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Has the Orphan Regulation met its aims? A critical review of the EU's incentives regime for rare disease patients

By the 1990’s the European Union had fallen behind other developed economies in the encouragement and support its pharmaceutical regulatory framework offered to the development
of new treatments for rare diseases. The aim of the Orphan Regulation was to remedy this. In the article “Has the Orphan Regulation Met its Aims? A Critical Examination of the European Union’s Regime Incentivising Research and Development of Medicines for Patients with Rare Diseases”, published in the European Pharmaceutical Law Review (Volume 2, Issue 4), my colleagues Laëtitia Bénard, Jacqueline Bore and myself examine critically the interplay of the legal instruments underpinning the scheme of orphan incentives with the judgments of the Court of Justice and conclude that the definitions and tests embodied by the Orphan Regulation, which decide which products should be rewarded, have served patients with rare diseases well.

Attributing changes in the behaviour of investors, researchers and pharmaceutical companies to a single legislative act such as the Orphan Regulation is challenging. There is clear evidence, nevertheless, of the increase in the number of medicines available in the European Union for rare diseases, increasing from only 8 prior to the Regulation to more than 140 by the end of 2017. In a sample of 304 orphan designations between 2002 and 2012, 44% were granted in respect of medicines for conditions where there was no satisfactory method of diagnosis, prevention or treatment with the remaining 56% providing ‘a clinically relevant advantage or a major contribution to patient care’. The Orphan Regulation has thus certainly proved its worth in providing a stimulus to innovative research whilst continuing to incentivise competitors to enter a nascent orphan market once it has been created, and is therefore arguably one of the biggest success stories in terms of improving health outcomes and transforming the lives of patients in the EU.

The European Commission has shown itself ready to refine and develop the process leading to the authorisation of new orphan medicinal products to keep pace with scientific developments and the Court of Justice has robustly upheld the entitlement of sponsors to orphan rewards where the tests set out in the Regulation have been met, consistently drawing on the economic imperatives underlying the Regulation to justify its conclusions. As the European Commission continues with its task of assessing the combined impact of the orphan and paediatric regulations on the development of treatments for rare diseases it will be important for them to take account of what we consider to be the demonstrated appropriateness of the legislative tools to their task: the development of over 130 new medicines for rare disease patients in the last 15 years.