What Risks Are MS Patients Willing to Take for Improvement?

Pauline Anderson

June 05, 2013

ORLANDO, Florida — When choosing a therapy, patients with multiple sclerosis (MS) are willing to accept a 1% risk for death or serious adverse effects for the promise of improved symptoms, a new survey suggests. Further, they would trade some risk for death for preventing disease progression and the convenience of an orally administered medication.

Although substantial improvement in symptoms is first on patients' wish list, this is not a proven benefit from available disease-modifying therapies (DMTs), the study shows. Last on their list is relapse prevention, which is the primary outcome of most DMT clinical trials.

"These results are giving us a first view of what patients think about the risks," said Leslie Wilson, PhD, adjunct professor, health policy and economics, School of Pharmacy and Medicine, University of California at San Francisco (UCSF). "They are the ones living with the disease and they say 'Look, for an improvement, I'm willing to take some of this severe side effect risk.'"

The findings were presented here at the 5th Cooperative Meeting of the Consortium of MS Centers (CMSC) and the Americas Committee for Treatment and Research in MS (ACTRIMS). The study was supported by Novartis.

Benefit Levels

Doctors and regulators talk a lot about risk/benefit ratios, but patients are not always a part of the discussion. The researchers decided to look into this and surveyed 300 adult patients with relapsing-remitting MS, mostly (75%) women. About 75% of the sample had mild disease, 18% had moderate disease, and 7% had severe disease. Over a third (38%) were taking an interferon, 24% were receiving natalizumab, and 24% were receiving glatiramer acetate.

Patients were given 16 cards, each containing 6 attributes of a hypothetical disease-modifying drug: delay progression, prevent relapse, improve symptoms, common side effects, severe side effects, and administration. Along with each attribute were various levels of benefit; for example, for "delay progression," the medication could prevent symptoms from getting worse for 2 years, 4 years, or 10 years.

Dr. Leslie Wilson

In the "improve symptoms" category, patients would feel no improvement, mild improvement, or substantial but rare improvement. And for "administration" mode, the levels included an oral pill taken once daily, an intramuscular shot weekly, a subcutaneous injection daily, or an intravenous infusion monthly.

"We took what attributes were important to these patients, the positive things, and asked them how much of this they would give up and for what gain," explained Dr. Wilson.

The patients were also asked whether they would take 2 separate drugs: One, an intravenous infusion given once every 4 weeks, has no risk for death or severe disability; causes 1 relapse every 5 years; results in no improvement; and causes headache, muscle/joint aches, and flu-like symptoms. This agent prevents disease progression for 10 years.

The second agent, an oral pill taken once a day, carries a risk for death or severe disability of 1 in 1000, and patients taking it will have 1 relapse a year, feel substantial improvement, and experience changes in mood. However, this agent prevents progression for only 4 years.

The analysis showed that patients are willing to accept a 1% risk for death or severe adverse effect for a substantial, but rare, improvement in their symptoms. To gain 10 years of disease progression prevention, patients are willing to risk a 0.7% chance of death or severe adverse effect.

Patient Preferences

Perhaps not surprisingly, patients prefer oral medications and ones given as infrequently as possible. Compared with daily subcutaneous administration, patients preferred daily oral administration (odds ratio, 2.15; P < .001), then monthly intravenous (odds ratio, 1.54; P < .001), and then intramuscular weekly (odds ratio, 1.19; P < .01).

The next step is to turn this into a tool that patients could use in deciding on therapies along with their physician, said Dr. Wilson.

Christine Bui

The group also plans to do subanalyses to determine, for example, whether men are greater risk-takers than women or whether there are any differences according to age, disease severity, length of time a patient has had MS, the number of treatment failures, and whether a women intends to get pregnant, said Christine Bui, a PharmD candidate at UCSF who presented the data.

Commenting on the study, Stephen Krieger, MD, Corinne Goldsmith Dickinson Center for MD, Mount Sinai, New York, New York, noted that in his practice he sees vast differences among patients in the risks of medications they're willing to take.

For example, he said, when a new drug came out recently that carried a 1 in 1000 chance of death, some patients told him they wouldn't think twice about taking that kind of risk if it meant they would gain symptom relief, while others felt it was way too risky.

The study was supported by Novartis.

5th Cooperative Meeting of the Consortium of MS Centers (CMSC) and the Americas Committee for Treatment and Research in MS (ACTRIMS). Abstract DX-03. Presented May 31, 2013.

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