Please note: this column has been translated from Dutch to English

Innovative medicines are emerging in various fields, including new indications for existing drugs and more complex or personalized treatments. These developments occur within industries, startups, or academic environments. Medicine authorities, like the ‘College ter Beoordeling van Geneesmiddelen’ (CBG) in the Netherlands, must keep pace with these innovations. Regulatory frameworks for approving new medicines and processes for monitoring efficacy and side effects need to be geared towards innovation. This requires mediators between disciplines.

At CBG, we have been engaged in scientific research for years to improve regulatory processes. We focus on system improvements (including optimization and deregulation) and innovations that add value to the role and tasks of medicine authorities (renewal). Our goal is to ensure the availability and accessibility of medicines for patients by adapting regulatory guidelines and processes. This is possible due to increasing research (including collaboration with FAST – Centre for Future Affordable Sustainable Therapy Development) published after peer-review. This “regulatory science” research has an impact, ultimately benefiting the patient.

Strengthening two worlds
At CBG, we are making great strides in this. And, of course, we don’t do this alone. We actively seek collaboration, particularly with various universities, such as Utrecht University (UU). This collaboration was confirmed last November. Since the first of November in 2023, I have been connected to UU as an Associate Professor of Drug Regulatory Science in the Division of Pharmacoepidemiology and Clinical Pharmacology, in addition to being the head of the Science Department at CBG. Through these dual roles, I monitor (inter)national developments in medicine evaluations and the necessary research to innovate this process, translating scientific findings into regulatory practice.

Rare diseases are a common focus in my work. This interest began with my doctoral research and its continuation, where I focused on the hereditary skin disease epidermolysis bullosa. Today, my broader focus includes stimulating therapy development for rare diseases. Although rare, many people worldwide are affected by rare diseases, many of which lack treatments.

Advancing drug repurposing
If there are no treatments, drug repurposing might be an alternative: using existing drugs for other diseases. On this topic, we are pulling together with FAST to exploit this arsenal of possible treatments. Together with Anneliene Jonker (University of Twente, Duchenne Parent Project Netherlands), I chaired a task force of the International Rare Diseases Research Consortium (IRDiRC) on this topic. In 2017, the consortium set a goal to have 1,000 new treatments for rare diseases by 2027. For academic groups and small and medium-sized enterprises (SMEs), we developed the Drug Repurposing Guidebook to help developers, including a useful checklist. CBG has also been involved for several years in a ZonMw call for drug rediscovery projects, providing advice.

Utilizing available data
For the past two years, I have been working on establishing the European Medicines Regulatory Database (EMRD) with Lourens Bloem, Jarno Hoekman, Stefan Verweij, and Vincent Haverhoek. EMRD is a dashboard containing a large set of information about medicines registered via the European Medicines Agency (EMA). The dashboard explains the various characteristics of each medicine, with options to download, visualize, or analyze selected data. Information that was previously fragmented is now more easily accessible, providing better insights into timelines and types of medicines. This makes the information more available to researchers, patients, and other stakeholders. EMRD is expected to be available online for free later in 2024.

In the further development of EMRD, we want to focus on regulatory data around rare diseases. We are investigating which classification is most suitable for unlocking orphan designation and indications, for example. We also aim to enable real-time tracking of how many new medicines for rare diseases emerge over time and for which conditions. This will allow us to assess if the previously mentioned IRDiRC goal of 1,000 new treatments for rare diseases has been achieved, or if additional actions are needed.

Importance of research and education
In my doctoral research topic, epidermolysis bullosa, there is significant variability in the use of outcome measures in clinical studies. Greater harmonization is crucial for developing potential medicines for these rare, hereditary skin diseases because results need to be comparable. With the international initiative COSEB (Core Outcome Sets for Epidermolysis Bullosa), we aim to harmonize these outcomes in collaboration with the COMET initiative, allowing experiences and expertise to be shared between similar initiatives for other diseases. Learning from each other is crucial.

We do more than research. Consider STARS: a European project to strengthen regulatory knowledge among academic researchers and thus advance drug innovation. We collaborate with FAST to implement the STARS recommendations, also providing scientific advice to academic researchers or small companies.

Bringing perspectives together
A common theme in these examples, and all other scientific initiatives, is bringing together different perspectives from various fields: industry, authorities, patient representatives, and academia. In the Netherlands, this is done through the Regulatory Science Network Netherlands (RSNN), where CBG, CCMO, UMCG, and UU participate alongside patient organizations and companies. This gives the Netherlands a model role in Europe. Europe is watching RSNN with great interest, and there are calls for a similar European network.

In summary: how do we ensure that new innovative medicines reach patients faster? By innovating in all areas, including regulation. In my dual role at CBG and UU, I aim to promote knowledge exchange and sharing, bridging regulators and academia. Different perspectives from regulators, industry, patient representatives, and academia are essential!

See you soon,

Marjon Pasmooij