A Prospective, Non-Interventional, Post-Marketing Observational Study Evaluating the Effectiveness and Safety of Selpercatinib Capsules in Adults and Pediatric Patients with Advanced or Metastatic RET Fusion-Positive Thyroid Cancer

Study type

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine, other:
  Retevmo
• Anatomical Therapeutic Chemical (ATC) code:
  (L01EX22) selpercatinib

  selpercatinib
• Medical condition to be studied:
  Thyroid cancer

Population studied

• Age groups:
  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  15

Study design details

• Main study objective:
  The primary objective of this study is to evaluate the effectiveness of selpercatinib by assessing real-world objective response rate (rwORR) in a routine clinical practice setting during the follow-up period.
• Outcomes:
  rwORR, rwPFS, rwDoR, rwDCR
• Data analysis plan:
  Primary Objective Analyses: The rwORR will be summarized using descriptive analysis, described in terms of frequencies and percentages, and provide a 95% CI. ; Secondary Objective Analyses: Time-to-event analyses (rwPFS and rwDoR) will be performed using Kaplan-Meier estimates to calculate median and 95% CI and estimates at specific time points (e.g. 6 months, 12 months). Patients who are alive and without documented PD as of a data analysis cut-off date will be right-censored. The censoring date will be determined from the date of last follow-up to the patient without progression. rwDCR will be summarized using descriptive analysis, described in terms of frequencies and percentages, and provide a 95% CI.