Patient Real-world Clinical, Neurological, Tolerability, and Safety Outcomes for Tecfidera® and Rebif®

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the proportion of patients who demonstrate no medical need to discontinue therapy among DMT-naïve patients with relapsing forms of multiple sclerosis after 1 year of treatment with Rebif 44 mcg tiw or with Tecfidera 240 mg bid based on real-world data.

Official Title
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Patient Real-world Clinical, Neurological, Tolerability, and Safety Outcomes for Tecfidera® and Rebif®: A Retrospective Study (PROTRACT)

Conditions
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Relapsing-remitting Multiple Sclerosis

Intervention / Treatment
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* Drug: Rebif
* Drug: Tecfidera


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of Clinically Isolated Syndrome (CIS) or a relapse remitting multiple sclerosis (RRMS). Age between 18 - 55 years at the time of index. No evidence of prior disease modifying therapy for MS. Initiated treatment with either Rebif or Tecfidera at the time of index. Patient is considered to have initiated treatment if they took at least one dose. Treatment must have been initiated after the product was approved by the FDA. Availability of a high quality baseline MRI brain scan, which must have occurred between 6 months prior to the index date to 2 weeks after the index date. Availability of clinical data in the patient's record for the full study observation period, as defined in the primary objective. Exclusion Criteria: Pregnant at any time during the study observation period. Presence of pre-existing medical conditions known to be associated with brain pathology (cerebrovascular and neurodegenerative diseases, presence of active alcohol or substance abuse). Patient discontinued initial therapy prior to completing 1 year of treatment due to a reason other than disease activity, tolerability, or safety (e.g. financial, convenience, preference, etc.). Phase III registrational trial patients

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Rebif - 1 year MRI cohort<br>Treatment naive patients starting with Rebif, 1 year follow-up available, MRI scan available at baseline and at 12 months | Drug: Rebif<br> <br> |
| Rebif - 1 year clinical cohort<br>Treatment naive patients starting with Rebif, 1 year follow-up available, MRI scan available at baseline | Drug: Rebif<br> <br> |
| Rebif - early discontinuation cohort - tolerability<br>Treatment naive patients starting with Rebif, MRI scan available at baseline, discontinuation within the first treatment year due to tolerability | Drug: Rebif<br> <br> |
| Rebif - early discontinuation cohort - adverse events<br>Treatment naive patients starting with Rebif, MRI scan available at baseline, discontinuation within the first treatment year due to adverse events | Drug: Rebif<br> <br> |
| Rebif - early discontinuation cohort - disease activity<br>Treatment naive patients starting with Rebif, MRI scan available at baseline, discontinuation within the first treatment year due to disease activity | Drug: Rebif<br> <br> |
| Tecfidera - 1 year MRI cohort<br>Treatment naive patients starting with Tecfidera, 1 year follow-up available, MRI scan available at baseline and at 12 months | Drug: Tecfidera<br> <br> |
| Tecfidera - 1 year clinical cohort<br>Treatment naive patients starting with Tecfidera, 1 year follow-up available, MRI scan available at baseline | Drug: Tecfidera<br> <br> |
| Tecfidera - early discontinuation cohort - tolerability<br>Treatment naive patients starting with Tecfidera, MRI scan available at baseline, discontinuation within the first treatment year due to tolerability | Drug: Tecfidera<br> <br> |
| Tecfidera - early discontinuation cohort - adverse events<br>Treatment naive patients starting with Tecfidera, MRI scan available at baseline, discontinuation within the first treatment year due to adverse events | Drug: Tecfidera<br> <br> |
| Tecfidera - early discontinuation cohort - disease activity<br>Treatment naive patients starting with Tecfidera, MRI scan available at baseline, discontinuation within the first treatment year due to disease activity | Drug: Tecfidera<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| NEDA-2 | Number of patients who had no need to discontinue therapy within the first 12months after index date. Unit of measure is number of patients. No medical need to discontinue therapy is reached if there are no relapses and no new lesions, and no enlarging lesions and the patient did not stop the index medication due to tolerability, adverse events or disease activity. If any of the above occurs, NEDA-2 is not reached. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical differences between the two treatment groups | Comparison of number of relapses between the two treatment groups. | 12 months | 
| Neurological differences between two treatment groups | Comparison of number of new lesions and number of enlarging lesions between the two treatment groups. Unit of measurement is number of each lesion type. | 12 months | 
| Proportion of individuals within each treatment group who discontinued, stratified by reason | Proportion of individuals within each treatment group who discontinued treatment due to tolerability to adverse events to disease activity Unit of measurement is the share of patients who discontinued compared to total patients stratified by reason of discontinuation. | 12 months |