SIG: Cell and Gene Therapy

We sincerely hope you can join us for this important and timely Rare Disease Week Joint SIG meeting, co‑hosted by the Rare Disease SIG and the Cell and Gene Therapy SIG. In honor of Rare Disease Week, this session "From Outcomes to Experiences: Embedding Patient Perspectives in Real World Evidence for Rare Diseases" will focus on an impactful and increasingly essential topic: how incorporating the patient voice strengthens rare disease pharmacoepidemiology, real‑world evidence, and decision‑making.

 

Through real‑world examples and lived experience, our speakers will highlight how patient engagement can improve study design, interpretation, and relevance. More details below:

 

Date & Time:
 Tuesday, February 24
 11:00 AM – 12:00 PM ET

Format: Virtual | 60‑minute interactive session

 

 Session Overview (60 minutes)

 

Welcome & Opening Remarks (15 min)
Kristen Hahn (Head of Scientific Solutions, PicnicHealth) - Why the patient voice is essential in rare disease pharmacoepidemiology

 

Panel Discussion: Patient Voice in Action (40 min)
An interactive, moderated discussion featuring:

• Kristin Archibald (Founder, NKH Crusaders; patient advocate & rare disease parent; IQVIA)
• Jacose Bell (Head of Global Public Affairs, Rare Blood Disorders, Sanofi)

 

Topics include:

• How patient lived experience improves study design, endpoints, and interpretation
• Avoiding misclassification and missed outcomes in rare disease RWE
• Practical models for incorporating patient voice (advisory boards, co‑design, registries)
• Ethical, feasible ways to engage patients early and meaningfully

 

Closing & Resources (5 min)

 

------------------------------
Kristen Hahn MPH, PhD
PicnicHealth
Old Lyme CT
+1 (860) 575-3411
------------------------------