Convening Stakeholders to Improve Treatment of Multiple Sclerosis

Multiple sclerosis (MS) is a progressive neurological disorder with an unpredictable course that typically unfolds over many years. Symptoms can range from mild to severely disabling. There are now a dozen disease-modifying therapies (DMTs) that may slow MS’ progression and other treatments that target specific symptoms, but no cure. To choose among treatment options, clinicians and patients need information comparing benefits and harms of available therapies, but such data are in short supply.

The kind of comparative clinical effectiveness research (CER) that we fund at PCORI—CER that takes into account patients' concerns, preferences, and particular circumstances—could help to address this issue. In fact, more than a year ago, our multi-stakeholder Advisory Panel on the Assessment of Prevention, Diagnosis, and Treatment Options prioritized MS as a topic for research funding, which resulted in its inclusion as a topic of particular interest in our Pragmatic Clinical Studies initiative. We have since received many Letters of Intent proposing pragmatic studies focused on MS treatments, although to date, none of the investigators suggesting such studies has been invited to apply.

The nature of MS introduces substantial challenges into the process of designing CER studies that will give patients and clinicians useful information. The disease can present with a variety of symptoms and its course is variable, with two major types—relapsing and progressive. The many available treatments, both drug and non-drug, may affect groups of patients differently.

We Ask the Experts

To help us determine whether there might be CER questions about MS that would be appropriate for PCORI to address, we turned for guidance to those who have the biggest stake in MS research: patients, clinicians and healthcare systems that treat MS patients, and companies that develop drugs to treat this condition and its symptoms. On April 2, we convened a large stakeholder workshop in Washington, DC, to consider promising CER questions that, if answered through research we’d support, could help patients and clinicians choose the best treatments for patients with MS.

The attendees didn’t start from scratch. We provided them with a topic brief summarizing what is known about MS and options for its treatment, including the potential for CER to improve patient-centered care, and the obstacles that must be overcome. Before the meeting, we compiled a list of potential questions submitted by the participants.

The Workshop at Work

To refine and clarify the potential CER questions and assign priorities, the workshop participants split up into four groups to consider different topics:

Comparisons of DMTs, including differential effects in patient subgroups
Care strategies
Non-DMT and non-drug treatments for specific symptoms and general health
Timing of therapy and study design

After three hours of discussion, all the participants came together again and the leaders of the four groups presented the CER questions they had identified as highest priority. Among them:

For newly diagnosed patients with relapsing MS, what are the comparative benefits and harms of different DMTs?
If symptoms continue, what are the benefits and harms of staying on the same DMT, compared to changing to a different drug?
For people with progressive MS, what effect do different care settings—an MS specialty center compared to a community neurology clinic, for example—have on their functional status and quality of life, symptoms, and rates of hospitalization?
Does adding integrative care (e.g. exercise, physical therapy, and diet management) to a DMT at the time of diagnosis affect the progression of disability and the physical, emotional, and cognitive symptoms of MS?
What are the benefits and harms of starting a DMT soon after diagnosis, while symptoms are still mild—compared with delaying treatment—in regard to symptoms, function, quality of life, and disease activity?
In relapsing MS, does counseling by a physician, an allied healthcare professional, or a patient navigator and/or the use of technology-enabled self-management tools influence patients’ involvement in initial decision making, decisional regret, quality of life, and adherence to treatment?

In the discussion that concluded the workshop, several patient-participants emphasized their feelings of powerlessness in the face of their disease, their hunger for information that will help them make informed decisions about treatment, and their hope that data from CER studies will be translated into patient-friendly training and communication formats.

The workshop’s task isn't yet completed. We are asking each participant to reflect on the day's deliberations and prioritize the full set of 13 CER questions suggested by all the groups.

Our staff will take into account the workshop's conclusions in subsequent deliberations as we further refine questions before presenting our recommendations to our Science Oversight Committee. Depending on those discussions, our Board of Governors may consider approving development of a PCORI Funding Announcement inviting researchers to submit proposals to address one or more high-priority MS questions.

We are eager to share what we are learning about developing CER relevant to the treatment of MS. The workshop was open to the public as a webinar and audio recordings are available. We hope that our in-depth consideration of potential CER questions about treatments for MS will lead to new data that support patient-centered care for this disease.