Session (CTI)

Qualitative research enables the in-depth exploration of the patient or caregiver experience of living with a disease. This is particularly valuable in rare diseases, where little is known about the burden of disease and impact on individuals and caregivers. However, conducting such research can be challenging, due to the small number of potential participants and competing demands on the time of individuals and caregivers. This forum will discuss the value of qualitative research in rare disease, along with solutions for overcoming challenges with recruitment, and strategies for optimizing data collection for multiple stakeholders, including patients and caregivers, regulators, and health technology assessment (HTA) agencies. Sarah Acaster (moderator) will start by introducing the topic and highlighting the value of qualitative research in rare disease populations, and why it is important to optimize data collection for different stakeholders. Paige Nues will provide a patient advocate perspective, describing her experience as a caregiver to a daughter with Rett syndrome, and discussing the value of qualitative research to patients and caregivers. Asia Sikora Kessler will provide an industry perspective, with examples of different qualitative research designs, including standalone and in-trial qualitative interviews. She will highlight how each can provide unique ways of generating data for regulators, as well as solutions of overcoming barriers to implementation. Kate Williams will present results from a novel multi-country qualitative study in aromatic L-amino acid decarboxylase (AADC) deficiency, where qualitative data was optimized for multiple audiences, including to describe the experience of living in different health states associated with an economic model, to support an HTA submission. The session will conclude with a Q&A and a discussion with the audience on their experiences in conducting qualitative research in rare diseases.