The competition surrounding clinical trials has been increasing for years, more and more trials are being conducted outside of the EU – should we be worried?

It is not unusual for clinical trials to be oriented internationally. Medicinal products are marketed worldwide, so studies should also reflect gender and ethnic diversity. On the other hand, pharmaceutical companies certainly also choose study sites based on strategic and financial considerations. Clinical trials are also increasingly being conducted outside Europe or the USA in order to keep costs low. We have been following this development very closely for years, both with regard to ethical as well as scientific standards. Study results generated in Asia, for instance, cannot always be applied easily to Europe. Therefore, it must be in all our interests to strengthen the EU as a location for clinical trials.
Basically, it's all about not being left behind. Medical progress is developing at a rapid pace. The pandemic has forcefully demonstrated how important rapid access to medical innovations is; clinical trials can provide this to patients. The medical employees in the study centres also benefit, because they can familiarise themselves with new therapies. This is a potential that we must - and want to - use.

Aside from financial aspects, the bureaucratic effort is also a reason given by applicants for deciding against conducting a clinical trial in Europe.

It is understandable that, e.g., having to submit applications for multinational clinical trials separately to the national authorities and Ethics Committees in each country, might have a deterrent effect. The goal is to strengthen and further expand the EU as a hub for clinical research. This includes simplifying and speeding up the applicants' navigation through the approval process - a tangible step to further promote the development of high-quality medicines. At the same time, we also offer and will continue to expand numerous different types of support, in order to assist applicants and sponsors during the process, e.g., checklists, training opportunities or a variety of advisory formats.

How can this be achieved while at the same time maintaining the high standards of the clinical trials?

These issues are addressed in the new Clinical Trials Regulation which came into force in February of 2022 and which harmonises the procedure within the EU by way of uniform legal requirements. The core of this regulation is the new Clinical Trials Information System "CTIS". Over the next three years, this system will gradually be expanded to become the one-stop shop for companies wishing to conduct clinical trials in the EU. With one single submission, they can apply for authorisation within the EU Member States or EEA countries. Licensing and regulatory authorities can also use the system to contact sponsors in an uncomplicated manner and for direct cooperation with other authorities and exchange of information. The high standards that apply to the conduct of clinical trials e.g., where the protection of the participants or the reliability of data are concerned, remain unaffected.

A new EU Regulation and the Clinical Trials Information System: are these measures enough to lastingly strengthen the European environment for clinical trials?

The changes begun now are just the first building blocks. Within the network of European health authorities, the BfArM is working towards a fundamental transformation of the clinical research environment. In the long term, the goal is to find new ways of initiating, designing and conducting clinical trials. For this purpose, the initiative "Accelerating Clinical Trials in the EU", or "ACT EU" for short, was launched almost at the same time that the new EU Regulation came into force. European licensing authorities, the European Medicines Agency EMA, as well as the EU Commission work together jointly in this initiative towards achieving relevant innovations.

What exactly is supposed to change due to the initiative?

The initiative aims at breaking down silo mentality and jointly promoting medical innovation. While the EU Regulation is about harmonising the submission, assessment and monitoring of clinical trials in the EU, ACT EU aims at ensuring that this coordinated approach is also applied to innovations in clinical trials.
Among other things, we have published a strategy paper describing ten priority actions for 2022/2023. These include, e.g., coordinated scientific advice on the approval of clinical trials or the establishment of a multi-stakeholder platform.

Another important point is the issue of transparency. Time and again, it has been criticised that study results are not published in full, thus keeping the public in the dark with regard to negative results.

It is undisputable that clinical trials are a driving force crucial for medical innovation and advances in patient care and disease prevention. Therefore, control and transparency are indispensable for the protection of public health and the promotion of innovation in the field of medical research. Broad access to trial results is an essential factor enabling science, the medical profession and policy-makers to arrive at well-founded decisions for healthcare and medical research.

Inadequate reporting in general and selective reporting on clinical trials with favourable outcomes can result in potentially avoidable redundancies in conducting clinical trials, thus compromising the economic and scientific efficiency of clinical research. Also, unpublished clinical trials with unfavourable outcomes can have a negative impact on public health - this must be avoided at all costs.

How does the BfArM ensure more transparency in this field?

We regularly request more transparency and a stronger commitment when results are submitted. The European Medicines Agency (EMA), together with the European Commission and national authorities such as the BfArM, requires sponsors of clinical trials to enter their results in the European database. The BfArM, for example, directly contacts sponsors who have not uploaded their final result reports to EudraCT within one year after the end of the trial with considerable follow-up actions.
These are accompanied by our cooperation with Ethics Committees, the associations of the pharmaceutical industry and representatives of medical faculties also prompting sponsors to upload the reports to EudraCT in due time. We use our networks, e.g., with academic sponsors of clinical trials, to raise awareness with regard to this important issue and to highlight obligations, precisely such as the publishing of final result reports.
And with the new Clinical Trials Information System "CTIS", we are consistently continuing this path with a new, comprehensive IT system to make even more results reports accessible even faster in the future.

Prof. Karl Broich

Prof. Broich is a physician (neurology, psychiatry, cognitive behavioural therapy) and has been President of the Federal Institute for Drugs and Medical Devices (BfArM) in Bonn since 2014.
His current activities in the European network of regulatory authorities are Member of the Management Board of the European Medicines Agency (EMA MB), Chair of the Network Portfolio Advisory Group (NPAG). He is a member of the Heads of Medicines Agencies (HMA) Management Group and is currently its Chair. He is also co-chair of the EMA's Darwin EU Advisory Board.
His scientific focus is on clinical psychopharmacology, imaging of neurodegenerative diseases and other potential biomarkers and dementia, and clinical trial methodology, among others. Prof. Karl Broich is author and co-author of more than 220 papers (original scientific papers, reviews, book contributions).