Proposed revisions to EU pharma strategy draw mixed reactions
On 26 April 2023, the European Commission proposed a massive overhaul of EU pharmaceutical legislation, including the long-awaited revisions to the Regulations on Orphan Medicinal Products (OMP) and Paediatric Medicines. These updates provide an opportunity to significantly change the lives of Europeans living with a rare disease, by improving drug development processes and access to treatment.
A main focus of the new legislation is on positioning Europe as a competitive market for innovation in medicines development, through the introduction of new incentives and by simplifying regulatory frameworks. This includes enhanced support for developers, tailoring regulatory protections to individual drugs, and streamlining evaluation and authorisation processes. Additionally, measures are being taken to improve the accessibility and affordability of innovative medicines, such as by making generics and biosimilars available earlier.
Members of the rare disease community have been calling for a revision to the OMP Regulation in particular for some time now. Such legislative improvements were in fact one of the key recommendations from the Rare 2030 study, which was led by EURORDIS and published in 2021. EURORDIS in particular has been active in advocating for an update which meaningfully addresses the needs of people living with a rare disease.
Yann Le Cam, CEO of EURORDIS, praised features such as modulated incentives and the inclusion of patient representatives in the Committee for Human Medicinal Products but noted that further amendments are needed to ensure no EU citizens are left behind. Commenting on the proposal, he stated: “Policymakers must also realise the opportunity being presented to them to place the EU on an ambitious and more competitive footing with the US and other regions when it comes to boosting the development and accessibility of rare diseases medicines.”
Other groups have had similarly mixed reactions. The European Patients Forum (EPF) called in particular for a strengthening of the meaningful engagement of patients throughout the regulatory lifecycle of medicines, while the European Public Health Alliance (EPHA) emphasised the importance of the new modulated incentives.
At the same time, the proposal has drawn significant criticism from the pharmaceutical industry. The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) and the European Federation of Pharmaceutical Industries and Associations (EFPIA) have both raised concerns that the changes to incentives will undermine intellectual property and hinder, rather than facilitate, innovation without improving access to existing medicines.
Despite the criticisms the new revisions have attracted, they are sure to fundamentally change the European rare disease landscape as they begin to be implemented. Only time will tell the extent of this change, and whether it brings about real benefit for people living with a rare disease.
