Comparative Effectiveness of Roctavian to Standard of Care

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Evaluation of patient-reported outcomes

  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  ROCTAVIAN
• Study drug International non-proprietary name (INN) or common name:
  VALOCTOCOGENE ROXAPARVOVEC
• Anatomical Therapeutic Chemical (ATC) code:
  (B02BD15) valoctocogene roxaparvovec

  valoctocogene roxaparvovec
• Medical condition to be studied:
  Haemophilia A without inhibitors

Population studied

• Short description of the study population:
  Adult PwSHA without a history of (or current) inhibitors to FVIII in Germany ;
• 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)
• Special population of interest:
  Other
• Special population of interest, other:
  Males with Haemophilia A
• Estimated number of subjects:
  300

Study design details

• Study design:
  Prospective non-interventional study
• Main study objective:
  • Primary objective: To compare the annualized bleeding rate for treated bleeds. ; • Secondary objectives: ; o To compare the annualized bleeding rate for major, life threatening, and joint bleeds. ; o To compare the proportion of people with zero bleeds for treated, major, life threatening, and joint bleeds. ; o To compare the use of hemostatic medications. ; o To compare joint health, quality of life, and pain. ; o To compare safety events of interest. ; o To describe time to resumption of prophylactic treatment among persons administered Roctavian.
• Setting:
  Germany; Data collection beginning August 30, 2024
• Comparators:
  Standard of Care (SoC) hemostatic prophylaxis treatments
• Outcomes:
  • Bleeding Events ; • SoC Hemostatic Treatment Usage ; • Clinical Outcome Assessment Tools ; o Hemophilia Quality of Life assessment (Haemo-QoL-A) ; o Hemophilia Joint Health Score (HJHS) ; o Brief Pain Inventory-Short Form (BPI-SF) ; o Safety Events
• Data analysis plan:
  Comparison of outcomes were planned between the Roctavian cohort and SoC cohort after propensity score adjustment for differences in baseline characteristics (e.g., demographic and clinical variables recorded in the DHR prior to the index date). Due to the small sample sizes, no comparative analyses were conducted.
• Summary results:
  Uptake and enrollment in 270-603 was <10% of the sample size required per the sample size calculation conducted by the Institute for Quality and Efficiency in Health Care (IQWiG) and detailed in the study protocol, at the time of study closure. The limited uptake and small sample size constrains interpretability of the results and indicated that the study was futile in providing meaningful data on the comparative effectiveness of Roctavian to SoC hemostatic prophylaxis after the AbD specified recruitment period.