Regulatory expertise on the one hand, and academic, clinical expertise on the other: if this expertise is brought together into a licensing process for new drugs, it will benefit people suffering from diseases. In a pilot project, scientists in clinical oncology now have the opportunity to get involved in drug evaluation. The BfArM is participating as a driving force at many points along the way.
If someone wants to get a drug licensed in the EU, they have to make an application to the European Medicines Agency (EMA). The documents are then scientifically evaluated there in the Committee for Medicinal Products for Human Use (CHMP). The committee brings together around 60 experts. They largely come from the national competent authorities, which are connected via the network of Heads of Medicines Agencies (HMA).
The Member States work through the assessment of the application in various groups to prepare their decisions. To do this, data and documents from the applicant are screened in the national licensing bodies and carefully evaluated. Recommendations for or against the licensing of a drug are then fleshed out and ultimately presented to the committee members for discussion. Finally, the official recommendation of CHMP is given, while the formal license is issued by the EU Commission.
Particular challenges
The Chair of CHMP is PD Dr. Harald Enzmann, head of the “EU and International” office at the BfArM. He has been a member of the multinational committee for 15 years and knows all about the meaning of scientific expertise in the assessment of licensing applications. “Experts with specialist knowledge or clinical experience are often consulted during assessment to enrich the scientific discussion,” reports Enzmann. “The scope ranges from specific questions regarding potential applications in clinical practice to the latest trial.”
This process has particular challenges. One example is oncological disease patterns in which access to innovative drugs improves the chances of a cure or can prolong life. This also applies to giving patients speedy access to these treatments. For this, knowledge of new scientific approaches as they exist in academic-clinical research is important. The same also applies for the anomalies in the provision of specific diseases. “This knowledge should flow into our application assessments,” says the CHMP chair.
STARS
Linking the regulatory work with clinical and scientific research is an important matter for the players at national and European level. The EU financed initiative “STARS – Strengthening Training of Academia in Regulatory Science” was thus launched. It aimed to strengthen regulatory knowledge in the academic research environment and make the clinical research more usable for the healthcare systems. The BfArM took part in this as one of the coordinating project partners. National authorities from a total of 18 European countries worked together on this project. They are now working on the implementation of strategies and experience from STARS.
The BfArM is playing an important role in several places at once, with the aim of getting these kinds of initiatives and projects off the ground across Europe. The BfArM president, Prof. Dr. Karl Broich, recognises the topic from the point of view of the BfArM, as the largest national approval body in the EU, and also in his role as chair of the HMA: “New scientific approaches for innovative medicines are frequently conceived in academic-clinical research,” explains Prof. Broich. “As the HMA, we see a demand in many EU member states for promoting dialogue between academic-clinical research and national medicines authorities. It involves speaking together about regulatory aspects and, at the same time, closing any gaps in information in the development of medicines.” Together with the EMA, we are searching for pathways and potentials which can be exploited. This resulted in the idea of systematically approaching scientists in clinical oncology from across the EU and to bring them on board for this cooperation.
No other area is displaying this many new modes of action and active substances as do oncological indications.
PD Dr. Harald Enzmannm, Chair of CHMP
Pilot project oncology
Against this backdrop, the HMA and EMA therefore started a pilot project in May of 2023, which is aimed at oncologists. It will be introduced over a time scale of twelve months. The focus lies on scientific mentoring and assessment of the licensing of human medicines. The regulatory competence is to be reinforced by academic researchers, among other things by way of targeted training programmes. A curriculum was developed for this which, among other things, systematically addressed regulatory principles.
This approach is particularly important to the CHMP chair, who himself acts as a contributor on several topics. “The pilot project provides us with very good opportunities. We can now already say that the trend among the central licensing applications being submitted to the EMA in the coming years will very clearly be in oncological areas of indication,” Enzmann stresses. “No other area is displaying this many new modes of action and active substances.”
It‘s a win-win situation for all involved: “As regulators, we are conveying to the experts the basic principles relevant to oncological specialist fields. In so doing, we are giving them the basis so that they are able to bring their knowledge into the assessment of medicines in a targeted way.” Principally, the HMA and EMA are assigned to have a close working relationship with academics and researchers: “In the licensing of medicines, we need to be prepared for future challenges and opportunities which arise from the advances in science and technology,” stresses Enzmann. Especially when it comes to innovative treatments, which should be made available to patients quickly.”
PD Dr. Harald Enzmann
The medical doctor who gained a doctorate and achieved professor status has been a member of the committee for human medicines (CHMP) for the European Medicines Agency since 2005. In 2016, he was elected deputy chair of the CHMP. Since 2018, he has been chair of the board and in September 2021 was unanimously re-elected for a second term. He has been working at the BfArM since 2002. Here he headed, amongst others, the “medicines licensing” department. Since 2016, he has been the head of the “EU and international” office. Enzmann is a Fellow of the International Academy of Toxicologic Pathology, Member of the European Society of Toxicologic Pathology, the Society of Toxicology and the European Association for Cancer Research.
Heads of Medicines Agencies (HMA)
The Heads of Medicines Agencies (HMA) is a network of the heads of the national competent authorities for human and veterinary medicines in the European economic area. The HMA works in the network for European medicines licensing with the European Medicines Agency and the European Commission. It is a one-of-a-kind model for cooperation and work sharing in regulatory matters, at both statutory and voluntary levels.
The HMA is coordinated and headed by the Management Group and supported by various working groups – responsible for various areas – and the permanent secretariat. The president of the BfArM, Prof. Karl Broich, is a member and current chair of the HMA Management Group. Furthermore, numerous experts from the BfArM work in the HMA working groups.
