title: Use and outcomes of targeted therapies in early and metastatic HER2-positive breast cancer in Australia: protocol detailing observations in a whole of population cohort.
abstract: The management of human epidermal growth factor receptor 2 (HER2)-positive breast cancer (BC) has changed dramatically with the introduction and widespread use of HER2-targeted therapies. However, there is relatively limited real-world information on patterns of use, effectiveness and safety in whole of population cohorts. The research programme detailed in this protocol will generate evidence on the prescribing patterns, safety monitoring and outcomes of patients with BC treated with HER2-targeted therapies in Australia.
  
  
  
  Our ongoing research programme will involve a series of retrospective cohort studies that include every patient accessing Commonwealth-funded HER2-targeted therapies for the treatment of early BC and advanced BC in Australia. At the time of writing, our cohorts consist of 11 406 patients with early BC and 5631 with advanced BC who accessed trastuzumab and lapatinib between 2001 and 2014. Pertuzumab and trastuzumab emtansine were publicly funded for metastatic BC in 2015, and future data updates will include patients accessing these medicines. We will use dispensing claims for cancer and other medicines, medical service claims and demographics data for each patient accessing HER2-targeted therapies to undertake this research.
  
  
  
  Ethics approval has been granted by the Population Health Service Research Ethics Committee and data access approval has been granted by the Australian Department of Human Services (DHS) External Review Evaluation Committee. Our findings will be reported in peer-reviewed publications, conference presentations and policy forums. By providing detailed information on the use and outcomes associated with HER2-targeted therapies in a national cohort treated in routine clinical care, our research programme will better inform clinicians and patients about the real-world use of these treatments and will assist third-party payers to better understand the use and economic costs of these treatments.
authors.affiliation: Medicines Policy Research Unit, Centre for Big Data Research in Health, UNSW, Sydney, New South Wales, Australia.
authors.forename: Benjamin
authors.initials: B
authors.lastname: Daniels
authors.affiliation: School of Medicine, University of Notre Dame Australia, Sydney, New South Wales, Australia.|NHMRC Clinical Trials Centre, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: Sarah J
authors.initials: SJ
authors.lastname: Lord
authors.affiliation: NHMRC Clinical Trials Centre, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: Belinda E
authors.initials: BE
authors.lastname: Kiely
authors.affiliation: Sydney School of Public Health, Sydney Medical School, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: Nehmat
authors.initials: N
authors.lastname: Houssami
authors.affiliation: Centre for Health Economics Research and Evaluation, University of Technology Sydney, Sydney, New South Wales, Australia.
authors.forename: Philip
authors.initials: P
authors.lastname: Haywood
authors.affiliation: Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, Massachusetts, USA.
authors.forename: Christine Y
authors.initials: CY
authors.lastname: Lu
authors.affiliation: University of Queensland, Brisbane, Queensland, Australia.
authors.forename: Robyn L
authors.initials: RL
authors.lastname: Ward
authors.affiliation: Medicines Policy Research Unit, Centre for Big Data Research in Health, UNSW, Sydney, New South Wales, Australia.
authors.forename: Sallie-Anne
authors.initials: SA
authors.lastname: Pearson
mesh_terms: D000080044:Ado-Trastuzumab Emtansine; D000368:Aged; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D000074322:Antineoplastic Agents, Immunological / Q000627:therapeutic use*; D001315:Australia; D001943:Breast Neoplasms / Q000188:drug therapy* / Q000378:metabolism / Q000473:pathology; D015331:Cohort Studies; D006801:Humans; D007150:Immunohistochemistry; D017403:In Situ Hybridization; D008453:Maytansine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D008875:Middle Aged; D058990:Molecular Targeted Therapy / Q000706:statistics & numerical data; D009362:Neoplasm Metastasis; D009367:Neoplasm Staging; D018719:Erb-b2 Receptor Tyrosine Kinases / Q000378:metabolism*; D000068878:Trastuzumab / Q000627:therapeutic use*
chemical_list: D000080044:Ado-Trastuzumab Emtansine; D061067:Antibodies, Monoclonal, Humanized; D000074322:Antineoplastic Agents, Immunological; D008453:Maytansine; D018719:Erb-b2 Receptor Tyrosine Kinases; D000068878:Trastuzumab; C508053:ERBB2 protein, human; C485206:pertuzumab
keywords: HER2; breast cancer; observational study; pharmacoepidemiology; trastuzumab