How Can the FDA Improve Processes for Rare Diseases? – 2023 RARE Drug Development Symposium

In May 2023, Global Genes and the Orphan Disease Center of the University of Pennsylvania hosted the RARE Drug Development Symposium and discussed using new technologies in early stage research. In his keynote, CBER Director Peter Marks discusses strategies that regulatory agencies are applying to accelerate treatment development for rare diseases. 

Peter Marks, MD, PhD
Director FDA’s Center for Biologics Evaluation and Research

Notable quote from session:

We should be applying the science, leveraging every last drop that we can out of that science. The opportunity in rare disease, we understand the disease, we know the gene and the marker, and we should be taking advantage of that. The bank is the biomarker. – Peter Marks

Key Takeaways:

• One way to streamline review and approval of therapies is to focus on platform strategies that leverage the homogeneity of gene therapies using the same backbone vector.

• The rapid and open communication process used during COVID-19 vaccine development could also be used to accelerate rare disease therapy development
• Collaborating with global regulatory colleagues would be mutually beneficial. Currently it’s very difficult and expensive to take a therapy across borders.