PCORI and Me and You!! – Help Prioritize Research Topics


Forty-one stakeholders with a keen interest in Multiple Sclerosis, gathered in Washington DC on April 2, to develop, discuss and submit topics to be considered by PCORI for Comparative Effectiveness Research (CER) in MS.  In preparation for the meeting, the stakeholders were invited to submit topics for consideration and the lead science writers and researchers for PCORI consolidated the ideas into central themes; you can read those ideas in the summaries available at http://www.pcori.org/events/2015/prioritizing-comparative-effectiveness-research-questions-treatment-multiple-sclerosis. Also available at this link are the other meeting materials, a list of attendees and their affiliations, and recordings of each session.

I was invited to participate because of my connection with the PCORI funded PCORnet MS-PPRN, and my role as the lead patient representative for Accelerated Cure Project’s  iConquerMS™  (please enroll today, if you haven’t already done so!) patient powered research network.


There were four central themes identified for the day’s discussions, and we worked within an assigned group to come to a consensus of how a CER question might reflect our individual submissions.

The four groups were:

Group 1 – Comparison across DMTs, including differential effects in subgroup

Group 2 – Care Strategies

Group 3 – Non-pharmacologic and non-DMT therapy for specific symptoms and overall health


Group 4 – Timing of therapy and study design



As you might already know,  PCORI begins with the words Patient Centered, and I raised concerns that of the 41 participants, only four of us identified ourselves as people living with multiple sclerosis.  There were a few more representatives from the Accelerated Cure Project, National MS Society and the Multiple Sclerosis Foundation.  The remaining participants were connected with MS pharmaceutical companies, government agencies, managed care (both medical and pharmacy benefits) organizations and physician groups.  The patient voice was definitely in the minority in these sessions.  That said, I was a participant in group #2 and felt my ideas were well received and the chair of the discussion, Dr. Alex Rae Grant, actively sought my input at several points.


Toward the end of the day, we reconvened and each group presented their revised questions, and it was then opened for discussion. There was little discussion or debate about the proposed questions and the chair of the talks turned the questions toward the people with MS who were in the room, asking for our thoughts about the day, the process and the proposed topics.  Although it felt as if there was empty time on the schedule that needed to be filled with the impromptu shout-out for our reactions, I believe we all welcomed this additional opportunity to speak to the larger issues.


The proposed CER topics for consideration for funding are now being prioritized by the 41 participants, myself included.  We have been polled electronically and asked to rank the importance of these topics, and from those rankings the PCORI decision makers will choose what might be put forth in a request for proposal (RFP) to fund Multiple Sclerosis research.  If you are familiar with PCORI, there are some restrictions on what they will fund – they must always look at CER, and the cost of treatment can never be considered.


An email was sent to all participants, and by Wednesday, April 8, I am asked to prioritize the order of these suggested topics and indicate which SIX (6) I would rank with the highest priority and add any additional comments I might wish to include to support my choices. Following are the proposed CER questions:


A: What are the comparative benefits and harms of different disease-modifying therapies in newly diagnosed relapsing, remitting multiple sclerosis on disease activity, disease progression, symptoms, and quality of life?

B: Among MS patients receiving a DMT who experience disease activity, what are the benefits and harms of continuing the same therapy versus changing to a new medication?

C: Is treatment escalation using DMTs as effective as starting treatment with higher efficacy treatments in early active, previously untreated patients?

D: What is the comparative effectiveness of smoking cessation efforts upon disease activity, progression, symptoms, and quality of life in MS?

E: What is the comparative effectiveness of stopping versus continuing therapy after a period of prolonged disease stability in patient with MS?

F: In people with progressive MS, what is the comparative effectiveness of different care delivery approaches (i.e., MS specialty center vs. community neurology; direct care vs. telemedicine; “specialized medical home” vs. community neurology delivery of care) in improving outcomes such as functional status, quality of life, symptoms, ER use, and hospitalization?

G: In people with relapsing MS within 2 years of diagnosis, what is the comparative effectiveness of changing DMT using a NEDA strategy (no relapse, no new MRI or enhancing lesion, no change in disability) vs. not changing DMT in terms of functional status, quality of life, symptoms, ER use, and hospitalization?

H: In people with relapsing MS, what is the comparative effectiveness of physician-directed vs. allied health-directed vs. navigator-directed, vs. technological-enabled self-management tools for improving initial decision making, patient care experiences, decision regret, quality of life and adherence to therapy?

I: Does an integrative model of care along with DMT in a newly diagnosed individuals affect disability progression and symptoms (physical, emotional and cognitive) compared to treatment with DMT alone?

J: What are the comparative benefits and harms of non-pharmacological and pharmacological approaches in relation to key symptoms (e.g., emotional health, fatigue, cognition, pain) in people with MS?

K: What are the comparative benefits and harms of specific dietary regimens in people with MS?

L: What are the benefits and harms of early vs. delayed treatment with DMTs, in terms of symptoms, function, QOL, and disease activity in treatment-naive patients recently-diagnosed patients (meeting McDonald criteria within 12 months)?

M: In patients who recently transitioned from relapsing to progressive MS or were recently diagnosed with SPMS, what are the benefits and harms of continuing compared to discontinuing DMTs on outcomes including but not limited to symptoms, QOL, function, disease activity, disability, and/or mortality?


Which brings me to the point I made in the meeting – I am just one person with MS and there are at least 400,000 of us in the United States alone, trying to make sense of living with multiple sclerosis.  Our perspective on what is important to study may be totally different than the perspective of these other stakeholder groups and I would like your input on this list of topics and what you believe should be prioritized.


Would you look at these topics, consider their significance to you, and tell me what you would rank toward the top of your list?   If you have reasons why they are important, I would love to hear that too.  I will consider all the comments and put those together with my own and submit ‘our’ rankings to PCORI. You don’t need to give me six but if you would at least give me a top three, that would be helpful to consult as I propose our slate.


This is due to PCORI  on Wednesday, so please help me by commenting no later than Tuesday afternoon.  Your help with this important assignment is greatly appreciated and I thank you in advance.


Be well,



10 thoughts on “PCORI and Me and You!! – Help Prioritize Research Topics

  1. #1 for me is – K – I have been following the Wahls Protocol (most of the time) the past 3 months while I know this is not a cure – it has helped me to have more energy to actually cook meals for my family and volunteer more hours at the humane society and the NMSS Miami County Walk. When I was growing up their wasn’t growth modifying hormones (GMO) or as many pesticides used on our food. I would love to see research on eating the way Dr Wahls talks about and the way people are eating with all the processed food/GMO.

    #2 – H – seems like an interesting and useful comparison

    #3 – B – this would be interesting to c what they come up with and how long you should wait to try a new medicine (even if your MRI are showing no changes but your slowing declining physically).

  2. Laura

    PCORI Rank by Laura R.:

    1. K…important for the multitudes of those diagnosed w/MS who have significant allergic reactions to DMT’s or other diseases which prevent the use of DMT’s
    2. E
    3. L
    4. M

  3. Nancy

    #1 K! More info is needed on food/nutrition and MS! It is empowering to me to make daily choices that will enhance and perhaps improve my overall wellness.
    #2 B and E appear to be two sides to the same coin – making informed choices about DMT.
    #3 J. What else can we do to maintain or improve our health beyond pharmaceuticals.

    As a person living with MS, I am most interested in finding answers for MSers rather than drug companies. I realize the drug companies provide the DMT options and I am thankful! But as an MSer trying to maintain control of life with MS, I want to have “answers” that directly impact my daily living.

    Thanks for being our voice!

  4. From Elin on Twitter – her assorted comments

    K: Which “diets”, specifically? Vegetarian, Lacto-V, Atkins, Mediterranean, Swank, Wahls, etc., etc.

    J. All symptoms, or just those mentioned? Does it include environment/heat, ON, ice-pick headaches, Lhermitt’s, etc.?

    It seems many of the Q’s need refined & clarified. Example: I (is there a standard “integrative care model”?) D-F: Poorly worded

    H is a total mess.

    I would choose the following for prioritization: A, B, E, J, K, L, M

  5. From Kim on FB –
    HI Laura, I most highly rated the integrative therapy approach early on vs DMT only study, followed by (and not necessarily in the order they appear): Drug vs non-drug approaches, early intervention vs delayed treatment, starting with most effective DMTs vs waiting, and progressive MS delivery of care types. Most of the options including those I listed left me wondering how valuable a study would be considering how we all respond to different DMTs and different other approaches in endlessly varied ways. I’d probably totally change my mind if I gave this list more thought, but the one that really jumped out as urgent was the integrative therapy vs DMT only. My reason being that if studies showed an integrative approach was most beneficial like I think it would, this might precipitate a revolution of changes in our care approaches. –Kim

  6. From Goldie Erowele on FB
    thank you for sharing. I will share on Carenovate Magazine pages. I definitely think my top 6 – B, C, F, H, J and K. Will love to hear other thoughts

I love to hear your thoughts - feel free to leave a comment.