CTGCT attended the World Orphan Drug Congress (WODC) Europe 2025 in Amsterdam from the 27th to the 29th of October, an event dedicated to strategies and partnerships in the orphan drug industry. There were many panels which focused on the future European strategy for life sciences, regulatory changes, health technology assessment, digitalisation and their implications for rare diseases.
Here are our key takeaways from the congress:
The EU Gap: Research vs. Clinical Practice
A primary concern discussed was the gap between Europe’s research excellence and its translation into clinical practice. Speakers noted that the proportion of clinical studies in Europe has halved and the number of approved cell and gene therapies is significantly lower than in the US.
Discussions also covered challenges from new pharmaceutical legislation and its potential impact on innovation. There was an emphasis on Health Technology Assessment (HTA) and the need for cooperation between Member States to ensure sustainable health ecosystems, particularly given the high cost of new advanced therapies.
Cell/Gene Therapies and Key Bottlenecks
The session on “The Future of Cell and Gene Therapies for Rare Diseases” presented new therapies and approaches to clinical studies for rare diseases, such as adjusting control groups. The importance of using digital technologies for data collection and involving patients and their advocates was also stressed.
It was highlighted that bottlenecks for rare diseases are not just scientific. Significant challenges also include complex and lengthy regulatory procedures, reimbursement issues for approved drugs, and standards for treatment comparison, all of which increase uncertainty for investors. The congress underscored the need for collaboration to bridge the gap between European research and patient access.
In conclusion, the World Orphan Drug Congress 2025 provided a clear overview of the strategic challenges facing the rare disease community, particularly the disconnect between European research and patient access. For CTGCT, the discussions reinforced that overcoming regulatory, reimbursement, and legislative hurdles is just as crucial as solving scientific ones. We remain committed to developing solutions that will bridge this gap and help advance the translation of vital therapies for rare
