Down, but definitely not out!

The WHO rejected our application to get glatiramer acetate, fingolimod and ocrelizumab/rituximab onto the Essential Medicined List. Why?

The following is the relevant section from Executive Summary:

“The Expert Committee recognized the public health need for effective and affordable treatments for multiple sclerosis (MS) but did not recommend the addition to the EML and EMLc of glatiramer acetate, fingolimod and ocrelizumab at this time. The Committee acknowledged the application’s approach to increasing access to MS treatments by prioritizing selected treatment options. However, the Committee noted that some relevant therapeutic options for MS were not included in the application (azathioprine and natalizumab) or were not given full consideration (rituximab). The superiority of presented medicines over other therapeutic options in the outcomes considered (benefits, harms, affordability) did not clearly emerge. The Committee would, therefore, welcome a revised application which comprehensively reviews the relative roles of relevant available medicines for MS. “

For the committee to recommend we look at azathioprine again, when the WHO themselves rejected azathioprine in 2015, is odd. To the best of my knowledge, nothing has changed in terms of new data since 2015 to change Azathioprines position as a potential off-label treatment for MS.

As for natalizumab, we did not add it to the EML application because it is still on patent, i.e. expensive, it needs to be given as a monthly infusion, which adds to its expense, and is associated with a high monitoring burden for PML. The latter is would be very difficult in resource-poor environments. At the moment the PML JCV serology assay is controlled by Biogen so when natalizumab comes off-patent, and say natalizumab biosimilars emerge, what will happen to the international JCV serology monitoring system that currently exists? Would the WHO take it over from Biogen? Would it be distributed to national labs? Will resource-poor countries be able to incorporate this into their already over-stretched systems?

We went through all these factors in our deliberations and came to the conclusion that 6-monthly ocrelizumab, and rituximab if ocrelizumab is not available, would be a better alternative to natalizumab. Another factor was that ocrelizumab is now licensed to treat PPMS. If we excluded ocrelizumab from the list what message would this send out to PPMSers in the world? In addition, the monitoring requirements for anti-CD20 therapies are much less burdensome than natalizumab.

So we take the punch on the chin, get up and start working on the next application that will be due in 2021. We are a resilient group and we owe it to people with MS all over the world to get them access to effective DMTs.

The following is the MSIF’s press release and the agenda for their meeting in London this Thursday and Friday. Instead of a celebratory mood, I suspect the atmosphere will be more sombre.

We may have lost a battle, but we have not lost the war.

28 thoughts on “Resilience”

  1. So glad you’ve taken this up, that the WHO is receptive and that you’re going in for round two.

  2. You lot have zero clout out there. The only fans you have are your clueless blog readers, and you’re lucky to even have them.

      1. I’m willing to pitch in and help. Collate documents etc. Basically, do the crappy stuff that sucks up everyone’s time. It’s a serious offer. You have my email.

    1. Nope! Very far from London here, and delighted to have got the Brain Health message and ‘switching the pyramid’ approach from my MS team. Was given a copy of Brain Health shortly after diagnosis.

      Change takes time and money, we are lucky to have researchers and clinicians like Prof Giovannoni (and the others involved!) promoting our cause so it happens as soon as possible.

      1. “. Was given a copy of Brain Health shortly after diagnosis. ”

        I’d prefer a neuroprotectant but sounds like you were happy to get
        a lousy pamphlet labeled brain health….to each their own.

      2. I get your frustration with the lack of treatments for progressive MS. Here’s the thing about the Brain Health initiative, it gives you something to throw at your neurologist and health service when they tell it will be five months before they can get the work up done and another month before treatment commences. It’s a standard that you can hold them to ensure timely diagnosis and treatment. It allows the patient to say ‘that’s not good enough and does not meet global expectations for treatment. How about you read this document you asshat’.

        The asshat comment can be left out if you actually like the neurologist in question (if they are telling you five months leave it in. I did and it worked a treat) 😀.

        That’s the real value of the document.

    2. Really? Clueless? Zero, what exactly are you doing to try and help the MS community? A member of any lobby groups? Been on any trial steering committees (as a service user or otherwise)? Helped pwMS connect to support groups or services? Contribute or take your diatribe elsewhere.

      1. Innit…..oh wait, that’s British. Word MD, word. I’m sure his raps are whack!

    3. Joseph Wayne McVey IV (born January 19, 1977), better known by his stage names Z-Ro and The Mo City Don, is an American rapper from Houston, Texas.

      Dre, Malc, Zero a split personality?

      1. Ah, mystery solved. I’m hoping for Chuck D with the next pithy (I may be lisping here) observation .

    4. Dear Dre, Not feeling particularly resilient today. The Multiple Sclerosis MonSter is wearing me out today. I’m not clueless and at first wasn’t going to respond but your Snarky nastiness has me riled. I’m a 61 year old disabled Registered Nurse with 35 years acute and homecare experience. I was a certified case manager in the USA. I was diagnosed with RRMS at age 49 after more than a decade of symptoms. I follow several MS Doctors, Dr. G being a favorite. I feel his honesty and respect for patients is true. I appreciate hearing about Limits on Treatment imposed by organizations like WHO. Clinicians fight every day for us, the community of MS Patients. So Dre, when you refer to readers as fans, you are correct. Clueless?? That’s just plain rude.

  3. Sorry but I am slightly perplexed by your choices for essential medications submitted to WHO.
    Copaxone decreases relapses by 30% above placebo and ZERO benefit above placebo on disease progression, the only marker in MS that matters. Why bother?
    Gilenya is only marginally better with a decrease in relapses by about 50% but only a decrease is disease progression by 30% and its trials in progressive MS withdrawn/failed..
    Why not recommend generic rituximab, generic cladribine or generic DMF for RRMS? Should the one time treatment with HSCT be considered for RRMS as lifelong it would be cheaper lifelong and much more effective?
    What do your group recommend to WHO for “non-active” progressive MS? Like possibly rituximab, biotin, alpha lipoic acid or ibudilast? Or have you just forgotten that progressive MS makes up the majority of worldwide patients?

    1. Rituximab was on the list. The evidence just isn’t there yet for biotin or lipoic acid. More research needed. I’m sure funding isn’t easy to find.

      1. DJ you need to study sociology and the field of adoption, which explains everything. As co-chair, I didn’t vote on the drug selection but respect, and support, the opinions and choices of my colleagues.

      2. ” As co-chair, I didn’t vote on the drug selection but respect, and support, the opinions and choices of my colleagues.”

        And that in a nutshell is why you never get anywhere…

      3. I got to London; I was born in Johannesburg and had to travel to get here 😉

      4. I think, Dr. G., you would never support some of these medication choices based on their respective trials and current efficacy data.

        We really need you on drug selection committees, even with your conflicts, to stop them from choosing low efficacy drugs with no or little effect of progression of MS, which is all that matters in the end.

      5. Hi Mighty Mouse. More non-urgent, insidious research is not a luxury progressive MS patients have. I agree with rituximab, just not fingolimod and never Copaxone’s generic.

        Most progressive MS are already taking, if you are not aware, biotin or ALA based on phase 1 and 2 trials because time is running out with nothing on the horizon to help improve the QOL, not just delay the inevitable (ie. ocrezulimab or siponimod)

        Sitting idle and waiting is definitely not the answer.

      6. Agree. The problem is you need the evidence to convince WHO. It’s Catch 22…

        There you go Dr Dre (Zero). That’s a nice little rap you can have pro-bono.

    1. Dominic

      Dre or Zero (his/her pseudonym) has almost zero engagement and negative energy. We call these people spectators with an opinion, or more appropriately zero opinion 😉

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