There are over 7,000 rare and orphan diseases known to impact approximately 1 in 17 individuals globally, or 50 million in the EU and USA alone.
The development of safe, effective, and accessible therapies against these diseases has been challenged by manufacturing, clinical and regulatory hurdles. Despite these obstacles, increased awareness, greater funding, and new research technologies are driving discoveries in this area. Join this webinar hosted by HealthEconomics.com to learn how ICON and other research groups are working in this space.
ICON presentation on qualitative research in rare disease
Getting to know rare disease patients lived experience: The first step to finding treatment options
For many rare diseases, there is no published data or documentation of patients’ lived experiences with their disease, their unmet needs, or improvements that are important and would be meaningful to them to experience. This information provides a foundation upon which to develop new therapies, and clinical outcomes assessment measures used in clinical trials to assess the efficacy of new therapies for drug approval.
This presentation will include a discussion on some of the key considerations for conducting qualitative research to understand patients’ lived experience with rare disease including:
- Participant recruitment
- Who to collect the data from and what data to collect
- Where to collect the data and collection methods
Speaker:
Patricia Koochaki, PhD
Patricia Koochaki has over 30 years' experience in outcomes research with expertise in qualitative and mixed-methods research. She is particularly interested in understanding rare disease patient journeys and patient burden to use as a foundation for developing fit-for-purpose clinical outcomes assessment measures.