TRENDS IN DESIGN, CONDUCT, AND OUTCOMES MEASURES FOR PATIENT REGISTRIES FOR MULTIPLE SCLEROSIS

Author(s)

Aggarwal S1, Kumar S2, Topaloglu H1
1NOVEL Health Strategies, Chevy Chase, MD, USA, 2Institute for Global Policy Research, Washington, DC, USA

OBJECTIVES: Multiple sclerosis (MS) is a chronic progressive neurological disease and the majority of patients will experience some degree of impaired mobility. The objective of this study was to review the trends in design of patient registries for Multiple sclerosis. METHODS: Systematic review was conducted to identify new and on-going pivotal registries for Multiple sclerosis. The inclusion criteria were the indication of Multiple sclerosis, registry status and study completion date of 2010 or after. The data field extracted were study title, intervention, sponsor, age subgroups, planned enrollment, study type, study design, completion date and outcome measures. RESULTS: Overall, 15 clinical studies with total planned enrollment of 113,604 patients were identified. The median enrollment for the studies was 500 patients. For pregnancy related studies, time frame was 1 year or less, while for others it ranged from 5-15 years (longest duration study is an academic registry called Global Demyelinating Disease Registry). Six of the fifteen studies were for pregnancy related outcomes. Primary outcome measures included: Adverse events, Disease progression over time of follow up, Time to resolution of lymphopenia, Negative birth outcomes, including spontaneous abortions and birth defects, Multiple Sclerosis Impact Scale version 1 (MSIS-29v1), Prevalence of PBA (using CNS-LS). Only a few registries provided information regarding secondary outcomes. The outcomes included: Contributing factors to change in MS disease status, Pregnancy outcomes and Neurodegenerative Diseases. Twelve out of 15 registries were sponsored by the industry (5 by Biogen, 2 by EMD Serono), and 3 studies were sponsored by academia. CONCLUSIONS: Current MS registries seems to be focussed largely on safety outcomes, there is a need for measurement of more long term effectiveness and patient reported outcomes evidence.

Conference/Value in Health Info

2016-05, ISPOR 2016, Washington DC, USA

Value in Health, Vol. 19, No. 3 (May 2016)

Code

PND67

Topic

Study Approaches

Topic Subcategory

Registries

Disease

Neurological Disorders

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