EU Parliament adopts position on pharmaceutical reforms at plenary session
On 10 April 2024, the European Parliament voted to adopt its position on the proposed reforms to the EU pharmaceutical legislation at their plenary session in Strasbourg, France. The aim of these reforms is to continue to foster European innovation while enhancing the security of supply, accessibility, and affordability of medicines. The vote followed new amendments introduced by the Committee on the Environment, Public Health and Food Safety (ENVI) last month.
While the new pharmaceutical package has implications for the development, approval and marketing of all human medicines, it is of particular interest to the rare disease community as it includes long-awaited revisions to the Regulations on Orphan Medicinal Products (OMP) and Paediatric Medicines. It includes proposals such as a modulated market exclusivity framework for orphan drugs, enhancements to the PRIME scheme, and mandated patient participation in EMA consultations. The ENVI compromise also redefines the definition of the “significant benefit” OMPs must demonstrate, as well as a clearer idea of what constitutes “high unmet medical needs.”
These changes, among others, are a promising step forward in building sustainable access to orphan drugs at the European level. EURORDIS, in particular, praised the Parliament’s decision to centre patient experience while continuing to incentivise OMP development. Responding to the results of the plenary vote, Valentina Bottarelli, Public Affairs Director at EURORDIS, said:
“These changes…represent not just progress but the result of our community’s advocacy to close the gap between fast-paced scientific discoveries and patient care…Importantly, this endorsement builds on an already strong and broad political consensus…on the urgent need for [a European Action Plan for Rare Diseases].”
While industry stakeholders such as the European Federation of Pharmaceutical Industries and Associations (EFPIA) also praised the move towards a more sustainable regulatory framework, they also raised concerns that measures such as reductions to regulatory data protections will hinder innovation. In response, they are calling for subsequent legislation to consider the importance of Europe as a competitive market for drug development.
Overall, the results of the vote are an encouraging sign of the growing commitment at the EU level to a European policy framework for rare diseases. Moving forward, it is important that the Council builds upon this momentum by further refining the legislation as it pertains to orphan drugs. Crucially, Member States must also acknowledge and build upon the Parliament’s recommendations so that when the time comes for implementation, no Europeans living with a rare disease are left behind.