Optimizing Global Submissions in the Age of Rapidly Advancing Innovation and Rare Disease

Panel discussion moderated By: Laurie Smaldone MD

At the upcoming Chief Medical Officer Summit 360°, NDA’s CMO/CSO Laurie Smaldone MD will moderate the panel  “The Challenges of Global Development: Optimizing Global Submissions in the Age of Rapidly Advancing Innovation and Rare Disease.”

To kick off the discussion and  explore their thoughts on the subject the panel met (via telephone ) earlier in the year. In this summary we capture the main points raised during the call.


Panel members:

Laurie Smaldone, MD CMO/CSO, NDA Group, 

Matt Ros, Chief Strategy & Business Officer, Epizyme,  

Adrian Senderowicz, MD, SVP, CMO, Constellation Pharmaceuticals,  

Christine Swenson, SVP Regulatory Affairs, Beam Therapeutics, 

Haishan Xiong, PhD, MBA, SVP Business Development, Fountain Medical Development 


The panel discussion highlighted the major challenges in global drug development, in particular country specific differences around the definition of acceptable study endpoints, in healthcare philosophy and standard-of-care, and also the impact of small, but well advocated patient populations.

“Anyone who is in the development space should pay attention to the patient voice” [Matt Ros, Chief Strategy & Business Officer, Epizyme ]

Working in rare diseases comes down to understanding the insights from the patient’s experience as they are the true experts of their condition. If performed properly, the clinical development program becomes meaningful, does not create tremendous burden and will most likely help patients already in the process.

One of the biggest challenges in developing treatments for rare diseases globally is the definition of an acceptable endpoint and path to approval across countries. “Accelerated approval strategies in the United States are very common, whereas conditional approval strategies in Europe are unique and limited, suggesting differences in these regulatory pathways” [Matt Ros, Chief Strategy & Business Officer, Epizyme ]

To make the matter even more complex, global development can impact studies from their initiation. In China, the healthcare trial resources are currently highly concentrated in big cities, but this is opening up, making trial participation more accessible for the patients.

The trial set-up impacts both timing and recruitment and is challenged by the small patient populations and the gold standard of randomized controlled trials (RCT). “In rare diseases there are often situations where you are not going to be able to do that,” [Christine Swenson, SVP Regulatory Affairs, Beam Therapeutics]

Additional complications may include regulatory constraints in the sense that different regulatory bodies may offer different scientific advice or have alternative requirements. How physicians treat, patients may vary, as well as the standard-of-care in different countries. If you don’t plan with that in mind, timelines are much likely to extend, while you wait for approval for registration trials.

To read the full discussion click here

The panelists will continue their discussion at the  Chief Medical Officer Summit 360°, October 26-27 , make sure to be there for additional insights. For more information and full agenda click here.