PSY82 - How influential are Patient and professional group submissions on reimbursement decisions for European Medicines Agency Orphan Drugs?

Abstract

Uncertainty often surrounds orphan-drug reimbursement, given the small sample sizes and limited availability of clinical trials. Consequently, manufacturers and Health Technology Assessment (HTA) agencies are turning to real-world evidence to understand the patient experience. Both SMC and NICE encourage patient/professional group involvement in the HTA reimbursement process, but do these submissions influence reimbursement decisions? Using the Context Matters’ proprietary database, the analysis was restricted to full HTA submissions from NICE and SMC for European Medicines Agency (EMA) orphan drugs. The reimbursement decision, the presence of a patient/professional group submission, and any agency comments on the submissions were extracted from each assessment. Reimbursement decisions were categorized as either positive or negative. Fifty assessments across 13 disease conditions were analyzed. There was no association between patient/professional group submissions and positive reimbursement decisions (p=0.70); 48% (20) of assessments with a patient/professional group submission resulted in a positive decision while 52% (22) of assessments with a patient/professional group submission resulted in a negative decision. When stratified by agency or disease condition, there was still no relationship between decision and patient/professional group submission. Individual assessments were examined to determine how SMC and NICE use patient/professional group submissions. Results indicate that patient/professional group submissions are not directly associated with positive reimbursement decisions; this presentation will detail how SMC and NICE use these submissions in their decision-making. Due to the increasing rate of development in the orphan-drug space and the importance of incorporating the patient voice, manufacturers need to understand how these submissions influence HTA agencies.