OBJECTIVES Compassionate use programmes provide peri-approval drug access based on unsolicited physician requests for patients with unmet need. Practice pattern evaluations in this context, using chart review methodology, permits the collection of pre-approval data outside of clinical trial settings that can approximate real-world post-market use. Data can be used to inform important economic evaluations, value dossiers, and drug safety assessments. METHODS Study design and operational considerations related to chart review studies of compassionate use populations have been summarized by evaluating three multi-national case studies. RESULTS The source populations of patients were drawn from compassionate use programmes providing oral anti-cancer therapies. These were initiated pre-approval following positive clinical trial findings.  Countries included Australia, Belgium, Greece, Ireland, Israel, Italy, Netherlands, New Zealand, Spain, Switzerland and the United Kingdom. Data including patient characteristics, patterns of care and drug dosing, duration of treatment and reasons for treatment discontinuation, overall survival, clinical benefit, progression free survival and adverse and serious adverse events are being collected to inform health economic and other burden of illness assessments. Site and patient selection was performed using compassionate use enrollment data, facilitating an efficient study start-up.  Data were cleaned at point of data entry and via an electronic query process in real-time resulting in tailored international datasets. Patients provided consent for their medical data to be used prior to initiating compassionate use therapy, and additional ethical approval for the chart review was sought on a local and/or national level. Chart review study design requires a balance between scientific and operational rigor and practicality and feasibility.  CONCLUSIONS Peri-approval chart review studies of patients in compassionate use programmes offer an important opportunity to characterize patterns of use and associated treatment costs as well as the clinical impact of investigational medications in non-trial settings to inform clinical, health economic and market access decisions.