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. 2016 Feb 4;5(1):e23.
doi: 10.2196/resprot.4473.

Multiple Sclerosis Therapy With Disease-Modifying Treatments in Germany: The PEARL (ProspEctive phArmacoeconomic cohoRt evaluation) Noninterventional Study Protocol

Stefan Viktor Vormfelde  1 , Sonja Ortler, Tjalf Ziemssen
Affiliations

Multiple Sclerosis Therapy With Disease-Modifying Treatments in Germany: The PEARL (ProspEctive phArmacoeconomic cohoRt evaluation) Noninterventional Study Protocol

Stefan Viktor Vormfelde et al. JMIR Res Protoc. .

Abstract

Background: Patients with multiple sclerosis (MS) require long-term therapy and have a wide variety of needs for health-related support. The efficacy and safety of MS therapy, as assessed by both clinicians and patients, are important parameters that need to be considered. However, few studies combine data on efficacy and safety outcomes with pharmacoeconomic data.

Objective: Here, we present the study design of the ProspEctive phArmacoeconomic cohoRt evaluation (PEARL), a prospective, multicenter, noninterventional cohort study on patients with relapsing-remitting MS (RRMS) treated with disease-modifying treatments (DMTs).

Methods: During a prospective observational phase of 24 months per patient, PEARL evaluated clinical and patient-perceived efficacy and safety measures, as well as pharmacoeconomic data on RRMS patients treated with DMTs-interferon beta and glatiramer acetate. Measurements of the patients' perceptions included the assessment of patient-reported quality of life, treatment satisfaction, and compliance. The study was planned to include 1800 outpatients from 180 German neurological practices who had continuously been treated with an approved DMT for at least 30 days. The primary statistical analyses of the PEARL study will be descriptive. Particular focus will be on specific subgroups, such as patients who switched DMTs during therapy and patients with disease worsening or disease activity. Subgroups will be compared using stratified analyses.

Results: Data collection for PEARL started in September 2010 and ended in July 2013. As of July 2015, the study is completed and is currently being analyzed and written up.

Conclusions: PEARL is evaluating both the health status and resource utilization of RRMS patients treated with DMTs in Germany. The combination of pharmacoeconomic data with clinical and patients' self-perceived efficacy and safety outcomes will add useful information to the currently incomplete picture of the overall RRMS burden in Germany.

Keywords: disease-modifying therapies; efficacy; multiple sclerosis; outpatient care; pharmacoeconomics; safety.

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Conflict of interest statement

Conflicts of Interest: Stefan Viktor Vormfelde and Sonja Ortler are employees of Novartis Pharma GmbH, Nuremberg, Germany. Tjalf Ziemssen has served on scientific advisory boards, and has received scientific grants and speaker honoraria from Bayer, Biogen Idec, Genzyme, TEVA, Merck Serono, and Novartis.

Figures

Figure 1
Figure 1
Timeline of PEARL, a noninterventional cohort study on relapsing-remitting multiple sclerosis (RRMS) patients treated with disease-modifying treatments (DMTs) in outpatient care. The study includes approximately 1800 RRMS patients from 180 neurological practices. Four major categories of data are evaluated during the observational phase of 24 months per patient (study visits 1–9). PEARL: ProspEctive phArmacoeconomic cohoRt evaluation.
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