A checklist for managed access programmes for reimbursement co-designed by Canadian patients and caregivers.

摘要: INTRODUCTION Reimbursement decisions on orphan drugs carry significant uncertainty, and as the amount increases, so does risk of making a wrong decision, where harms outweigh benefits. Consequently, patients often face limited access to drugs. Managed programmes (MAPs) are mechanism for managing while enabling potentially beneficial Patients their caregivers have expressed support these see patient input critical successful implementation. However, they yet be systematically involved in design. OBJECTIVE The aim this study was co-design with tool development managed programmes. METHODS Building upon established relationships Canadian Organization Rare Disorders, project team collaborated using principles participatory action research. Data were collected at two workshops analysed thematic network approach. RESULTS co-designed checklist comprised six aspects an ideal MAP relating accountability (programme goals); governance (MAP-specific committee oversight, input, international collaboration); evidence collection (outcome measures continuation criteria, on-going monitoring registries). They recognized that health-care resources finite considered disease or drug eligibility criteria deciding when use (eg treating diseases which there no other legitimate alternatives). CONCLUSIONS A caregiver-designed created, emphasized involvement transparency. Further research is needed examine feasibility roles stakeholders.