Overview
This webinar will broadcast 11:00AM-12:30PM ET
Rare Diseases 3-Part Webinar Series: On the Road to Approval: The Impact of Patient-Driven Data
Part 1- Conducting Clinical Research in Collaboration with Patient Groups
Series Overview
While Rare Disease Product approvals are held to the same evidentiary standards as common diseases, designing and conducting clinical trials in rare diseases face unique challenges meeting these standards. Patient-focused drug development has become a critical component to overcome these challenges. This three part webinar series illustrates the importance and impact of patient-driven data in rare disease clinical trials with specific examples. The series begins with a discussion focused on the benefits and challenges associated with patient-focused approaches throughout clinical trial design and execution, featuring both industry and patient advocacy group perspectives. Our second installment shifts the focus to defining clinically meaningful endpoints that are relevant to the rare disease patient’s definition of unmet medical need while satisfying regulatory requirements and expectations. Finally, this series concludes with a close look at opportunities for registry data and natural history studies to impact clinical trial design in rare diseases, along with a frank discussion on registry data quality.
Part 1 Overview
Patient engagement in drug development and clinical research is a hot topic, but methods to implement and measure the impact of doing so are still at an early stage. In the setting of rare diseases, patients and patient advocacy groups bring a perspective unique from that of key opinion leaders and regulators with regard to meaningful endpoints, risk-benefit assessment, and burden of clinical study visits or exclusion criteria. Although the risk of bringing yet another perspective to the table might appear to complicate and slow the process, the risks of not doing so may be much higher. Clinical trials in rare diseases can suffer from poor enrollment, irrelevant endpoints, and delayed timelines if the needs of patients are not incorporated into plans throughout the stages of development.
During this webinar, we will share both industry and patient advocacy group perspectives on the benefits and challenges associated with patient-centered approaches throughout clinical trial design and execution. We will include specific examples and case studies to demonstrate opportunities for patient impact on clinical research.
Rare Diseases Part 2
June 9, 11:00AM-12:30PM ET
Rare Diseases Part 3
July 14, 11:00AM-12:30PM ET
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