Overview
This on demand webinar will take approximately 1 hour and 30 minutes to complete.
Rare Diseases 3-Part Webinar Series: On the Road to Approval: The Impact of Patient-Driven Data
Part 3- Evidentiary Standards for Rare Diseases: How Much Data is Enough?
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 3 Overview:
The statutory requirement for FDA to approve a new drug is that it must demonstrate that it is safe and effective. As defined in 1962 in the Food, Drug, and Cosmetics Act, there must be “substantial evidence” of effectiveness from “adequate and well-controlled investigations.” In today’s world of precision medicine, targeting ever smaller patient populations and complex rare diseases where knowledge is continually evolving, assessing what is safe and effective has become increasingly challenging and requires a more dynamic approach.
The third and final webinar in the Rare Disease Webinar Series will:
- Discuss opportunities and challenges in applying flexibility to the kind and quantity of data and information that can be used to meet the statutory standards for drug approval
- Types of data include but are not limited to: smaller studies, natural history studies, and studies using surrogate or intermediate clinical endpoints
- Explore the emerging science of patient perspectives and whether current regulatory processes are optimal for incorporating such input in assessing if a product is “safe and effective”
Rare Diseases Part 1
On Demand
Rare Diseases Part 2
On Demand
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