Using existing medicines to develop new therapies for patients with unmet treatment needs. This is known as drug repurposing. This way of making new and affordable treatments available has huge treatment potential for a variety of conditions, as the development track can be relatively shorter and cheaper in comparison with traditional innovative drugs. Modern technology enables rapid screening for possible new applications. Nevertheless, it turns out that the drug repurposing route to the patient still contains many bottlenecks. A proper business model is often lacking, and limited regulatory options often slow down the route from initial discovery to continued availability for patients.   The limited successful examples of drug repurposing show both the enormous untapped potential as well as the lack of a regular development route.

The Netherlands has a long tradition of professional patient representation and a well-organized network of patient organizations. At FAST, we further focus on embedding patient participation in therapy development, from the early research phase to inclusion in the insurance package. We do this by actively engaging in dialogue with patient representatives. The development of knowledge and skills among both patient representatives and their counterparts (for example, through education) is an important focus for this year.

Innovative method and technologies have the potential to speed-up therapy development and derisking the development process in an early stage. For example, through rapid screening methods, innovative data mining, and a better understanding of complex disease mechanisms, the number of substances and biological targets to be investigated have strongly increased. Innovative technologies, including human stem cell models like organoids and organs-on-a-chip, have the potential to evaluate the feasbility of therapy lead earlier in the development process, due to significantly enhanced predictive value. Innovative methodologies, including integrative mathematical modeling, trial methodology, real-world data and learning healthcare systems can speed-up clinical studies and implementation in the clinical practice. The field faces the challenge of implementation including further development, regulatory acceptance, often including qualification or validation, and adoption in therapy development practice of these enabling methods and technologies.

Therapy development is highly regulated. New therapies can only become accessible to patients, if data and results from (pre-)clinical trials must demonstrate that the therapy is of sufficient quality, has a favourable benefit-risk ratio for patients and is cost-effective. To ensure that new therapies reach patients efficiently, it is important that regulatory pathways and requirements are are flexibible and adaptive to enable the implementation of new methods and the evaluation of new therapies based on innovative technologies and state-of-the-art science.

Rare diseases are the most common – that is to say, if someone has a disease, it is usually a rare disease. This fact is only becoming stronger as our disease categories become more refined. With the rise of personalized medicine, the number of orphan diseases will further increase. Thanks to incentives in this area, new orphan drugs have been brought to market in recent years, about 40% of which are for rare cancers. However, the downside is that many of these drugs are expensive, and even after the patent expires, the price hardly drops because there is insufficient competition (generic preparations or biosimilars). Conversely, for some rare conditions, simple but crucial treatments for the patient are available, where there is sometimes limited incentive to keep these drugs sustainably available. The Netherlands is well positioned to play a pioneering role in this field. A lot of research is being done in our country into disease mechanisms based on new models, (molecular) diagnostics, but also into appropriate use and new ways to arrange early access for patients.

The innovation potential of the Netherlands in the nuclear sector is expanding with opportunities for new nuclear diagnostics and therapy (theranostics), enabling personalized patient treatment. To support therapy development within nuclear medicine, FAST will bring together researchers and entrepreneurs in this sector and provide advice to innovators where needed.

ATMPs are innovative therapies that hold transformative potential for treating a wide range of diseases, particularly those with unmet medical needs. These therapies include gene therapy, somatic cell therapy, and tissue-engineered products, which offer targeted and often curative treatments for complex conditions. While ATMPs have a great promise, getting these treatments to patients remains challenging. The road to market approval involves rigorous and often lengthy regulatory processes due to the novelty of these therapies. Additionally, the scalability and complexity of manufacturing, combined with high development costs, make it difficult to ensure widespread availability of these therapies. Nevertheless, advances in gene editing, regenerative medicine, and biomaterial engineering are accelerating the development of ATMPs, offering hope for groundbreaking treatments in areas like genetic disorders, cancer, and rare diseases. To use the full potential of ATMPs, it is crucial to streamline regulatory pathways, improve access to manufacturing technologies, and encourage collaboration among researchers, industry, and healthcare providers to ensure these therapies can reach patients more efficiently and affordably.