Post-marketing access to orphan drugs

A new review article explores the current rationale of post-marketing access to orphan drugs, focusing on health technology assessment (HTA) and reimbursement decision-making considerations. An analytical framework is developed to address three overlaying issues: what is currently done and what needs to be done in the field of HTA of orphan drugs; which are the important variables relevant to the reimbursement decision-making about orphan drugs; and what is the relationships between theory and practice. Methods for economic evaluation, cost-effectiveness threshold, budget impact, uncertainty of evidence, criteria in reimbursement decision-making, and HTA research agenda are all explored and discussed from an orphan drug perspective. The study recommends that health authorities need to pursue a multidisciplinary analysis on a range of criteria, ensuring an explicit understanding of the trade-offs for decisions related to eligibility for reimbursement. Early constructive dialogue among orphan drug stakeholders and elaboration of orphan drug-tailored methodology tools could set the scene for ongoing accumulation of evidence, as well as for proper and timely assessment and appraisal. The article is published in Orphan Drugs: Research and Reviews Journal of Dovepress.