EXCEED - A Pan-European Post-Authorisation Safety Study: Risk of Pancreatic Cancer Among Type 2 Diabetes Patients who Initiated Exenatide as Compared with those who Initiated Other non-Glucagon-Like Peptide 1 Receptor Agonists based Glucose Lowering Drugs

Study type
Protocol
Date of Approval
Study reference ID
23_003566
Lay Summary

Diabetes (a condition that causes a person's blood sugar level to become too high) is currently one of the major common chronic diseases worldwide and the number of new individuals that are diagnosed is increasing rapidly. If the high blood sugar levels are caused by the body not making enough of a hormone called insulin, or the insulin it makes is not working properly, this is known as type 2 diabetes. Exenatide is a new treatment for type 2 diabetes among adults, used as a combination treatment together with other blood sugar lowering drugs when desirable effects have not been achieved on previous treatments. During the preclinical and clinical development of exenatide, uncertainties were raised around the potential risk for pancreatic cancer. Additional reports have prompted regulatory communications regarding the potential association between exenatide and risk of pancreatic cancer.
This study will assess the possible risk of pancreatic cancer associated to exenatide use, among patients with type 2 diabetes residing in Europe. Results will be generalisable for the European population of patients with type 2 diabetes using exenatide. Understanding of the treatment’s safety is important to all individuals using exenatide and also benefits individuals with type 2 diabetes in the UK.

Technical Summary

The aim of this study is to assess the association between pancreatic cancer and exposure to any exenatide, compared with exposure to non-Glucagon-Like Peptide-1 Receptor Agonist (GLP-1 RA) based glucose lowering drugs (GLDs), among patients with type 2 diabetes mellitus (T2DM), aged 18 years or older. This is an observational, comparative, cohort safety study based on electronically recorded longitudinal secondary databases, collected separately in each of the included 7 countries (France, Spain, Finland, Denmark, Norway, Sweden), including the UK.
The use of CPRD-Aurum, linked to the Hospital Episode Statistics (HES) database, the Office for National Statistics (ONS), and the National Cancer Registration and Analysis Service (NCRAS) databases enables the inclusion of patient-level data for a large number of T2DM patients, including data on drug prescriptions, cancer outcome, medical history, and other clinical characteristics.
The primary objective of this study is to estimate the incidence rate (IR) and hazard ratio (HR) for pancreatic cancer associated with exposure to exenatide (BYETTA or BYDUREON/ BYDUREON BCise), compared with exposure to non-GLP-1 RA based GLDs, among adult patients with T2DM. Included patients will be followed from index date (study entry, 01January 2006) until the end of the study period (31 December 2023), or censoring (outcome of interest, loss to follow-up, emigration, or death), whichever comes first. The latency period for developing pancreatic cancer will be defined as the first 12 months counting from the index date. All analyses will be conducted in the matched study population using an ‘intention-to-treat’ (ITT) approach, and will be performed separately for each country. The results will be combined using meta-analysis to address the primary and secondary objectives of the study. This post-authorisation safety study (PASS) of exenatide benefits the potential users of this medication, including individuals with T2DM living in the UK.

Health Outcomes to be Measured

The primary outcome of interest will be incidence rate; cumulative incidence; and HR of pancreatic cancer in patients exposed to exenatide (BYETTA or BYDUREON/ BYDUREON BCise), compared with exposure to non-GLP-1 RA based GLDs. The secondary outcome of interest will be incidence rate; cumulative incidence; and HR of pancreatic cancer in patients exposed to exenatide once-weekly formulation (BYDUREON/BYDUREON BCise), compared with exposure to non-GLP-1 RA based GLDs.
A sensitivity analysis (sensitivity analysis 7) planned for the assessment of unmeasured confounding will assess incidence rate; cumulative incidence; and HR of lung cancer/ malignant melanoma of the skin (“negative outcomes”).

Collaborators

Rachel Armstrong - Chief Investigator - IQVIA Ltd ( UK )
Ella Baird - Corresponding Applicant - IQVIA Ltd ( UK )
Fabian Hoti - Collaborator - IQVIA Finland Oy
Farokh Master - Collaborator - IQVIA Ltd ( UK )
Giorgi Tskhvarashvili - Collaborator - StatFinn Estonia OU
Sophia Fleming - Collaborator - IQVIA Ltd ( UK )

Linkages

HES Admitted Patient Care;HES Outpatient;NCRAS Cancer Registration Data;NCRAS Systemic Anti-Cancer Treatment (SACT) data;ONS Death Registration Data;Patient Level Index of Multiple Deprivation