Suzette James, a BDSRA Foundation Board of Directors member and late-onset CLN2 Batten mother to Maya and Xavier, recently served as a panelist for the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) public virtual workshop titled, “Clinical Trials: The Patient Experience.”

In this Q&A, James recaps her presentation and unveils her advocacy plans moving forward. 

Briefly summarize your presentation and the main points you wanted to make.

Presentation included:

  • Maya & Xavier’s story
  • Experience with clinical trials
  • The need for accelerated approvals
  • Too many pharma and biotech companies are now gone. FDA requirements are too stringent.
  • Parents know their children best and are able to make difficult medical decisions. We are a critical part of the equation.

Click here to watch Suzette’s presentation (begins at 00:58:10). 

How do you believe the presentation was received by those who watched?

“From what I’ve heard, it was received very well. I have heard from many people who were able to attend and listen in (and) some folks that heard about it secondhand. I’ve received some very positive comments about it.”

Overall, how would you describe your experience through this process from preparation to presentation day?

“At times frenetic, focused, rollercoaster emotional, clarifying, enlightening, gobsmacking, cathartic, trauma-inducing all in one!”

What were your main takeaways from the other three panelists? Were there any similarities between their stories to what families in the Batten community experience?

“I think the diagnostic odyssey that everyone goes thru is definitely a similarity we all share – the unknown territory, the challenges of daily life in the midst of a life-altering disease, the pain and heartache of it all – sheer grit and determination….”

Looking ahead, where do you go from here?

“I’ll continue to turn up the volume, continue to tell the Batten story, continue to tell the raw truth… respectfully of course – to serve on the Board of BDSRA and serve the Batten community, focusing on science, research, and fundraising.”