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. 2022 Aug 23;10(9):1600.
doi: 10.3390/healthcare10091600.

Making Sure That Orphan Incentives Tip the Right Way in Europe

Denis Horgan  1   2 , Jasmina Koeva-Balabanova  3 , Ettore Capoluongo  4   5 , Beata Jagielska  6 , Ivana Cattaneo  7 , Marta Kozaric  1 , Birute Tumiene  8 , Jean-Paul El Ahl  9 , Jonathan A Lal  2   10 , Dipak Kalra  11 , Núria Malats  12
Affiliations

Making Sure That Orphan Incentives Tip the Right Way in Europe

Denis Horgan et al. Healthcare (Basel). .

Abstract

The delicate balance of funding research and development of treatments for rare disease is only imperfectly achieved in Europe, and even the current provisional equilibrium is under a new threat from well-intentioned policy changes now in prospect that could-in addition to the intrinsic complexities of research-reduce the incentives on which commercial activity in this area is dependent. The European Union review of its pharmaceutical legislation, for which proposals are scheduled to appear before the end of 2022, envisages adjusting the decade-old incentives to meet objectives that are more precisely targeted. However, researchers, physicians, patients and industry have expressed concerns that ill-considered modifications could have unintended consequences in disrupting the current balance and could reduce rather than increase the flow of innovative treatments for rare diseases.

Keywords: EU regulation; drug development; incentives; orphan medicines; patients; pharmaceutical strategy; policy framework; rare diseases; regulation.

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Conflict of interest statement

D.H. and M.K. are employees of the European Alliance for Personalised Medicine, which receives funding from both the public and private sectors which includes medical societies, pharmaceutical and diagnostic companies, patients’ organisations, as well as EU grants. Jean-Paul El Ahl is an employee of Merck. Ivana Cattaneo is an employee of Novartis receives compensation (salary, stock options).

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