Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)

Study Overview
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Brief Summary
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This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL. A survey will be used to measure the knowledge and comprehension of the REBLOZYL aRMMs among European Economic Area (EEA) based healthcare professionals (HCPs). The PASS will be conducted among HCPs in a representative sample of EEA countries where REBLOZYL is commercially available, potentially including Austria, Germany, Italy, Norway, Sweden, the Netherlands, Poland, and Spain. Additional EEA countries may be included, as needed, based on commercial availability and reimbursement status.

Official Title
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A Study to Evaluate the Effectiveness of the Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs) in the European Economic Area (EEA)

Conditions
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Myelodysplastic Syndromes, Beta-thalassemia

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Healthcare professionals (HCPs) experienced in treatment of haematological diseases who may intend to prescribe REBLOZYL in the participating European countries and were on the target group for dissemination of the additional risk minimization measures (aRMMs). The HCP provides permission to share their responses in aggregate with European Medicines Agency (EMA) or National Competent Authorities (NCAs), if requested. Exclusion Criteria: HCPs who previously participated in the cognitive pre-testing of the survey questionnaires to be used for the study. HCPs who have been direct employees of the marketing authorization holder (MAH), the EMA, or the study vendor within the past 5 years.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Healthcare Professionals (HCPs)<br>The PASS will be conducted among HCPs in a representative sample of EEA countries where REBLOZYL is commercially available. A sample of HCPs from EEA countries who manage care for patients with certain haematologic conditions and who may/do prescribe REBLOZYL will be recruited from the target population of HCPs who were sent the REBLOZYL aRMMs in these countries. The final list of countries to be included may include 1) only countries where reimbursement has been sought and gained, 2) a geographically representative sample (e.g., northern, southern, eastern, and western EU Members States to the degree possible based on the first criteria), 3) a mixture of countries with higher and lower REBLOZYL usage, and 4) other feasibility considerations such as the ability to conduct direct-to-HCP non-market research studies. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Knowledge that although there are no data from the use of REBLOZYL in pregnant women, studies in animals exposed to REBLOZYL have shown reproductive toxicity and embryo-fetal toxicity | Calculated as the percentage of healthcare professionals (HCPs) who answer True to question 1A | Up to 3 months | 
| Knowledge that women of childbearing potential (WCBP) who become pregnant during treatment with REBLOZYL should not continue REBLOZYL treatment even if they receive appropriate monitoring | Calculated as the percentage of HCPs who answer False to question 1B | Up to 3 months | 
| Knowledge that HCPs must counsel WCBP of the potential teratogenic risk of REBLOZYL | Calculated as the percentage of HCPs who answer True to question 1C | Up to 3 months | 
| Knowledge that for WCBP who become pregnant during treatment with REBLOZYL, the pregnancy outcome should be evaluated | Calculated as the percentage of HCPs who answer False to question 1D | Up to 3 months | 
| Knowledge that the use of REBLOZYL is contraindicated during pregnancy and in WCBP who are not using at least 1 highly effective method of contraception | Calculated as the percentage of HCPs who answer True to question 1E | Up to 3 months | 
| Knowledge of what information about contraception methods should be provided when counselling WCBP | Calculated as the percentage of HCPs who answer WCBP should use at least 1 highly effective method of contraception during treatment with REBLOZYL and for at least 3 months after stopping treatment to question 2 | Up to 3 months | 
| Knowledge of how many pregnancy tests should be performed for WCBP before they start treatment with REBLOZYL | Calculated as the percentage of HCPs who answer A single pregnancy test must be performed and medically verified before starting treatment with REBLOZYL to question 3 | Up to 3 months | 
| Knowledge of the frequency pregnancy tests should be repeated for WCBP who are receiving treatment with REBLOZYL | Calculated as the percentage of HCPs who answer At suitable intervals and medically verified as negative to question 4 | Up to 3 months | 
| Knowledge that WCBP should be informed to report a pregnancy that occurs during treatment with REBLOZYL and should receive counselling if they become pregnant | Calculated as the percentage of HCPs who answer True to question 5 | Up to 3 months | 
| Knowledge of how long after stopping treatment with REBLOZYL should WCBP be informed to report a pregnancy that occurs and should they receive counselling if they become pregnant | Calculated as the percentage of HCPs who answer Pregnancies that occur within 3 months after stopping REBLOZYL should be reported and participants should receive counselling to question 6 | Up to 3 months | 
| Always providing WCBP with the REBLOZYL Patient Card | Calculated as the percentage of HCPs who answer Yes, always to providing the REBLOZYL Patient Card to WCBP (question 10-A). The denominator for this variable is the n who answer question 10 (not the n who answer 10-A) | Up to 3 months | 
| Composite knowledge for all 5 items in the core question set | A composite endpoint calculated as the percentage of HCPs who answer: None of questions 1E, 2, 3, 4, and 10-A correctly; 1 of questions 1E, 2, 3, 4, and 10-A correctly; 2 of questions 1E, 2, 3, 4, and 10-A correctly; 3 of questions 1E, 2, 3, 4, and 10-A correctly; 4 of questions 1E, 2, 3, 4, and 10-A correctly; All 5 of questions 1E, 2, 3, 4, and 10-A correctly | Up to 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Awareness of the HCP Checklist | Calculated as the percentage of HCPs who answer Yes to question 7 | Up to 3 months | 
| Read/reviewed the HCP Checklist | Calculated as the percentage of HCPs who answer Yes, all of it or Yes, some of it to question 8 | Up to 3 months | 
| Use of the HCP Checklist prior to initiating treatment | Calculated as percentages of HCPs who answer Always or Sometimes to question 9A | Up to 3 months | 
| Use of the HCP Checklist at each subsequent administration | Calculated as percentages of HCPs who answer Always or Sometimes to question 9B | Up to 3 months | 
| Use of the HCP Checklist for at least 3 months after stopping treatment | Calculated as percentages of HCPs who answer Always or Sometimes to question 9C | Up to 3 months | 
| Primary source from which HCPs learned about the appropriate indication, contraindications and precautions for use, and potential risks for REBLOZYL | Calculated as the percentage of HCPs who tick each source in question 11 (for example, percentage of HCPs that ticked the REBLOZYL summary of product characteristics (SmPC), etc.) | Up to 3 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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REBLOZYL, Myelodysplastic Syndromes, Healthcare Professionals, Post-authorization safety study, Additional educational measures, Beta-thalassemia, Luspatercept