Clinic Speak: treat-2-target of NEDA vs. current practice

What you think of a trial to test treat-2-target of NEDA? #MSBlog #MSResearch #ClinicSpea

“As you know neurologists in the UK are handcuffed by NICE and NHS England Guidelines on treating MS. These guidelines are very prescriptive and don’t allow switching or escalation of treatment using MRI. MSers have to fail on their therapy clinically, and sometimes badly, to access more effective therapies. MSers with smouldering MS are left to accrue end-organ damage on lower efficacy medication because we can’t escalate their treatment. To try and address this issue a group of MSologists and I are discussing doing a pragmatic clinical trial to see if MSers manage using a standard care pathway do worse than those managed more actively with a treat-2-target of no evident disease activity (NEDA). The latter algorithm allows one shot at a lower-tier efficacy drug before being escalated to a highly-efficacy therapy and incorporates MRI monitoring. The standard care pathway allows cycling on the lower tier and only escalation based on clinical criteria as per our current guidelines. The picture below sketches out the trial. Please note this has to be a randomised trial so that we can minimise any biases. Do you think this trial is ethical and would you be willing to participate in it? We are suggesting that it last 5 years.”

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 it is what is needed to pressure NICE etc to endorse this approach then, obviously, it is worth doing. My own view is that it is stating the extremely obvious! Adopting approach that seeks to give the patient the oppotunity to prevent brain damage versus allowing brain damage to continue/slightly reduce brain damage seems to be, if you'll excuse the pun, a no-brainer! I was treatment naive before I took Alemtuzumab (privately) and was less than 2 years from my first symptoms/abnormal MRI. I'm also minimally affected by my MS (relatively speaking at least – it doesn't feel minimal to me!). I intend on doing everything in my power to stay that way. I have absolutely no regrets about that approach and still won't even if I am unfortunate enough to succumb to one of the potential side-effects of my treatment.

  • The question of ethics depends on the criteria. As I understand it, the trial would give a portion of patients access care they otherwise would not get. The control group would simply get the same treatments they would normally get without the trial. So from the standpoint of helping the condition of the patients in aggregate, it seems easy to defend. Helping some is better than leaving all alone. This assumes the treatment towards no evident disease activity is the better approach.

    On the flip side, it would seem the research should be possible using meta data from across hundreds of studies like the one you posted last week saying patients on Tysabri took less days off and went to the hospital less. Measuring the control group shouldn't be too hard either with access to medical records from British healthcare. (I am assuming the British single payer insurance system could track this) From this stand point, the cheaper alternative might be to look at existing data saving resources to treat patients.

    This also assumes those making treatment guidelines understand meta analysis and would use it in determining appropriate available treatments. For that matter, would the research as outlined be adequate to influence decision makers who may be looking at things like cost per quality year of patient life to determine which disease will be treated with what resources. I say this knowing I am a lucky patient whose Neurologist escalated me straight to Tysabri after copaxone failed for me.

By Prof G



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