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 2- Developing Clinically Meaningful Patient-Focused Outcome Measures
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. The 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 2 Overview:
The development of clinically meaningful patient-focused outcome measures has advanced substantially in recent decades, following the enactment of the FDA’s Orphan Drug Legislation in 1983. However, several challenges remain when designing and implementing meaningful outcome measures, including heterogeneity of outcomes, availability of suitable measures, recruitment, and selection of appropriate data collection methods. The second webinar within the Rare Disease webinar series will focus on clinical outcome measures, biomarker development, and the role of partnerships with patient organizations, methodologists, and investigators in evaluating the impact of disease and treatment. Patient experiences in rare disease clinical trials can be assessed successfully with the utilization of careful planning and precise methods.
Rare Diseases Part 1
On Demand
Rare Diseases Part 3
July 14, 11:00AM-12:30PM ET
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