03.23.23 -- Lessons From The Past: How Yesterday's History Can Improve Today's Treatment

Understanding patient and caregiver journeys can help to inform the development of new treatments and services for those living with rare diseases. Discover how designing trials that account for both individual's needs can improve the overall experience of study participation and possibly contribute to improved retention and higher-quality data.

The most fruitful partnerships involve true collaboration — not just information exchange, but mutual learning where patients and researchers can gain insight into each other’s challenges. Learn why there is an increased emphasis on processes and frameworks that encourage sustainable patient partnerships and establish a paradigm of patient-centered research.

To design reliable clinical trials, sponsors must apply thorough knowledge of disease presentation, manifestations, and progression, which may be challenging in rare diseases where this information is minimal. Consider how registry and natural history studies can be very important to the clinical development of therapeutics in rare diseases.

Natural history studies play a crucial role in rare disease drug development, providing insights that advance discovery, shape clinical trial design, and present opportunities to improve patient care. Explore tools for designing and operationalizing natural history studies to support successful rare disease therapeutic development.

Recruitment for rare disease studies has always been difficult, due to a myriad of factors inherent to the low incidence and prevalence of these conditions. Delve into key rare disease recruitment challenges and explore strategies for differentiating trials in today’s competitive landscape.