Rare disease research is changing.
That was one of the clearest conclusions emerging from IDR 2026, the International Drug Repurposing congress, where researchers, clinicians, patient organisations, industry representatives and regulatory stakeholders gathered to discuss current challenges and future directions in therapeutic research for rare diseases.
While the congress covered multiple scientific and clinical topics, several discussions repeatedly converged around the same broader idea: rare disease research increasingly depends on the ability to connect disciplines, infrastructures and expertise that have traditionally worked separately.
For DREAMS Project, many of these discussions closely reflect the wider context in which the project operates.
DREAMS focuses on rare neuromuscular disorders that share common pathological mechanisms related to autophagy dysfunction and protein disorganisation. The project combines induced pluripotent stem cell models, biological data generation and AI-supported approaches to investigate shared disease mechanisms and explore future therapeutic opportunities.
At IDR 2026, several conversations highlighted why this type of integrated approach is becoming increasingly important across the rare disease research landscape.
Patients are no longer considered external to research
One of the strongest messages throughout the congress was the growing recognition of patients and patient organisations as active contributors to research itself.
In rare diseases, patients often accumulate a deep understanding of disease progression, unmet clinical needs and practical challenges linked to diagnosis, care pathways and treatment expectations. As a result, patient organisations are increasingly involved in shaping research priorities, identifying meaningful outcomes and contributing to the design of future clinical strategies.
This shift reflects a broader transformation across biomedical research. Patient involvement is no longer viewed only as a communication or outreach component. It is progressively becoming part of the research structure itself.
That evolution is particularly relevant in rare diseases, where clinical heterogeneity and small patient populations make real-world experience especially valuable.
Within DREAMS, patient engagement is also integrated into the project structure through dedicated activities linked to future clinical and translational considerations in rare neuromuscular disorders.
At IDR 2026, discussions repeatedly reinforced the idea that meaningful innovation in rare diseases requires not only scientific expertise, but also continuous dialogue with the communities directly affected by these conditions.
Artificial intelligence is becoming part of the research infrastructure
Artificial intelligence was another central topic throughout the congress.
Over the last few years, AI has become increasingly present in biomedical research, especially in areas involving large and complex datasets. Rare disease research is one of those areas.
Researchers working on rare diseases often face fragmented biological information, limited patient cohorts and heterogeneous clinical presentations. AI-based approaches are increasingly being explored as tools that may help researchers identify patterns, prioritise hypotheses and connect molecular pathways more efficiently.
At IDR 2026, many presentations focused on how AI can support the integration of omics, clinical, imaging and scientific literature data in order to facilitate target discovery and therapeutic exploration.
However, the tone of the discussions was notably pragmatic.
The congress did not present AI as a replacement for biology or clinical expertise. Instead, AI was consistently discussed as a support tool that may help researchers navigate scientific complexity more effectively while still requiring robust experimental validation.
This distinction is important.
In practice, one of the major challenges in rare disease research is not simply generating data, but understanding how to connect different layers of information in a meaningful and interpretable way.
That challenge is also highly relevant for projects such as DREAMS, where computational approaches and experimental disease models are developed together rather than separately.
Rare disease innovation depends on collaboration
Another recurring theme at the congress was the need for stronger collaboration across the rare disease ecosystem.
Scientific discovery alone is rarely sufficient to move therapeutic research forward. Translational progress also depends on clinical expertise, regulatory dialogue, infrastructure availability, patient engagement and long-term coordination between institutions.
This challenge becomes even more visible in rare diseases, where patient populations are often geographically dispersed and scientific resources remain fragmented.
At IDR 2026, speakers repeatedly emphasised the importance of building more connected research environments capable of linking academia, hospitals, industry, patient organisations and regulatory stakeholders.
The growing interest in international data-sharing initiatives and collaborative infrastructures reflects this wider shift.
For rare disease research, collaboration is increasingly becoming a practical necessity rather than an optional advantage.
This broader movement is also reflected in DREAMS through the combination of biological research, AI-supported methodologies, translational planning and stakeholder engagement activities developed across the consortium.
Clinical trial design is also evolving
Clinical development was another important area of discussion throughout the congress.
Traditional clinical trial models can be difficult to apply in rare diseases because of small patient populations, heterogeneous disease progression and limited natural history data.
For this reason, adaptive trial designs, real-world evidence approaches and digitally supported outcome measures are receiving growing attention across the field.
At IDR 2026, discussions explored how more flexible and patient-informed methodologies could help improve future rare disease clinical research pathways.
The objective is not necessarily to simplify clinical development, but to make it more realistic and more compatible with the structural realities of rare diseases.
These discussions are increasingly important across the European rare disease ecosystem, where researchers and stakeholders continue searching for more effective ways to connect early-stage discovery with future clinical translation.
Looking ahead
One of the clearest conclusions from IDR 2026 was that rare disease research is moving towards more integrated models of innovation.
Biology, computational analysis, patient engagement and translational planning are no longer being treated as isolated dimensions of research. Increasingly, they are being developed together.
Many challenges remain, from regulatory complexity to fragmented infrastructures and limited patient populations. But the conversations at IDR 2026 showed a growing consensus around one idea: progress in rare diseases will depend on building research ecosystems that are more collaborative, more connected and better equipped to manage complexity.
For projects such as DREAMS, these discussions are highly relevant because they reflect the wider scientific and translational environment in which future rare disease research is evolving.
