5 Key Takeaways

• 1

  Regulatory agencies are adopting risk-based approaches for rare diseases, allowing flexibility in therapy pathways without compromising safety.

• 2

  Manufacturing and GMP expectations remain high, posing challenges for small biotech companies in the evolving landscape of advanced therapies.

• 3

  Quality is defined by understanding the product and process, requiring iterative knowledge building and a focus on critical patient safety factors.

• 4

  Effective quality management involves robust communication and documentation, ensuring transparency between sponsors and CDMOs throughout the process.

• 5

  Quality-by-Design should start early in R&D, emphasizing intentional quality integration rather than testing it into the product at the end.