Contributions
Abstract: P1304
Type: Poster
Abstract Category: Therapy - symptomatic - Quality of life
Background: Clinical trials characterize efficacy and safety of MS therapies, but patient acceptance of the risk/benefit balance is less well understood. This deficit presents challenges to regulators, who must decide if the known risks are appropriately balanced by the known benefits. We conducted a survey of people with MS to better understand risk tolerance to MS therapies among people with MS.
Methods: A series of focus groups across the United States and formal cognitive testing of the derived questions were used to design a survey for people with MS. While North American Research Committee on MS (NARCOMS) registry participants were recruited directly, the survey was also made available through an open link on the National MS Society (NMSS) website. Using a standard gamble paradigm, we identified the highest tolerated risk level for a hypothetical therapy that would reduce rate of clinical relapses by 50% and disability by 30%. Starting from a risk of 1:1000, the risk was iteratively changed to identify the highest risk tolerated, ranging from “would take regardless of the risk of death” to “no acceptable risk.” Risks included skin rash or infection (both of which could require hospitalization), kidney injury (which could require life-long dialysis), thyroid injury (which could require life-long thyroid medication), liver injury (which requires blood test monitoring), and progressive multifocal leukoencephalopathy (PML, which is often fatal).
Results: 3719 people with MS completed the survey (2446 NARCOMS, 1273 NMSS): mean(SD) age 55.4(11.1) yrs, 78% female, disease duration 16.8(9.8) yrs, median(IQR) Patient Determined Disease Steps 3-Early Gait Disability (1-Mild Disability, 5-Late Cane Use), 89% had been on a DMT with 51% reporting current use. The median (IQR) risk tolerance for each complication was (in descending order): infection 1:1000 (1:100, 1:500 000); thyroid injury 1:1000 (1:500, 1:1 000 000), skin rash 1:2000 (1:500, 1:1 000 000), liver injury 1:10 000 (1:1000, 1:1 000 000), kidney injury 1:1 000 000 (1:5000, no acceptable risk), and PML 1:1 000 000 (1:5000, no acceptable risk).
Conclusions: People with MS report different levels of tolerance to various risks. Respondents were most willing to accept infection or thyroid injury and least willing to accept kidney injury or PML. Risk tolerance data can help inform clinicians and regulatory agencies about patient preferences regarding the balance of risks and benefits.
Disclosure: This study was funded by the National MS Society Grant HC1411-02010 awarded to Robert Fox.
Disclosures:Robert Fox: Dr. Fox has received consultant fees from Biogen Idec, GlaxoSmithKline, Novartis, Questcor, Teva, and Xenoport; grant and research support from Novartis.
Carol Cosenza: nothing to disclose
Lauren Cripps: nothing to disclose
Paul Ford: nothing to disclose
Marybeth Mercer: nothing to disclose
Sneha Natarajan: nothing to disclose
Amber Salter: nothing to disclose
Tuula Tyry: nothing to disclose
Stacey Cofield: Dr. Cofield has received consulting fees from The American Shoulder and Elbow Society and Oxford University Press; grant support from Pfizer and the American College of Rheumatology; DMSB service fees from MedImmune, Inc.
Abstract: P1304
Type: Poster
Abstract Category: Therapy - symptomatic - Quality of life
Background: Clinical trials characterize efficacy and safety of MS therapies, but patient acceptance of the risk/benefit balance is less well understood. This deficit presents challenges to regulators, who must decide if the known risks are appropriately balanced by the known benefits. We conducted a survey of people with MS to better understand risk tolerance to MS therapies among people with MS.
Methods: A series of focus groups across the United States and formal cognitive testing of the derived questions were used to design a survey for people with MS. While North American Research Committee on MS (NARCOMS) registry participants were recruited directly, the survey was also made available through an open link on the National MS Society (NMSS) website. Using a standard gamble paradigm, we identified the highest tolerated risk level for a hypothetical therapy that would reduce rate of clinical relapses by 50% and disability by 30%. Starting from a risk of 1:1000, the risk was iteratively changed to identify the highest risk tolerated, ranging from “would take regardless of the risk of death” to “no acceptable risk.” Risks included skin rash or infection (both of which could require hospitalization), kidney injury (which could require life-long dialysis), thyroid injury (which could require life-long thyroid medication), liver injury (which requires blood test monitoring), and progressive multifocal leukoencephalopathy (PML, which is often fatal).
Results: 3719 people with MS completed the survey (2446 NARCOMS, 1273 NMSS): mean(SD) age 55.4(11.1) yrs, 78% female, disease duration 16.8(9.8) yrs, median(IQR) Patient Determined Disease Steps 3-Early Gait Disability (1-Mild Disability, 5-Late Cane Use), 89% had been on a DMT with 51% reporting current use. The median (IQR) risk tolerance for each complication was (in descending order): infection 1:1000 (1:100, 1:500 000); thyroid injury 1:1000 (1:500, 1:1 000 000), skin rash 1:2000 (1:500, 1:1 000 000), liver injury 1:10 000 (1:1000, 1:1 000 000), kidney injury 1:1 000 000 (1:5000, no acceptable risk), and PML 1:1 000 000 (1:5000, no acceptable risk).
Conclusions: People with MS report different levels of tolerance to various risks. Respondents were most willing to accept infection or thyroid injury and least willing to accept kidney injury or PML. Risk tolerance data can help inform clinicians and regulatory agencies about patient preferences regarding the balance of risks and benefits.
Disclosure: This study was funded by the National MS Society Grant HC1411-02010 awarded to Robert Fox.
Disclosures:Robert Fox: Dr. Fox has received consultant fees from Biogen Idec, GlaxoSmithKline, Novartis, Questcor, Teva, and Xenoport; grant and research support from Novartis.
Carol Cosenza: nothing to disclose
Lauren Cripps: nothing to disclose
Paul Ford: nothing to disclose
Marybeth Mercer: nothing to disclose
Sneha Natarajan: nothing to disclose
Amber Salter: nothing to disclose
Tuula Tyry: nothing to disclose
Stacey Cofield: Dr. Cofield has received consulting fees from The American Shoulder and Elbow Society and Oxford University Press; grant support from Pfizer and the American College of Rheumatology; DMSB service fees from MedImmune, Inc.