SC20
Health-Related Quality of Life Instruments Used in Multiple Sclerosis Clinical Trials: A Systematic Review

Thursday, May 29, 2014
Trinity Exhibit Hall
Lixian Zhong, PhD , Global Market Access, Biogen Idec, Cambridge, MA
Xiaoli Niu, BS , Health Economics, University of Southern California, Los Angeles, CA
Sujata P. Sarda, PhD , Global Market Access, Biogen Idec, Cambridge, MA
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Background:

Multiple sclerosis (MS) leads to physical disability associated with fatigue and depression and have a significant impact on patient’s life. Health-related quality of life (HRQoL) measurements provide a comprehensive view of health status of individuals with MS from the patient’s perspective and provide valuable information for clinical trials.

Objectives:

To systematically review and identify HRQoL instruments used in clinical trials for approved and late phase disease modifying therapies (DMTs) for MS.

Methods:

A database search was performed using ClinicalTrial.gov to identify clinical trials on MS. A literature search through MEDLINE and relevant conference abstract databases was also performed to identify published trials with HRQoL measurements in MS patients. The selection criteria included phase II, III, and IV clinical trials on 12 DMTs (interferons, glatiramer acetate, dimethyl fumarate, natalizumab, fingolimod, teriflunomide, alemtuzumab, daclizumab, laquinimod) with HRQoL measurements. All forms of MS were included.  All assessments of relevant studies and data extraction were performed by two reviewers independently.

Results:

A total of 211 MS clinical trials on listed DMTs were identified from clinicaltrial.gov, with 37 included HRQoL measures as the primary or secondary endpoints. Ten additional published clinical trials, which either were not registered on clinicaltrial.gov or only included HRQoL measures as tertiary endpoints, were identified from MEDLINE. Both generic and MS-specific HRQoL instruments were applied in these trials. The most frequently used generic instruments included FIS, SF-36, EQ-5D and SF-12; most frequently used disease-specific instruments included MSIS-29, MSQoL-54 and FAMS.  In the clinical trials with published results in HRQoL measurement, improvement in HRQoL in the active arms was observed in some trials when compared to their specific placebo populations. A detailed analysis of the results will be presented at the conference.   

Conclusions:

HRQoL instruments, both generic and MS disease specific, have been increasingly used in clinical trials. While the sensitivity varies, they provide important insights on overall health status assessment from patient’s perspective alongside physician-assessed clinical outcomes and other patient-reported outcomes (PROs). Maintaining good HRQoL, as measured by HRQoL instruments, should be regarded as an important indicator of therapeutic efficacy.