PSY106 - To What Extent Do Disease and Treatment Characteristics Influence Hta-Based Recommendations for A Sample of Orphan Drugs In Three Countries, and Could These Indicate Whether Orphan Drugs Have A “Special Status”?

Abstract

Routine HTA methods may not adequately capture all the important considerations of a treatment’s value and the impact of the condition on the patient given that evidence is often incomplete. This study aims to explore the influence of broader considerations of scientific and social value judgments on reimbursement decisions for a sample of orphan drugs. To identify and compare the extent to which these broader considerations not captured by the incremental cost-effectiveness ratio (ICER) influenced HTA decision-making process in three countries; and, on this basis, explore whether orphan drugs have a “special status”. Countries included were England, Scotland and Sweden. Ten drug-indication pairs with EMA orphan designation and all appraised by NICE were selected. Publicly available HTA reports were coded using thematic analysis to systematically identify and compare these broader considerations across countries using an existing analytical framework. 108 different other considerations were identified and grouped into 15 clusters based on the information provided. The most common related to the nature of the disease, and considerations based on rarity or unmet need. 52% were one of the main reasons for the decision, and in some cases, were also a pivotal factor in accepting high and uncertain ICERs. Categorising these as social or scientific value judgments was done to identify areas where further elicitation of societal preferences, and where more consistency and transparency in their use are needed, respectively. Each of these was then compared to determine whether they pertained specifically to orphan drug or rare disease characteristics. Considerable variation was seen in the application of these broader considerations. Identifying these is a way forward to highlight areas where more research, or consistency and transparency are needed. Some of these other considerations may also favour orphan drugs, furthering the debate around whether orphan drugs deserve special status.