Event summary

Handling Innovation in nanomedicines: regulatory changes needed to realise new treatment opportunities and ensure patient safety

Science and technology have never moved as fast as now. With personalised medicines and the advent of gene therapy the standard rule book is clearly coming under pressure. And it would appear that the same is true for the technology behind nanomedicines.

Nanomedicines are medicinal products that have a size in the nanoscale range. They have unique properties which allow them to distribute in the body in a way that were previously impossible. This is one of the reasons why nanomedicines have been gaining increasing attention in academia and research, as they have the potential to open up new and improved therapeutic opportunities.

A breakfast meeting in the European Parliament co-hosted by the European Alliance for Access to Safe Medicines (EAASM) and MEP Jose Inacio Faria (PT, EPP) took place on 20 March to discuss the appropriateness of the current regulatory approval process for nanomedicines and their follow-on products, also known as nanosimilars, and analyse the potential implications for policymakers, regulators, physicians, pharmacists, healthcare workers and ultimately patients.

Nanomedicines are highly complex products. The complexity and number of this new class of medicines is even increasing at a rapid pace and so are the questions around assessing their quality, biological properties and therapeutic profiles. Such assessments are exacerbated in the absence of a legal definition on nanomedicines.

Harmonisation of terminology was one of the issues raised during the debate. We all need to use the same type of words. At the moment, the definition of a nanomedicine keeps changing because the technology itself is in constant evolution, as highlighted Prof. Dr. Stefaan C. De Smedt, Pro-dean of the faculty of Pharmaceutical Sciences and Director of Ghent Research Group on Nanomedicines.

Current regulatory approvals for nanomedicines and follow on products (also referred to as nanosimilars) are decided on an ad-hoc basis by product category. With the accelerating progress in the development of clinically significant nanosimilars and in in the absence of a specific regulatory pathway for them, the EAASM believes that it is the right time to set the scene for building a consensus so that this regulatory weakness can be addressed. And to make medicinal products of high-quality and safety.

The European Commission does not intend to update existing regulations. They stress that nanomedicines and nanosimilars should be treated as any other medicinal product i.e. in accordance with the EU pharmaceutical legislation. They quote that there are several new medicines approved in the EU. These new medicines benefit from new modes of action and innovative drug delivery properties that were only made possible by the use of nanotechnology. As recently stated in a response from Commissioner Andriukaitis to a Written Question posed by MEP Faria (EPP, PT), efforts are however being made to better understand their specific properties and to continuously improve the available scientific guidance. For example, the European Medicines Agency has published specific guidance for nanomedicines. The EAASM as previously stated given the growing weight of evidence and the rapidly developing clinical interest that the regulatory framework should be urgently reviewed.

In the presentation by Susanne Bremer Hoffmann of the Joint Research Centre -European Commission, it was underlined that a more proactive pharmacovigilance system with a rapid reporting system to act on adverse events was needed. And so new tools need to be developed to identify and monitor weak signals indicating safety concerns in scientific literature, real world databases. “We should think how we can best obtain the information for regulatory decision making by using the whole portfolio of new analytical methods and in vitro technologies” she stated. This is why the identification, development and standardisation of test methods will be necessary for quality, safety and efficacy assessments of nanomedicines and nanosimilars.

Another important element brought up is the fact that nanomedicines and nanosimilars can be approved through a decentralised procedure in the EU. As argued by Jon de Vlieger, Coordinator of the Working Group on Non-Biological Complex Drugs (NBCD) at Lygature, the centralised procedure has the potential to ensure better coordination and understanding across Europe. He emphasised that Europe has successfully pioneered and implemented a solid regulatory framework for biosimilars that ideally should serve as model for the approval of nanosimilars. Such framework avoids potential confusion and ambiguity of having a different policy or interpretation per country. Nanomedicines are used to treat a variety of serious medical conditions including cancer, auto-immune diseases, infectious diseases, anaemia, and more. Through a centralized procedure, the collective intelligence and experience of European Regulators will best serve the European citizens, he stated. Translating science into health care practice is a difficult task; appropriate science-based approval and post approval standards need to be globally introduced for nanomedicines and nanosimilars to ensure patient safety and benefit to all.

As emphasised during the debate, nanotechnology in health can offer opportunities to address unmet medical needs and hence regulatory changes are needed to realise new treatment opportunities that will help ensure patient safety and wellbeing. This is the reason why training a new generation of experts through working groups is vital to tackle the lack of consistent standards, address side effects issues and develop relevant analytical methods for pharmacovigilance.

The paramount issue of patient safety was emphasised by Mike Isles, EAASM Director. He stated that “…clearly nanomedicines and nanosimilars are complex and patients must not be put in harm’s way from products that are copies of the original without absolute scientific assurance that their therapeutic profile has been tested as rigorously as the originator’s. It is somewhat alarming to read in the scientific press that this may not always be the case. Patient safety must always be the most important criteria when assessing the granting of a new product licence. That is why we need to create a robust and totally fit for purpose regulatory framework which is clearly needed in this new, exciting and developing field of medicine”.

In the absence of a specific regulatory pathway and a legal definition, more scientific, policy and practice knowledge on the quality, safety, and efficacy of nanomedicines and nanosimilars must be gained and there is a need to build a consensus dialogue as well as alignment between all players in Europe and beyond to further explore the field of nanomedicines at EU and national level. Awareness as well as a well-controlled robust manufacturing process are indeed fundamental to ensure quality, safety and efficacy.