PSY16 - Orphan Drug Access: Risk/Reward Analysis of Local Clinical Development in Chile

Abstract

As many orphan drug (OD) manufacturers expand their businesses across emerging markets(EM) they are faced with restrictive and often non-existent funding pathways. As a result pricing and reimbursement (P&R) negotiations remain very challenging, pushing manufactures to explore the value of local clinical development in supporting access. To a) understand the value of local clinical development for payers, to support reimbursement in Chile, and b) compare and contrast against four other emerging markets: Russia, Turkey, India and China. In-depth interviews were conducted with 3 stakeholders per market including national payers, reimbursement committee advisors and rare diseases clinical specialists, to understand a) Current OD funding pathways; b) P&R decision drivers; and c) Impact of local development on reimbursement. Selected value drivers were used to develop a scale and qualitatively measure the risk/reward analysis of local clinical development on OD access. The impact of local development on OD reimbursement varies across markets. Respondents in Chile (n=3) highlighted that clinical development will have a strong influence on future willingness to pay, but the opportunity will remain small due to lack of clarity around long-term funding. In Turkey (n=3), providing access to patients through compassionate use schemes, prior to obtaining registration, positively impacts reimbursement.While in Russia, conducting a local clinical trial adds no value to the P&R decision process as cited by payers (n=3). As for India and China where price remains the overriding factor, respondents (n=6) stated that national reimbursement remains a distant prospect. High clinical experts’ awareness, strong influence of advocacy groups and developing OD funding pathways renders Chile and Turkey the most attractive EM for local development. While in comparison, the generation of local patient data is also favoured across the remaining markets, price remains the overriding factors influencing access and reimbursement.