Recap Report: Key Moments from World Orphan Drug Congress

Trend Community
Trend Community

TREND Community was honored to lead several talks at the recent World Orphan Drug Congress USA 2023 on May 23, 2023. Below are some details about the different sessions.

Unlocking the Power of Data for Patient Advocacy – Leveraging Lived Experiences to Empower the Community and Inform Clinical Development and Commercialization

Maria Picone of TREND Community led a patient data session with Cate McCanless (Harmony Biosciences, LLC) and Kristina Davidson (Horizon Therapeutics, plc). They shared case studies that showed how to use data to drive advocacy, achieve better outcomes, and inform clinical development and commercialization efforts. TREND and Harmony Biosciences explained how real-world insights from the Prader-Willi syndrome community showed the need for a drug for excessive daytime sleepiness and increased understanding of the challenges to clinical trial recruitment. Horizon Therapeutics shared the role of TREND’s analysis of the Gout Support Group of America’s community insights to raise awareness of the importance of monitoring uric acid levels and the strong association between proactive management of gout and better mental health outcomes for the community. The power of data is significant to benefit patients across a range of conditions.

Data Collection, Ownership, and Use and Sharing With External Partners

Christopher DeFelice from TREND led a discussion with Michelle Davis (International Fibrodysplasia Ossificans Progressiva Association), Sanath Ramesh (CuresDev), and Sharon Hesterlee (MDA). They talked about data challenges in rare disease research, helping patients get a faster diagnosis, and improving treatments. They shared promising stories of successful partnerships between patient communities, industry, and researchers who are driving scientific progress.

Living With Autoimmune Diseases: Exploring the Shared Experiences of Sjögrens, Arthritis, and Lupus Community Members

Dr. Al Freedman of Rare Counseling and Dr. Max Flurie of TREND led a discussion with Sjögrens, arthritis, and lupus group members. They presented information from social media conversations and discussed the results with the group. Mental health was mentioned in approximately 20% of conversations analyzed on social media. People in the study talked about “pain” and “flare” the most for physical symptoms. “Stress” and “anxiety” were in the top 10 mentions overall. They also talked about fatigue and other sleep issues, how their condition may affect pregnancy, and questions about medications. This means that physical and mental health symptoms were both identified as important considerations to drive further investigations for these communities.

Patient Engagement: Accessibility Isn’t a Checkbox – It is a Form of Patient Empowerment

Rachelle Cook from TREND Community led a discussion on accessibility (inclusion for all people), which included the following important points:

  • Designing for inclusivity benefits everyone—e.g., touch screens, Siri—and it is worth it no matter how many other people benefit
  • There is a medical model of disability, where the person’s limitations are the focus and so the goal is to “fix” those. The social model focuses on modifying a specific environment that in effect is the disabling factor, not the person
  • Person-first language vs identity-first language: what matters most is how the person with a disability represents themselves
  • If you make a mistake in how you interact with someone with a disability, don’t make a big deal out of it—just apologize, learn from it, and move on
  • Case studies in accessibility, with considerations and suggestions for improvement, were shared as a teaching tool

Rachelle ended her talk with changes that can be made at conferences in particular and some readings and websites about accessibility.

For more information about these sessions or about working with TREND, please contact Maria Picone at