POSB270 Does Orphan Drug Status Confer Any Benefits for Products Undergoing HTA Appraisal By NICE in England?

Abstract

Orphan drug status is granted for medicines proposing to target rare diseases with a high level of unmet need, offering incentives for such products. Health Technology Assessment (HTA) agencies will often consider orphan status during their assessments. The purpose of this study is to compare the assessments of orphan and non-orphan drugs by the National Institute of Health and Care Excellence (NICE) since 2018 and determine if orphan status confers any benefit. Single Technology Appraisals and Highly Specialised Technologies published by NICE between January 2018 and June 2021 were identified. For each appraisal, data were extracted on the outcomes, the use of financial agreements and the time from final scope to FAD/FED. The data were analysed to compare the outcomes of orphan and non-orphan drugs. 163 STAs were identified, of which 22% were orphan drugs. A further 8 HSTs (100% orphan drug status) were identified. The overall STA success rate was 90.8%, with no significant difference between the orphan (90.6%) and non-orphan (91.4%) group. In contrast, the HST success rate was 100%. Orphan drugs were more likely to require a financial agreement: 87% STAs and 87.5% HSTs had one, compared to 77% of non-orphan drugs. The timelines for orphan drugs also took ∼10% longer on average, 412 days for STAs and 542 days for HSTs, with non-orphan drugs taking an average of 369 days. Orphan drug status does not confer any benefit during a NICE assessment. The STAs of orphan and non-orphan drugs have a comparable success rate, and orphan drugs are more likely to require a financial agreement and take longer to be assessed. The 100% success rate of HSTs is an exception, but with so few orphan drugs meeting the strict criteria for the HST process, it is a small one.