this post) that can track

    Activity
    per day and per week
    steps
    distance (km)
    elevation (m)
    activity (mn)
    active cal / day
    total cal / day

    Sleep
    per day and per week
    time in bed total, from – until
    hours of sleep
    expressed as a % of 8 hours

    minutes to fall asleep
    hours awake

    hours light sleep
    hours deep sleep
    number of wakenings

    etc
    And it is possibility to share these data with your doctor by email

    This is just an example, but no doubt other devices are possible as well. You yourself suggested developing an App for this purpose.

    • I'd be interested in knowing what device it is… I have recently bought really neat pedometer that does the first 4 things on your list, but not the sleep data.

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