How Advanced Therapies Are Changing The Landscape Of Rare Disease

After years of innovative research, advanced therapies are dramatically changing the way patients are treated for a wide variety of diseases – particularly for patients with rare diseases and orphan indications. New developments in advanced therapy medicine products (ATMPs) comprising cell and gene therapy products show the potential to modify or even cure severe chronic conditions for which previously no treatment existed. As a consequence, the dynamic market for innovative therapies such as CAR-T, immuno-oncology, and gene and cell-based therapies grows across the globe. Academic institutes and biotech companies are contributing to these developments both in terms of scientific discovery and undertaking clinical trials aiming for regulatory approvals to treat specific patient populations. While we might be in a golden age of medicine due to advancements in ATMPs, innovative clinical research and development remain crucial for getting treatments to patients.

Join experts from Medpace’s medical, regulatory and operational team with hands-on experience in rare disease and ATMP development as they explore:

• Scientific Advancements: The new and exciting landscape for advanced therapies in rare disease for a variety of indications, with a deeper dive into hematology and ophthalmology
• Regulatory Landscape: Key characteristics and considerations for obtaining orphan drug development (ODD) designation in the US as well as an overview of the comparative differences among current global orphan product laws and regulations
• Operational Considerations: Lessons-learned and best practices for operationalizing rare disease clinical trials including identifying, enrolling and retaining patients, as well as special considerations for ATMPs

Even though many ATMPs are in early development, the potential to cure severe chronic conditions suggest that ATMPs have the potential to reach the market earlier than standard therapies. After so many years of innovative research, we are at the point to develop advanced therapies that bring life-changing treatments to patients with rare diseases.