CTSI Special Populations: Current Events

Asian American, Native Hawaiian, and Pacific Islander Heritage Month, 2023

 

A Conversation About Health Equity and Community-Based Research 

This May, as we celebrate Asian American, Native Hawaiian, and Pacific Islander (AANHPI) Heritage Month, the FDA OMHHE invites you to view our latest Health Equity Lecture Series webinar featuring a conversation with Dr. Frankie Wong, Professor of Health Equity Research at Florida State University. Watch the recording of the webinar here

Register here for the webinar “Inclusion of People with Disabilities in Clinical Research: Accessibility by Design (AbD) in Clinical Research Toolkit” on May 4 from 11 AM – 12 PM ET.

National Academy of Sciences, New Release

2023

Using Population Descriptors in Genetics and Genomics Research: A New Framework for an Evolving Field

Genetic and genomic information has become far more accessible, and research using human genetic data has grown exponentially over the past decade. Genetics and genomics research is now being conducted by a wide range of investigators across disciplines, who often use population descriptors inconsistently and/or inappropriately to capture the complex patterns of continuous human genetic variation.

Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development

NEW VIRTUAL EVENT

Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development

June 7, 2023

1:00 - 5:00 PM ET

&

June 8, 2023

1:00 - 5:00 PM ET

Virtually via Zoom

Register to Attend Virtually

Although rare disease is defined as a condition affecting fewer than 200,000 individuals, collectively they impact an estimated 30 million people in the U.S. Significant unmet treatment needs remain for many of those living with one of the 7,000-10,000 known rare diseases. In order to facilitate rare disease drug development, and as part of a performance goal and requirement related to the FDA User Fee Reauthorization Act of 2022 and the Food and Drug Omnibus Reform Act of 2022, respectively, the U.S. Food and Drug Administration (FDA) has established a pilot program for supporting the development of efficacy endpoints for rare disease treatments. The new Rare Disease Endpoint Advancement (RDEA) Pilot Program offers additional engagement opportunities with the FDA to sponsors of rare disease development programs that meet specific criteria. 

 

On June 7 & 8, 2023, the Duke-Margolis Center for Health Policy, under a cooperative agreement with the FDA, will host an event, "Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development." This event will illustrate challenges and opportunities in rare disease endpoint development, introduce stakeholders to the RDEA Pilot Program, and highlight how RDEA is structured to support sponsors who may encounter challenges with endpoint development.

A recording of this event will be available on the event webpage shortly after the event. Workshop materials will also be added to this webpage as they become available. Please use the link below to learn more about this event and to register.

 

Learn More & Register Here

Contact margolisevents@duke.edu with inquiries regarding this workshop.

Visit our events page to view other upcoming events hosted by the Duke-Robert J. Margolis, MD, Center for Health Policy.

Connect with us!

Facebook Twitter  Youtube  

Special Populations and COVID-19

 

Translational Science Case Study: Determinants of Pain in Sickle Cell Disease

 

Ten Redefined by Hertz Nazaire - sickle cell disease art

Duke CTSI announces the publication of a new case study, "Biopsychosocial Determinants of Pain in Sickle Cell Disease," a research study led by Dr. Mitchell Knisely of the Duke University School of Nursing. 

This case study discusses not only the methods and results of the research, but explores the challenges the researchers faced and the strategies they employed to overcome those challenges. It also clearly demonstrates the benefits to patients and society of this translational science project. 

Here is the Knisely Case Study using the Translational Science Benefits Model. 

Funding Opportunities