Brussels, January 2022
Event summary of the educational workshop on “Nanomedicines in the EU: innovative therapies and regulatory needs” held on 7 th December 2022 16.00-17.30 CET
The European Alliance for Access to Safe Medicines (EAASM) along with the members of the Nanomedicine Regulatory Coalition organised an educational workshop (held virtually) on “Nanomedicines in the EU: innovative therapies and regulatory needs”, bringing together high-level speakers from the Food and Drug Administration (FDA), National Competent Authorities, patient representatives, academia and other experts.
The objective was to discuss the critical issues of the regulatory framework for nanomedicines, from adopting a definition of a nanomedicine that is harmonised at EU level, to developing protocols and common guidelines for the characterisation, evaluation and control of the nanomedicine production process. Moreover, the event provided an opportunity to discuss the results of a survey designed to gather knowledge and views of the National Competent Authorities for the regulation of medicines and medical devices for human use on nanomedicines and non-biological complex drugs.
Mike Isles, Executive Director at EAASM, kicked off the discussion by highlighting the support and attention this issue has received from European institutions, in particular from the European Parliament. In its Own-Initiative Report of 24 November 2021 on the Pharmaceutical Strategy for Europe, it expressly indicated the need for a centralised framework for the approval of nanomedicines and nanosimilars (off-patent follow-on products). The workshop’s host added that the EU has the opportunity to exploit the potential of these technologies to address unmet medical needs and enable the development of a competitive and innovative European industry. The EU should not miss the political impetus represented by the upcoming Revision of the EU General Pharmaceutical Legislation, he pointed out.
Jon de Vlieger, Coordinator of the Working Group on non-biological complex drugs and Strategy Director at Foundation Lygature (a Dutch-based not-for-profit organisation) presented the regulatory challenges for non-biological complex drugs. He stated that the rise of bio- and nanotechnologies has accelerated the development of complex medicines which in turn led to the establishment of guidelines by regulatory agencies around the globe. The current regulatory approaches for the approval of this type of product in Europe is highly variable and heterogeneous as many companies apply for marketing authorisation using mostly the decentralised procedure route. In such cases, a drug not yet authorised in the EU, can be simultaneously authorised in several EU Member States but pharmacovigilance is not centrally organized. Approval through the centralised procedure would benefit European citizens as it arranges centralised safety monitoring and ensures that product information will be made available in all European languages at the same time. For a more concerted advance in this field an alignment of science-based regulatory views worldwide is needed. “We need all stakeholders to be engaged in discussions and scientific findings to be published and quickly made available to the public”, Jon de Vlieger remarked.
Prof. Paola Minghetti from the University of Milan (Italy) continued by emphasising the risk of different data requirements resulting from the choice left to the applicant between centralised, mutual recognition and decentralised procedures for marketing authorisation. Given that small differences in the manufacturing process of the nanomedicine product may have a strong impact on its quality profile, she recalled the importance of quality assessment. Moreover, she criticised that in the EU, several copies of nanomedicine products have been authorised via the generic pathway. Prof. Minghetti concluded by echoing the suitability of the centralised procedure for nanomedicines and nanosimilars.
The debate continued with the presentation by the US FDA Liaison Officer to the European Medicines Agency (EMA), Katherine Tyner. She touched upon the regulatory differences and similarities between FDA and EMA, pointing out that contrary to EMA clinical trials are regulated by FDA. Similar to Europe, the United States lacks a regulatory definition of nanotechnology or related terms. Tyner further added that early discussions on novel formulations between innovators and agencies, together with regulatory research are essential for marketing these products. Developing standards guarantees consistency, predictability and credibility.
Prof. Anthony Serracino-Inglott, Chief Executive Officer at Malta Medicines Authority, led us through the lessons learnt from the introduction of biosimilars. In this case, there was a disconnect between the healthcare professionals and the patients using them. In the development of a nano-medicinal product, in addition to quality, safety and efficacy, Prof. Serracino-Inglott emphasised accessibility and environmental aspects. When defining nano-medicinal products, he called for not being too exclusive. Linked to previous presentations, he pointed out the innovative features of these medicines of significant benefits to patients. “The time has come for the Union to decide whether all nanomedicines should be centrally authorised in the EU in order to gain marketing authorisation”, said Prof. Serracino-Inglott.
Closing the session, Mike Isles presented the results of the survey that the Nanomedicines Regulatory Coalition submitted to the National Competent Authorities of the Member States as part of its outreach campaign to raise awareness around nano-medicinal products. Among the most important findings, was an indication that there were different levels of expertise among the Member States. Some national competent authorities reported a lack of expertise to answer the questionnaire. Importantly there is a consensus for the 10.3 hybrid process pathway for the approval of follow-ons nanomedicines. Furthermore, it was widely agreed that there was a need for a legal definition of nanomedicines at European level, along with additional EMA guidance with harmonised criteria for assessing such products.
Overall, the experts agreed on the importance of avoiding duplication and enhancing innovation – we should not be prohibitive in the development of these medicines. The centrality of patients has been a common thread in the keynote speeches.
In line with these considerations, the Nanomedicines Regulatory Coalition calls on European and national health authorities to commit to: