The majority of people newly diagnosed with MS go through periods of relative health followed by episodes of new or worsening symptoms, referred to as relapses. Since 1993, disease-modifying therapies have been available that can lessen the frequency and severity of relapses and prevent new MRI lesions. The Food and Drug Administration has approved 14 drugs to treat the symptoms and slow the course of this relapsing-remitting form of MS.

A PCORI-funded study is now testing whether some patients, after years of successful treatment, could safely stop taking those drugs.

Why is this an important issue? The average annual wholesale cost of the drugs is $90,000. Some of the treatments are taken orally, but others require injections. Most have side effects, some of which can be serious. “I give myself a shot three times a week. I don’t want to do that for the rest of my life,” says Robin Beland, MBA, a vice president of sales at a technology company, who was diagnosed with MS around 20 years ago.

Beland plans to sign up for the study. “I do MRIs yearly, and I don’t have any new lesions. I don’t know if that’s because of the drug or not,” she says. She’s willing to risk having a relapse if the study randomly assigns her to the group of the patients who stop taking medicines. “If I go off the medicine and something happens, then I’ll go back on,” she says.

Corboy’s team is getting ready to enroll patients at 15 study sites, three of which are supported by funding from the National Multiple Sclerosis Society. To gauge the likelihood of success in recruiting participants, the team did a preliminary survey asking patients whether they’d consider joining such a study.

“Some people are scared about going off medication,” Corboy says. “They say, ‘If it ain’t broke, don’t fix it.’ They won’t want to join our study, and we understand their concerns. But everybody is interested in the study’s results.”

If the research finds no downside to stopping medication, Corboy thinks a larger study should take place, perhaps homing in on the best age to stop taking the medications. If, however, going off disease-modifying drugs carries significant risks, Corboy says, “that would reassure physicians and patients that it’s worthwhile to continue to use these medications in spite of the costs and hassle and side effects.”

There could, of course, be a gray area in the results, where patients face a slight risk of relapse or lesion if they stop treatments. Each patient and doctor would then need to weigh the options together.

Patients and other stakeholders have been involved from the beginning of the study through two stakeholder advisory committees based at the University of Colorado and the Cleveland Clinic, one of the study sites. The advisory committees worked intensively on the language in questionnaires to ask patients about how satisfied they are with their treatment or lack thereof.

“Ultimately, patient satisfaction is the bottom line,” Corboy says.