Abstract: Evidence Resulting from Chart Review Methodology Applied to Named Patient Programme Participation and Compassionate Medication Use: Periapproval Approximation of Post-Market Practice Patterns and Costs

 

Abstract: Evidence Resulting from Chart Review Methodology Applied to Named Patient Programme Participation and Compassionate Medication Use: Periapproval Approximation of Post-Market Practice Patterns and Costs

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.