A systematic review of moral reasons on orphan drug reimbursement

Bettina M Zimmermann,Matthias R Baumgartner,Johanna Eichinger

doi:10.1186/s13023-021-01925-y

Abstract

BackgroundThe number of market approvals of orphan medicinal products (OMPs) has been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for patients suffering from rare diseases, they are usually very expensive. The growing number of approved OMPs increases their budget impact despite their low prevalence, making it pressing to find solutions to ethical challenges on how to fairly allocate scarce healthcare resources under this context. One potential solution could be to grant OMPs special status when considering them for reimbursement, meaning that they are subject to different, and less stringent criteria than other drugs. This study aims to provide a systematic analysis of moral reasons for and against such a special status for the reimbursement of OMPs in publicly funded healthcare systems from a multidisciplinary perspective.ResultsWith a systematic review of reasons, we identified 39 reasons represented in 243 articles (scientific and grey literature) for and against special status for the reimbursement of OMPs, then categorized them into nine topics. Taking a multidisciplinary perspective, we found that most articles came from health policy (n = 103) and health economics (n = 49). More articles took the position for a special status of OMPs (n = 97) than those against it (n = 31) and there was a larger number of reasons identified in favour (29 reasons) than against (10 reasons) this special status.ConclusionResults suggest that OMP reimbursement issues should be assessed and analysed from a multidisciplinary perspective. Despite the higher occurrence of reasons and articles in favour of a special status, there is no clear-cut solution for this ethical challenge. The binary perspective of whether or not OMPs should be granted special status oversimplifies the issue: both OMPs and rare diseases are too heterogeneous in their characteristics for such a binary perspective. Thus, the scientific debate should focus less on the question of disease prevalence but rather on how the important variability of different OMPs concerning e.g. target population, cost-effectiveness, level of evidence or mechanism of action could be meaningfully addressed and implemented in Health Technology Assessments.

Highlights

The number of market approvals of orphan medicinal products (OMPs) has been increasing steadily in the last 3 decades
Not all approved OMPs are targeted towards rare diseases: In light of the ongoing research progress in precision medicine, targeted therapies for common diseases might fall into the definition of orphan drug regulations
In light of the increasing number of high-priced OMP approvals and the unsolved ethical challenges concerning their reimbursement in publicly funded healthcare systems, this study aims to provide a systematic analysis of moral reasons for and against special status for the reimbursement of OMPs in publicly funded healthcare systems from an interdisciplinary perspective

Summary

Introduction

The number of market approvals of orphan medicinal products (OMPs) has been increasing steadily in the last 3 decades. According to the European Commission Regulation [1], an orphan designation pertains exclusively to OMPs that are “intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10,000 persons in the Community” (Article 2.1) or that fulfill the condition that “without incentives it is unlikely that the marketing of the medicinal product in the Community would generate sufficient return to justify the nece[s]sary investment”. Not all approved OMPs are targeted towards rare diseases: In light of the ongoing research progress in precision medicine, targeted therapies for common diseases might fall into the definition of orphan drug regulations. A US-based empirical study did not find targeted therapies to mainly cause the increase in OMP approvals [7]

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