Presentation (CTI)

OBJECTIVES: European Medicine Agency (EMA) designate Priority Medicine (PRIME) for products that address unmet medical needs to incentivize companies. Following Marketing Authorization (MA) of such products, reimbursement submission begins with formal Health Technology Assessment (HTA). Despite high unmet medical needs, HTA agencies across Europe may assess the evidence differently leading to varying reimbursement decisions.Objectives: To analyze HTA decisions of five major European countries’ HTA agencies for drugs with PRIME designation (France, Germany, Italy, Spain, and the UK).
METHODS: EMA database with approved products was used to select products with PRIME designation. We further refined the list to focus on orphan designation products without Advanced Therapy designations. Withdrawn, suspended, or refused products were excluded. We applied our proprietary artificial intelligence (AI) tool to analyze the HTA decisions. The main outcome measures were standardized into common framework to allow across-country comparison; favorable, inconclusive, or unfavorable.
RESULTS: A total of 2,576 entries were in the database and 1,817 were authorized. Of which, 36 products had PRIME designation, and 16 products met the criteria of PRIME, orphan designation and not an Advanced Therapy. Of the 16, one product was not in our database; two products were Opinion not full HTA evaluation. Final sample included 13 products. A total of 59 assessments were made across five HTA agencies; 35 (59%) were favorable, 18 (31%) were inconclusive, and 6 (10%) were unfavorable with noticeable between-country differences in the favorable vs inconclusive.
CONCLUSIONS: Despite products having high unmet needs by regulatory designation, HTA varied significantly with 41% of assessments being inconclusive or unfavorable. Findings have significant implications on timely access to treatment in disease areas with high unmet needs.