BfArM head on plans for research data infrastructure, real world data and more

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During his time at the Federal Institute for Drugs and Medical Devices (Bundesinstitut für Arzneimittel und Medizinprodukte, BfArM), Prof. Karl Broich learned that drugs or medical devices are usually tested under laboratory conditions "without knowing whether it is the right drug for the right patient in the right dose at the right time. We don't have this data. We need to ensure that the data comes from practice," said BfArM President Karl Broich at the Hasso Plattner Institute's Digital Health Innovation Forum. He hopes for more data, for which the legal basis has been created in recent years with laws such as the Health Data Utilization Act.

The expansion of the cancer registry is already "very good". Interoperability is also a priority for digital health applications. The data could all be used in the BfArM's Health Data Lab, which is designed to develop approaches for using and analyzing data in the healthcare sector. "This can support the research and development of new therapies, improve the safety and efficacy of medicines and medical devices and contribute to the overall improvement of healthcare," says Broich. In addition to data analysis, which could be important for regulatory decisions, it is also about improving data quality. In addition, the data should be used securely and in compliance with data protection regulations and only passed on if it is possible to draw conclusions about individuals. In the past, there had been criticism because the billing data of all those with statutory health insurance was only available in pseudonymized form.

Broich was proud of the European Health Data Space that recently came into force: "The Health Data Lab team has been working intensively on pilot procedures to participate, and so we are fully functional in the European Health Data Space and can use this data together with others – for the next steps," Broich said confidently. In the beginning, the BfArM was a "normal government agency that focused on improving the efficacy and safety of medicinal products and medical devices". This has now changed and the BfArM is even involved in the classification of terminology systems – for DiGA and the establishment of the Health Data Lab. This means that the BfArM is now "at the center of the data-driven regulatory authorities".

Together with the European Medicines Agency, the EMA, Broich is working on the DARWIN initiative (Data Analysis and Real-World Interrogation Network). "There, we use real-world data for secondary purposes to clarify regulatory issues in particular, in addition to what we already know from the usual randomized controlled trials," says Broich. Through DARWIN, "very important information has already been obtained".

So far, it has been difficult to obtain secondary data. Germany has fallen behind with developments. "The Scandinavian countries were much faster than Germany, but we are catching up [...] We are now collecting secondary data from various institutions, hospitals, digital health applications, randomized controlled trials, and all this information is being brought together. And the FAIR approach (Findable, Accessible, Interoperable, Reusable) is a good description of what we have in mind here," says Broich.

Using data silos

According to Broich, there are data silos everywhere, but it is not easy to bring all the players together. In order to break down data silos in the future and enable EU-wide health data research, the BfArM is currently setting up a Data access and coordination office (DACO). "Our national legal basis for this is the new Health Data Utilization Act (GDNG). The DACO will play a key role in setting up the EHDS structures for the secondary use of health data (HealthData@EU) in Germany," explains the BfArM on request. The BfArM is receiving support from the EU-funded pilot project "HDP4Germany", the aim of which is to develop a national access point – a "Health Data Access Body" (HDAB) – for health data. "This access point will be set up as a DACO at the BfArM. In this way, national requirements and European developments will interlock," explains the BfArM. Researchers can then submit applications and the purpose will determine access to the data.

Electronic patient file mandatory by the end of 2025

The data of the 74 million people with statutory health insurance already exists with the health insurance companies, for example prescription data. The electronic patient file was launched a few months ago and will be mandatory by the end of the year and all this data will be available. This also emerges from a leaked document on the coalition negotiations: "We will roll out the electronic patient file in stages before 2025, from a nationwide test phase to mandatory use subject to sanctions".

Data from around 400 medical registers will be added to the data at the FDZ Gesundheit. According to the BfArM, the DACO will also enable researchers to analyze data from the FDZ Gesundheit [...] in so-called "Secure Processing Environments". According to the BfArM, the HDP4Germany pilot project is running for this purpose. "In addition to HDP4Germany, DACO is also involved in other EU initiatives, including TEHDAS2 – a project to develop the so-called 'Implementing Acts' for the EHDS – and the Community of Practice (CoP). The Community of Practice is a network of all European projects that are setting up a national health data access point (HDAB). The DACO is active in all working groups of the CoP and is thus in regular contact with the various stakeholders in the other EU member states," the BfArM continues.

"World first" DiGA

"It was really something new in the world that digital health applications could be prescribed and then reimbursed," explained Broich about the DiGA. At the beginning, there were many questions and concerns, especially from the payers, "but it's a success story". Last year, there was a sharp increase in prescriptions. There are now 1 million prescriptions for DiGA. The majority of these are DiGAs to support therapy for mental illnesses such as depression and anxiety disorders. A further high number of DiGAs are prescribed to support obesity. According to Broich, DiGAs are particularly relevant for the self-empowerment of patients.

"IT freaks" help with penetration tests

In the future, patients will be able to release the data from the DiGA to the Health Research Data Center for research purposes. A script will then be used to perform an analysis on the original data. "We check whether identification is possible or not. And then we look at the results and go back to the researcher [...] We are in the final stages of setting up and hope to be more active with the Health Data Lab by the end of May or early summer. Many people are already waiting to access this data. The balancing act between security and user-friendliness is difficult," says Broich. The BfArM is therefore working on penetration tests with the help of a few "IT freaks" to ensure data security. In the past, a lawsuit against the mass storage of health data was put on hold, as the security concept is still lacking at the FDZ Gesundheit.

(mack)