Tafamidis Pregnancy Surveillance Study

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Non-interventional surveillance study

Study drug and medical condition

• Name of medicine:
  VYNDAQEL
• Study drug International non-proprietary name (INN) or common name:
  TAFAMIDIS MEGLUMINE

  TAFAMIDIS
• Anatomical Therapeutic Chemical (ATC) code:
  (N07XX08) tafamidis

  tafamidis
• Medical condition to be studied:
  Acquired ATTR amyloidosis

Population studied

• Short description of the study population:
  All women diagnosed with ATTR exposed to tafamidis during or within 1 month prior to pregnancy, reported as exposure during pregnancy (EDP) events in interventional or noninterventional clinical studies in the tafamidis development program, via spontaneous reports, solicited cases or compassionate use reports, will be included.
• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)
• Special population of interest:
  Pregnant women

Study design details

• Study design:
  Routine pharmacovigilance activities and data collected via the TESPO program will be used to describe birth outcome frequency (live full term or premature birth, spontaneous or induced abortion, and stillbirth) of women exposed to tafamidis during or within 1 month of pregnancy.
• Main study objective:
  1) Describe birth outcome frequency within the reported pregnancies (live full term or premature birth, spontaneous or induced abortion, and stillbirth) in women with ATTR exposed to tafamidis during or within 1 month prior to pregnancy.; 2) Describe the frequency of reported fetal, neonate and infant outcomes within the reported pregnancies (major congenital malformations/anomalies [overall and specific], low birth weight, small for gestational age (SGA), preterm birth, low Apgar score at 1 and 5 minutes, and infant milestone status at 6 and 12 months) following tafamidis exposure in women with ATTR during or within 1 month prior to pregnancy.
• Setting:
  Events of pregnancy are reported to Pfizer:; via clinical trials (when clinical trial participant reports pregnancy during trial);; via non-interventional studies;; via spontaneous reporting (post-marketing);; through direct contact from patients and health care professionals;; through unsolicited requests for pregnancy safety information (via Pfizer Medical Information Department);; or through contact with sales representatives.; ; Both maternal and paternal exposure to tafamidis were historically captured in TESPO, however, since initiation of this program it has been determined in nonclinical studies that the risk for male-mediated developmental toxicity is considered low. Consequently, exposure during pregnancy due to paternal exposure will not be required to be captured in study B3461091.; ; For EDP cases occurring during sponsored clinical trials investigators are trained on how to report the case to the Sponsor, using standard pharmacovigilance forms.; ; For all other reportings, information regarding how to contact the Sponsor (per country specific labeling guidelines) in the event of a known pregnancy exposure is provided in the summary of product characteristics, the US package insert, the patient information leaflet, and in informational materials provided (eg, scientific meetings and direct communication) to health care professionals.;
• Outcomes:
  Measures of pregnancy and birth outcomes include preterm birth, very preterm birth, low birth weight, very low birth weight, infant mortality, fetal death (stillbirth), and spontaneous fetal losses.; ; In the event of a live birth, the obstetric HCP will be sent a release form to be signed by the female individual to allow the Sponsor to contact the infant’s pediatric HCP for additional infant outcome data. The Sponsor will contact the pediatric HCP to complete the TESPO Infant 6 and 12-Month Follow-up Form by phone or fax.; ; If anomalies are noted on the EDP Form or the TESPO Infant Follow-up Forms, a more detailed investigation may be initiated by the Sponsor. For efficiency and practicality, the focus is commonly on major structural anomalies. These are defined as structural changes that have significant medical, social or cosmetic consequences for the; affected individual and typically require medical intervention. Examples include cleft lip and spina bifida. Major structural anomalies are the conditions that account for most of the deaths, morbidity and disability related to congenital anomalies.
• Data analysis plan:
  No formal data analysis or hypothesis testing will be conducted. Data collected on exposures and outcomes will be summarized descriptively.