The Intersection of Rare Disease and Advanced Therapies
And what it means for clinical development
Eighty percent of rare diseases are known to be of genetic origin, making rare diseases an obvious target for advanced therapies (gene therapy, somatic-cell therapy and tissue-engineered products). However, these complex medicines present challenges over and above the well-documented hurdles of rare-disease research. This white paper explores hard-won lessons learned and best practices in providing safe and life-changing advanced therapies for people with rare diseases as the field evolves.
“These trials are inspiring and – if this is the right word – intimate. The community is so small and the number of patients in the world is so tiny, that there’s a sense of unity for everybody involved: they’re exciting for the science behind them and the potential benefits for these patients and the sites are enthused to participate.”
— Nicholas Vanneman, Clinical Trial Manager, Medpace
This paper discusses:
- Stakeholder interest in advanced therapies
- Added challenges of clinical development of advanced therapies for rare diseases
- Setting up for success in advanced-therapy trials for rare diseases
- Commitment to collaboration
- Relevant and simple endpoints
- Integrate patients into all trial functions
- Partner with regulators early
- Plan for site variability and long follow up
- Adjust for COVID-19
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