Real-World (RW) Elranatamab Administration: Step Up Dosing (SUD), Treatment Patterns, and Healthcare Resource Utilization (HCRU) in Japan MDV Data (SUMMIT)

Study type

• Study topic:
  Disease /health condition
• Study topic, other:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Healthcare resource utilisation

  Other
• If ‘other’, further details on the scope of the study:
  Treatment patterns with secondary collection of structured data
• Data collection methods:
  No individual level data collected for the purpose of the study

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Retrospective cohort study

Study drug and medical condition

• Name of medicine:
  ELREXFIO
• Study drug International non-proprietary name (INN) or common name:
  ELRANATAMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01FX32) elranatamab

  elranatamab

Population studied

• Short description of the study population:
  The study population will comprise of individuals aged 18 and older who initiated elranatamab between March 26, 2024, the PMDA approval date for elranatamab, and the end of data availability. ; All patients who meet the inclusion and exclusion criteria will be extracted from MDV and included in the study.; ; Inclusion criteria:; - First hospital health claim with an administration of elranatamab on or after March 26, 2024. ; - Have a diagnosis of MM.; - Aged ≥18 years at the time of first hospital claim for elranatamab (i.e., index date).; ; Exclusion Criteria:; - Evidence of administration of elranatamab as part of a clinical trial.
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)

Study design details

• Study design:
  This is a retrospective observational study utilizing de-identified data from Japan Hospital administrative claims data (Medical Data Vision [MDV]). ; The study population will include all adults aged 18 years or older with an MM diagnosis who initiate elranatamab on or after March 26, 2024.
• Main study objective:
  Research question and objectives: ; ; Research question: What are the characteristics of adult patients with MM who initiate elranatamab in Japan, and the SUD process, treatment patterns, HCRU, and safety associated with elranatamab administration? ; ; The following objectives will be assessed among patients with MM receiving elranatamab: ; ; Primary objectives: ; Objective 1: To describe patients with MM initiating elranatamab, including demographics, treatment and medical history. ; Objective 2: To describe and characterize elranatamab utilization, including timing, administration and dosing, during the follow-up period, including the step-up dosing (SUD) period. ; ; Secondary objectives: ; Objective 3: To describe HCRU and costs associated with elranatamab usage during the follow-up period, including the SUD period. ; Objective 4: To describe the use of other treatments for MM including supportive therapy during the follow-up period. ; Objective 5: To describe incidence and prevalence of cytokine release syndrome (CRS), immune effector cell associated neurotoxicity syndrome (ICANS), and cytopenias during the SUD period. ; Objective 6: To describe infection incidence and prevalence during the follow-up period. ; ; Exploratory objectives: ; Exploratory Objective 1: To describe the use of supportive medications during the SUD period.
• Setting:
  The study population will be extracted from MDV, a comprehensive resource based on hospital claims data and Diagnosis Procedure Combination (DPC) data. Information on demographics, mortality, laboratory tests, IP, OP, and ED claims, treatments, procedures, and costs are available for approximately 50 million patients. Patients will be identified using the inclusion and exclusion criteria below:; ; Inclusion Criteria; ; Patients must meet all of the following inclusion criteria to be eligible for inclusion in the study: ; First hospital health claim with an administration of elranatamab on or after March 26, 2024. ; Have a diagnosis of MM. ; Aged ≥18 years at the time of first hospital claim for elranatamab (i.e., index date). ; Exclusion Criteria; Patients meeting any of the following criteria will not be included in the study.; Evidence of administration of elranatamab as part of a clinical trial. ; ; The inclusion and exclusion criteria ensure the study captures patients with MM who initiate elranatamab treatment in RW setting after its approval date in Japan. The exclusion criterion removes patients treated within clinical trials to avoid bias from controlled study conditions and ensure generalizability to routine clinical practice. These criteria may limit the number of eligible patients, especially early in the elranatamab post-approval period.
• Outcomes:
  Study endpoints include: elranatamab timing and dosing (i.e., types of doses, time between doses, relative administration intensity), HCRU and costs (i.e., IP/OP/ED claims and associated costs, degree of nursing), other MM treatment post elranatamab administration (i.e., PIs, SCT, supportive therapy), known adverse events (i.e., CRS events occurrence, frequency and associated length of stay, time from index to CRS event), infection (i.e., any infection occurrence, time from index to infection, antibiotics), and supportive medication (i.e., IVIG, acetaminophen, dexamethasone).
• Data analysis plan:
  All analysis will be descriptive in nature and no formal statistical comparisons will be performed between groups. All characteristics and endpoints will be reported separately for each assessment period and patient stratification of interest. ; ; Dichotomous and categorical variables will be summarized by the number and percentage of patients in each category. Continuous variables will be described using mean, standard deviation (SD), median, interquartile range (IQR), minimum, and maximum. If applicable, the frequency and percentage of patients with missing data for each variable will be described. For reporting conventions, mean, median, and SD will be rounded to 1 decimal place. Percentages will be rounded to 1 decimal place. ; ; RAI will be calculated as the cumulative frequency of administration received over the expected number of administrations. Treatment exposure will be assessed across the different follow-up periods. Patients will be censored at last hospital claim for elranatamab. ; ; All-cause HCRU will be measured as the total number IP, OP, and ED that occurred over the follow-up period. Health claims will only be counted once per day to estimate visits. Additionally, the total length of IP stays will be reported among patients with at least one IP visit. ; ; PPPM standardizes HCRU by calculating the average of a specific variable or a number of specific events (e.g., ED health claims, IP health claims) per month for each patient. In this study, PPPM will be used to report ED, IP and OP visits for the follow-up period. ; ; This calculation will allow meaningful comparisons across patients with varying follow-up period lengths, by normalizing resource use to a monthly rate. Summary statistics (mean, median, min, max) will then be reported to understand average monthly utilization patterns across patients.; ; No sensitivity analysis will be conducted as a part of this analysis.; ;