InformedDNA’s Coverage Decision When targeting rare genetic diseases for clinical trials or research, patient engagement is critical. In this webinar, a panel of experts address proven and emerging ways to empower a diverse group of patients as critical stakeholders in clinical trials, leading to improved participation and more meaningful outcomes.

When targeting rare genetic diseases for clinical trials or research, patient engagement is critical. In this webinar, a panel of experts address proven and emerging ways to empower a diverse group of patients as critical stakeholders in clinical trials, leading to improved participation and more meaningful outcomes.

There are more than 2,500 gene-based therapies in the biotech pipeline, many of which are targeted for rare diseases. Clinical trials for these emerging therapies typically target very small patient populations, leading to significant challenges in the identification and enrollment of qualified patient participants.

Biotech and pharmaceutical companies routinely reach out to patient advocacy groups, but don’t often know how best to support their efforts, or how to engage a diverse population of patients in the clinical trial development process. In addition, the patient voice is often restricted to those who have the means to travel to academic research centers. Success in rare disease clinical trial enrollment and retention necessitates expanded access, more inclusive outreach strategies, and technology-driven solutions.

Topics addressed in the webinar:

• Finding sufficient patients with rare genetic diseases for clinical trial enrollment
• Effective partnerships between industry and patient foundations
• The use of genetic testing in clinical trials
• Embracing patient burden – listening to the needs of patients and addressing barriers to clinical trial participation
• Diversity and inclusion – economic parity, breaking down geographic and historical ethnic barriers through community physician engagement and telemedicine services
• Embracing the expertise of the rare disease community to help develop meaningful patient reported outcomes for clinical trial design