Medpace is excited to announce the publication of a new whitepaper in collaboration with Xtalks. This authoritative 20-page publication sets up innovators for success with a review of current challenges (including COVID-19), best practices and hands-on wisdom from industry leaders.
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.
The paper discusses stakeholder interests in advanced therapies, added challenges of clinical development of advanced therapies for rare diseases, and setting up for success in advanced-therapy trials for rare diseases. Then dives deeper into:
- 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