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

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)

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.

Study Overview

• Status: Completed
• Conditions: Myelodysplastic Syndromes; Beta-thalassemia

• Study Type: Observational
• Enrollment (Actual): 187

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria
    • Local Institutions
• Belgium
  • Brussels, Belgium
    • Local Institutions
• Germany
  • Berlin, Germany
    • Local Institutions
• Italy
  • Rome, Italy
    • Local Institutions

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Healthcare Professionals (HCPs) who were part of the REBLOZYL aRMMs dissemination list and who practice in any of the participating EEA countries.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Healthcare Professionals (HCPs); 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?

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

Collaborators and Investigators

• Sponsor: Celgene
• Investigators: Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

Helpful Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

Study record dates

Study Major Dates

• Study Start (Actual): July 26, 2021
• Primary Completion (Actual): July 21, 2022
• Study Completion (Actual): July 21, 2022

Study Registration Dates

• First Submitted: July 13, 2021
• First Submitted That Met QC Criteria: July 13, 2021
• First Posted (Actual): July 22, 2021

Study Record Updates

• Last Update Posted (Actual): July 12, 2024
• Last Update Submitted That Met QC Criteria: July 11, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: Myelodysplastic Syndromes; Luspatercept; Beta-thalassemia; Healthcare Professionals; REBLOZYL; Post-authorization safety study; Additional educational measures
• Additional Relevant MeSH Terms: Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Precancerous Conditions; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Myelodysplastic Syndromes; Preleukemia; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: ACE-536-MDS-005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria.

Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at:

https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html

• IPD Sharing Time Frame: See Plan Description
• IPD Sharing Access Criteria: See Plan Description
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated device product: No