IRDiRC is proud to announce the publication of the latest article in Therapeutic Advances in Rare Disease: “From Roadmap to a Sustainable End-to-End Individualized Therapy Pathway.”
This publication builds on the foundational work of the IRDiRC N-of-1 Task Force, expanding from the initial roadmap to explore how N-of-1 therapies—custom treatments for individuals with ultra-rare genetic conditions—can evolve from experimental efforts into a scalable, sustainable healthcare model.
Key themes:
- Bridging the gap between one-off experimental treatments and broader implementation
- Prioritizing patient safety, efficacy, and clear regulatory pathways
- Establishing registries, data-sharing platforms, and systems for long-term monitoring
- Partnering with patient communities and developing new reimbursement frameworks
- Addressing ethical considerations, promoting education, and fostering cross-sector collaboration
This work envisions a future where individualized therapies become a practical, integrated option within the healthcare system—not just rare exceptions.
Read the full article here: https://journals.sagepub.com/doi/10.1177/26330040251339204