A Drug Utilization Study to Evaluate Indicators of Adherence to the Risk Minimization Measures for Ritlecitinib Using Electronic Healthcare Data in Denmark, France, and Sweden

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

Study drug and medical condition

• Name of medicine, other:
  ritlecitinib tosilate
• Study drug International non-proprietary name (INN) or common name:
  RITLECITINIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AF08) ritlecitinib

  ritlecitinib

Population studied

• Short description of the study population:
  The study population will include patients identified in each data source with a record of treatment with ritlecitinib from 15 September 2023 through 14 September 2028. ; Patients will not be required to have a recorded diagnosis of AA.

Study design details

• Study design:
  This will be a descriptive drug utilization study using data from healthcare databases in Denmark, France, and Sweden.
• Main study objective:
  The study objectives are to: ; 1. Evaluate, to the extent measurable in the available routinely collected data, indicators of health care professional’s (HCP) adherence to the risk minimization measures (RMMs) in accordance with the ritlecitinib SmPC, HCP guide, and Patient Card, specifically: ; • Performing laboratory tests of lymphocyte count, platelet count, hepatitis B/C, and tuberculosis (TB) screening prior to initiation of ritlecitinib treatment; • Performing laboratory tests of lymphocyte count and platelet count at week 4 (± 2 weeks) from initiation of ritlecitinib treatment ; • Avoiding live attenuated vaccines shortly before and during treatment with ritlecitinib ; • No use during pregnancy ; • No use in patients aged < 12 years ; • No use during serious infections; ; 2. Describe the characteristics of patients before initiation of ritlecitinib treatment, in terms of:; • Risk factors for thromboembolic events (including deep vein thrombosis (DVT), pulmonary embolism (PE), and arterial thrombosis) ; • Risk factors for malignancy; • Risk factors for cardiovascular disease (CVD)
• Setting:
  Denmark, France, and Sweden have universal healthcare, whereby routinely collected data continuously accrue in the participating nationwide databases. ; The use of the three population-based data sources will contribute to generalisability of the study results to AA patients in the EU. ; Data sources have been selected given adequate capture of some or all key study variables and relatively large underlying population sizes of patients across the regions. Indeed, the Danish, French, and Swedish databases capture information from nearly the entire populations of their residents. ; These large real-world data sources are assumed to be representative of patient and physician populations in their respective countries and will provide invaluable data to evaluate measurable indicators of HCP’s adherence to the RMMs.
• Outcomes:
  The RMMs recommend performing laboratory screening tests prior to ritlecitinib initiation, including lymphocyte count, platelet count, hepatitis B/C, and TB; performing laboratory tests of lymphocyte count and platelet count 4 weeks after initiation, avoiding live attenuated vaccines shortly before and during treatment, and avoiding use in pregnancy and during serious infections. Ritlecitinib is currently only indicated for those age 12 years or older. ; • Lymphocyte count; • Platelet count; • Screening for TB; • Screening for viral hepatitis B and C; • Live attenuated vaccines; • Measles; • Mumps; • Rubella; • Others as available in data sources; • Pregnancy; • Serious infections; ;
• Data analysis plan:
  This is a descriptive study and the data will be analysed and results presented separately by country. ; Counts and proportions for categorical variables and mean and median with ranges for continuous variables will be calculated to address the study objectives, as below. All proportions will be reported with 95% CIs. ; Data sources vary in collection of laboratory test and vaccine information (data on both laboratory tests and their results are available in Denmark; data on performed laboratory tests, but not results, are available in France; laboratory data are not available in Sweden). Data on vaccines are not available in Sweden.; Counts and proportions will be calculated for:; 1. Patients with evidence of having performed the following laboratory tests and screenings within 3 months before index date:; • Lymphocyte count; • Platelet count; • Screening for TB; • Screening for viral hepatitis B and C; 2. Patients with evidence of having performed the following laboratory tests at week 4 (± 2 weeks) after index date: ; • Lymphocyte count; • Platelet count; 3. Patients with evidence of having received live attenuated vaccines within 4 weeks before index date ; 4. Patients with evidence of having received live attenuated vaccines during treatment with ritlecitinib ; 5. Females in whom pregnancy, as recorded in the available data sources, overlaps with ritlecitinib use; 6. Patients aged <12 years on the index date; 7. Patients with a serious infection before index date; 8. Patients with evidence of having measured risk factors and the number of measured risk factors for thromboembolic events within 12 months before index date; 9. Patients with evidence of having measured risk factors and the number of risk factors for malignancy within 12 months before index date; 10. Patients with evidence of having measured risk factors and the number of risk factors for CVD within 6 months before index date;