A systematic review of moral reasons on orphan drug reimbursement

Abstract

Background The 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. Results With 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. Conclusion Results 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.

How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries

“…In addition, our results remained inconclusive as to whether disease rarity legitimizes the implementation of a separate framework for (ultra-)orphan drugs or to what extent this criterion warrants more flexibility when assessing these treatments. As a way forward, the implementation of decision-making frameworks such as multi-criteria decision analysis would allow a fair and consistent evaluation of these arguments ( Blonda et al, 2021 ; Zimmermann et al, 2021 ).…”

Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives

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“…Arguments have been made for higher WTP thresholds for gene therapies [8,52,[82][83][84][85]. However, as noted, there appears to be inconclusive support among the general public for such special considerations [62][63][64][65][66]. As a result, reimbursing these treatments will ultimately lead to inefficiencies, as more health could have been produced at the same or lower cost with other health technologies [58].…”

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“…A relatively new challenge for solidarity-based healthcare systems is to incorporate very expensive medicines for rare diseases [1][2][3][4][5][6]. In the Dutch healthcare system, based on solidarity, citizens pay taxes and mandatory health insurance premiums to finance accessible healthcare for everyone.…”