Last month the PCORI group charged with looking into Multiple Sclerosis clinical effectiveness research questions convened a meeting of people who had various interests in the task. I was asked to participate because I am the lead patient representative for iConquerMS™ with the Accelerated Cure Project for the MS PCORnet.
I wrote about this meeting and asked for input from anyone who cared to share their preferences of choices for possible research questions. Thank you if you took the time – I did include your preferences in noting the selections i entered.
If you did not see it, you might want to back up and read my original post before you go any further with reading this one so you understand the context of the rest of what I am going to write.
Now the prioritized questions have been returned to us and I have those in two different forms; the first is the overall ranking of the questions and the second group is a look at how each interest group cast their preferences. Keep in mind there were four people with MS out of 40+ people who sat in on this discussion, and our voice did not carry the weight to match that of the other parties. The number of participants in each group were:
Clinician/Medical Professional (7)
Patient Advocacy Organization (5)
PCORI will further refine these questions and then possible issue a call for proposals from researchers to do one or more of these. The earliest that might happen would probably be August or September.
Here are the results in both forms –
|Question||Score||# who ranked it at all||# who ranked it #1 or #2|
|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?||101||23||14|
|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?||93||29||7|
|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?||89||24||9|
|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?||87||20||11|
|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?||85||22||9|
|C. Is treatment escalation using DMTs as effective as starting treatment with higher efficacy treatments in early active, previously untreated patients?||65||19||5|
|E. What is the comparative effectiveness of stopping versus continuing therapy after a period of prolonged disease stability in patient with MS?||60||16||6|
|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)?||60||21||5|
|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?||59||17||7|
|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?||54||18||2|
|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?||45||16||3|
|K. What are the comparative benefits and harms of specific dietary regimens in people with MS?||37||14||3|
|D. What is the comparative effectiveness of smoking cessation efforts upon disease activity, progression, symptoms, and quality of life in MS?||26||7||1|
This attachment is an Excel spreadsheet with all of the various stakeholder groups itemized. I have to share this as an attachment because the file is so wide and spreads across a large number of columns and rows.
Please be sure and tell me what you think after you have the chance to review these results. And a special thanks to everyone who helped me in the first round to rate them on our behalf.