Dr. Éloïse Gennet, junior professor in European health law at Aix Marseille University, CNRS, DICE, CERIC, Aix-en-Provence, France, has contributed valuable insights to the European Joint Programme on Rare Diseases’ (EJP RD) latest MOOC on “Innovative Therapies and Personalized Medicine for Rare Disease.”

In her comprehensive module, Dr. Gennet addresses the complex landscape of equity access to innovative treatments for rare diseases within the European regulatory framework. Her contribution demystifies the intricate legal and ethical considerations that shape treatment accessibility for the estimated 36 million Europeans affected by rare diseases.

The module explores critical aspects of the European Union’s approach to rare disease treatment, including the pivotal Regulation 141/2000 on orphan medicinal products and its impact on treatment development. Dr. Gennet’s analysis reveals how the regulation has established crucial incentives for pharmaceutical companies while highlighting persistent challenges in ensuring equitable access across member states.

A key focus of her contribution is the examination of various stakeholders’ roles, from governmental institutions to patient advocacy groups, in shaping rare disease treatment policies. The material also addresses recent developments in EU pharmaceutical legislation and ongoing efforts to improve the regulatory framework’s effectiveness.

Dr. Gennet’s work forms part of the ANR-funded I-BioLex project and represents a significant contribution to understanding the intersection of legal frameworks, ethical considerations, and practical challenges in rare disease treatment access across Europe.