The EU Innovation Network of regulators has released a Horizon Scanning Report on Nanomedicines, highlighting key trends, challenges and recommendations for regulatory collaboration. It emphasises the growing applications of nanotechnology in medicine and the need for improved regulatory frameworks. A new platform for regulatory science research is set to launch in 2025, aimed at enhancing dialogue between researchers and regulators. This call for regulatory preparedness aligns with similar recommendations and initiatives in the field of nanomaterials.

Release of horizon scanning report on nanomedicines

On February 3, 2025, the EU Innovation Network (EU-IN) for medicinal products released a report on Nanotechnology-based medicinal products for human use. The European Medicines Agency (EMA) and national regulatory agencies started the EU-IN to strengthen collaboration on innovative medicines and associated technologies and support medicine developers. The report highlights the current status and trends in nanomedicine, addressing challenges, opportunities and regulatory aspects. Important recommendations include early and continuous engagement between European regulators, academia, industry researchers and regulatory science researchers.

Trends in nanomedicines: expanding applications and complex formulations

The report states that most approved nanotechnology medicines are based on optimised formulations of approved active substances. However, it notes that lipid nanoparticles (LNPs) have emerged as an essential technology for effective delivery of exogenous mRNA, a topic we covered in our previous newsletter. Furthermore, the range of clinical applications is expanding. While the primary focus remains on cancer treatments, including vaccines for prevention and therapy, there is also ongoing development for autoimmune diseases, infectious diseases and various genetic or rare illnesses. Lastly, the report mentions a shift from simpler nanoparticles to more complex and multi-component ones. These new complex nanoparticles are often designed to carry new active substances, instead of reusing those already approved.

Enhancing the regulatory framework for nanomedicines

The report summarises the current regulatory guidance for nanomedicines and available regulatory support structures. It highlights challenges such as classification of medicines versus medical devices and the need to standardise critical physical, chemical and biological criteria to assess quality, safety and efficacy properly. Several changes are suggested for the regulatory framework.

1. Implement a tracking system for nanotechnology-based medicines in clinical development by adding a tick box in the Clinical Trial Information System
2. Review and expand existing EU guidance to include marketing authorisations and clinical trials requirements.

Currently, developers face uncertainty regarding required evidence due to incomplete guidance, leading to inadequate evidence and regulatory issues regarding quality and safety. More collaboration among regulators, product developers and researchers is recommended to improve guidance.

European Platform for Regulatory Science Research

A new platform has been launched in 2025 to advance European regulatory science research. The platform will connect academic and nonprofit researchers with regulators across Europe and beyond to enhance collaboration and dialogue. The goal is to make research more efficient, have a greater impact, and speed up the application of findings in clinical practice. Unlike the EMA Innovation Task Force, which enables dialogue between regulators and applicants concerning a specific therapy in the applicant’s pipeline, the new platform will focus on understanding and aligning the needs of researchers and regulators more broadly.

Other European initiatives foster collaboration and knowledge sharing

In recent years, several European initiatives have focused on regulatory aspects of nanomedicine. One of these is the NANO Working Party of the European Pharmacopoeia Commission (previously Non-Biological Complex Drugs working party). This group includes experts from universities, research organisations, the industry and regulatory agencies. Internationally, regulators share knowledge and approaches in different jurisdictions through the nanomedicines working group of the International Pharmaceutical Regulators Program (IPRP). Other initiatives include the​​​​​​​ ETP Nanomedicine, a project led by industry to address the application of nanotechnology in healthcare. There is also a project funded by the European Commission aimed at improving understanding of regulations about nanomedicines and enhancing dialogue between academia and regulatory authorities. Strengthening Training of Academia on Regulatory Sciences (STARS).

Reflections by RIVM

The Horizon Scanning Report on Nanotechnology-based medicines highlights important developments and initiatives in the field of nanomedicines. It points out that while nanomedicine is advancing, moving from research to clinical usage is challenging due to the uncertainty of regulatory requirements. The REFINE project, which RIVM participated in, has identified these challenges and proposed solutions, such as encouraging early discussions between regulators and innovators and fostering cooperation among various sectors applying nanomaterials.

The European Network for Safety and Sustainability of Chemicals and Materials (NSC, former EU NanoSafetyCluster) has found similar issues for nanomaterials and supports early engagement between regulators and stakeholders. This concept of ‘regulatory preparedness’ is also on the agenda of the OECD Working Party on Manufactured Nanomaterials (OECD WPMN).

Efforts by the NSC and OECD WPMN align with the recommendations for nanomedicines. Improving collaboration and knowledge sharing between the fields of nanomedicines and nanomaterials can enhance innovation. The call for early engagement between researchers and regulators boosts ongoing initiatives such as the NANO WP, ETP Nanomedicine and the STARS project, further strengthening the European regulatory science network.