Tag: James Lind Alliance

What are the most important outstanding research questions for people with MS?

Depending on how long you have been following this blog you may or may not be aware of the COMBAT-MS study. This is a study that is funded by the US government as part of  the Patient-Centered Outcomes Research Institute (PCORI).

COMBAT-MS is a US-Swedish initiative for a hybrid study between a traditional retrospective cohort and a structured prospective cohort study. COMBAT-MS (clinicaltrials.gov NCT03193866) aims to recruit up to 3,700 MS patients at all of Sweden´s seven university clinics (serving about 50% of the total population) who are starting their first MS therapy or switching therapies. By doing the same structured follow-up with annual disability scorings, MRIs and patient-reported QoL outcomes the COMBAT-MS study will generate high quality, real-world long-term efficacy outcomes. This will help the MS community sort out which drugs work or don’t work in a real-world setting. To address safety concerns, COMBAT-MS will obtain data from Swedish national healthcare registries, as well as from Kaiser Permanente Southern California. By combining these resources, COMBAT-MS will be able to address major risks, such as cancer, as well as transient treatable concerns like recurring bladder infections and bothersome skin rashes. So, which of the approved MS drugs will be the winner?

I will be attending the annual COMBAT-MS stakeholder meeting for an update this week and have been asked to give a talk on ‘Identifying and addressing research questions of high importance to patients’. I could simply use the MS Society’s James Lind Alliance Top 10 list (below) or ask you the Barts-MS Blog readers to add to, or critique, this list for my talk this Thursday. Please note that this list below was published in 2013 and what was a priority then may not be a priority today.

1. Which treatments are effective to slow, stop or reverse the accumulation of disability associated with MS?
2. How can MS be prevented?
3. Which treatments are effective for fatigue in people with MS?
4. How can people with MS be best supported to self-manage their condition?
5. Does early treatment with aggressive disease modifying drugs improve the prognosis for people with MS?
6. Is Vitamin D supplementation an effective disease modifying treatment for MS?
7. Which treatments are effective to improve mobility for people with MS?
8. Which treatments are effective to improve cognition in people with MS?
9. Which treatments are effective for pain in people with MS?
10. Is physiotherapy effective in reducing disability in people with MS?

The following is this week’s programme; any comments to help me prepare my talk would be much appreciated. I was invited onto this trial as a stakeholder because I am considered a patient advocate and for my role in formulating and promoting an ‘Essential Off-label DMT‘ list. To fulfil my role as a patient advocate I need your help. Thank you.