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Post-marketing access to orphan drugs: a critical analysis of health technology assessment and reimbursement decision-making considerations

Authors Iskrov G, Stefanov R

Received 10 November 2013

Accepted for publication 3 December 2013

Published 9 January 2014 Volume 2014:4 Pages 1—9

DOI https://doi.org/10.2147/ODRR.S43409

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3


Georgi Iskrov, Rumen Stefanov

Department of Social Medicine and Public Health, Medical University of Plovdiv, Plovdiv, Bulgaria

Abstract: This study aims to explore the current rationale of post-marketing access to orphan drugs. As access to orphan medicinal products depends on assessment and appraisal by health authorities, this article is focused on health technology assessment (HTA) and reimbursement decision-making considerations for orphan drugs. A critical analysis may identify important factors that could predetermine the combined outcomes of these two processes. Following this objective, an analytical framework was developed, comprising three overlaying issues: to outline what is currently done and what needs to be done in the field of HTA of orphan drugs, to synthesize important variables relevant to the reimbursement decision-making about orphan drugs, and to unveil 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. Reimbursement decision-making for orphan drugs is a debate of policy priorities, health system specifics, and societal attitudes. 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. The only reasonable way to accept a higher valuation of orphan drug benefits is if these are demonstrated empirically. Rarity means that the quality of orphan drug evidence is not the same as for conventional therapies. Closing this gap is another crucial point for the timely access to these products. The generation of evidence goes far beyond pre-market authorization trials and requires transnational cooperation and coordination. 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.

Keywords: orphan drugs, health technology assessment, reimbursement, decision-making


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