PNS188 MULTI-Criteria Decision Analysis on the Inclusion of Orphan Drugs for Reimbursement in Bulgaria, France, Germany, Sweden, UK: A Comparative Analysis

Abstract

The objective of this study is to identify, describe, analyze & compare the main criteria in appraising medicines for rare diseases (RD) – Bulgaria vs. France, Germany, Sweden, UK. This study demonstrates some of the main differences and similarities in the HTA appraisal for orphan drugs across the selected countries. A comparative analysis of published regulatory requirements & appraisal criteria by HTA bodies for RD medicines was conducted for Bulgaria, France, Germany, Sweden, UK. The choice of countries is made due to a mandatory requirement in Bulgaria for at least one positive opinion by an HTA body from the selected countries as a condition for HTA approval. Bibliographic databases, the Internet and government/HTA organization websites were searched. The collected data was synthesized in Excel, qualitative comparative analyses were performed. Five key domains were analyzed and compared: special HTA appraisal process for orphan drugs and medicines for RD, clinical effectiveness, cost-effectiveness and budget impact, moral and ethical considerations. Almost all countries have multiple criteria for HTA appraisal of orphan drugs. All of them except Bulgaria have special HTA process for orphan drugs and RD. Almost all of them typically require clinical assessment, cost-effectiveness, and budget impact analysis (BIA). However, BIA is not taken into account in committee's considerations in the UK (only value is). Moral and ethical aspects are an additional criterion considered only in Bulgaria. Results presented in table. This study demonstrates that HTA process for orphan drugs and RD typically require similar criteria as drugs for common conditions. However, specific submission requirements could be beneficial for a faster, and more transparent decision-making by national authorities. When comparing with countries which require different criteria for HTA appraisal, the moral and ethical considerations In Bulgaria seem neglected.