ADVANCE POC I Benefit-Risk pillar – testing new approaches to monitoring benefit/risk with pertussis vaccines as test case: benefit-risk analysis of pertussis vaccines in pre-school children comparing whole-cell and acellular formulations in the post-marketing setting

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  Benefit-risk modeling
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Cohort state transition model, Multi-criteria decision analysis (MCDA)

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  PERTUSSIS VACCINE
• Medical condition to be studied:
  Injection related reaction

  Somnolence

  Crying

  Convulsion in childhood

  Lip swelling

  Pertussis

Population studied

• Short description of the study population:
  Children from birth until their school-entry pertussis booster if any (4th or 5th dose) within all eligible ADVANCE databases.
• Age groups:
  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)
• Estimated number of subjects:
  1

Study design details

• Main study objective:
  1.Analyze the benefit-risk of pertussis vaccines in children comparing wP and aP at the time of switch from wP to aP (historical)2.Investigate the impact of uncertainty in benefits, risks and preferences3.Identify the criteria that most likely modify the benefit-risk4.Assess the feasibility of monitoring benefit-risk over time5.Re-analyze the benefit-risk using currently available evidence
• Outcomes:
  Exposure of interest: any whole-cell and acellular pertussis-containing vaccines and their doses in the vaccine scheduleOutcomes:Injection site reactions, fever, somnolence, persistent crying, generalized convulsive seizures, HHE, extensive limb swelling, pertussis, pertussis related death
• Data analysis plan:
  The benefit-risk assessments will be carried out following the Multi-Criteria Decision Analyses (MCDA) methodology. MCDA is a quantitative methodology for appraising alternatives on individual, often conflicting criteria and combining them into one overall appraisal, through incorporating elicited preferences (weights). The preferences will be elicited using MCDA-swing weighting. In addition, several sensitivity analyses will be conducted to investigate the impact of uncertainty in the benefits, risks and preference estimations on the overall benefit-risk balance. A state transition model will be build to generate the effects table, which will be used for the MCDA swing weighting.