Rare Disease Poster Tour

Moderator

Lucinda Orsini, MPH, COMPASS Pathways, Skillman, NJ, United States

This tour will take place during Poster Session 2, Poster will be hung from 4:00 - 7:00 PM.

Posters featured in this tour:

PT13: Clinician-Reported Outcomes — A Rare Opportunity for Orphan Labels?

PT14: Do Breakthrough Therapies Demonstrate Breakthrough Value to Payers? An Evaluation of US Hemophilia B Gene Therapy Access Policies

PT15: Assessing Minimal Clinically Important Difference Estimates for the Rett Syndrome Behavior Questionnaire (RSBQ) Using Data From the Trofinetide Clinical Program

PT16: Assessing the Impact of Disparity on Hospital Outcomes: Review of 5 Diseases and Their Unique Challenges

PT17: Challenges for PAIC in Rare Diseases: Results of a Systematic Literature Review

PT18: Prescribing Trends of Disease-Modifying Medications in Texas Medicaid Enrollees With Sickle Cell Disease

Code

056