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EU revises its pharmaceutical legislation after 20 years: A unique opportunity to put the patient at the center

The European Union (‘EU’) published on 26 April 2023 its long-awaited EU pharmaceutical package. The package includes the revision of legislations that have been in place for nearly 20 years in the EU. In the field of rare diseases, the 2001 Orphan Medicinal Product Directive contributed to bringing many new orphan drugs into the EU market, benefiting among others, NF patients.

The legislative revision, proposes to change the EU’s regulatory regime for approval of medicines through a proposal for a Directive targeting generic and biosimilar medicines and a proposal for a Regulation targeting pediatric and orphan medicinal products.

This revision intended to establish a competitive, innovation-driven and patient-centric regulatory framework that would contribute to bringing more drugs to patients across Europe. The COVID-19 pandemic demonstrated that when in need, collaboration and flexibility can lead to phenomenal achievements, when there is a common goal. Ultimately, the common goal of this legislative revision for the regulatory ecosystem should be clear: to deliver drugs to patients fast. However, we would like to express our concern that a reduction of incentives for pharmaceutical companies could hinder innovation and risk reducing the appetite for pharmaceutical companies to invest in rare diseases.

The Children’s Tumor Foundation (CTF) is fully aware of the challenges linked with drug discovery and development in rare diseases. Anticipating these challenges, CTF collaborated to develop a fully comprehensive preclinical and clinical platform that should significantly reduce cost and time of rare disease drug discovery and development.  CTF participated in an EU project over the last years, the EU Patient-cEntric clinicAl tRial pLatforms (EU-PEARL), funded by the private public partnership, the Innovative Medicines Initiative (IMI). The project, just came to its conclusion this week, coinciding with the publication of the EU Pharmaceutical legislative revision. EU-PEARL is conceptualizing the design of clinical platform trials in 4 diseases areas, enabling patient-centric drug development in Europe, with NF set to become a prototype for rare diseases.

While the publication of the proposed legislation is only the first step in the lengthy EU decision making process, CTF wishes for constructive negotiations that will contribute towards faster access of drugs to clinical trials, ultimately leading to finding cures faster for patients across Europe.

The Chair of the Board of CTF Europe, Annette Bakker, highlighted “Investing in accelerating the drug development should be one of the primary goals for the EU policy makers. We hope the European Parliament and the Council of the EU can enter the negotiations with a constructive mindset, without neglecting the crucial role patients can have, not only in the drug development regulatory lifecycle, but also including them in the definition of novel concepts introduced in these proposals, such as the High Unmet Medical Need and exceptional therapeutic advancement. Striking the right balance on such concepts will be essential to ensure an inclusive and patient-centric approach that will leave no one behind, especially in the rare diseases field.