Rare and Orphan Diseases in Regulated Development
So, you didn’t receive a rare/orphan disease designation as expected. What does that mean? What are your next steps?
During the Food and Drug Administration (FDA) orphan drug designation process, it’s important to maximize your chance for success. Therapies to treat rare/orphan diseases are often not available, and even more importantly, the development and regulatory challenges for these diseases are constantly evolving. Orphan drugs have historically faced a number of barriers, such as limited research and development (R&D) investment due to an expected lack of profitability as well as challenges in clinical trial design and recruitment.
The good news: The Orphan Drug Act’s (ODA) original impetus was to spur drug development to help treat orphan/rare diseases. It has resulted in measurable improvements in the number of drugs approved over the 30 years of the ODA.
In other news: Clinical development challenges and meeting key agency regulatory milestones continue to impede the impetus of bringing life-changing, innovative therapies to market quickly and safely. Understanding FDA expectations is the first step to ensure a successful designation and go-to-market process.
Join the conversation with Sharon Ayd, VP and Principal and Scott Oglesby, Managing Expert at Advarra Regulatory Center of Excellence on Thursday, January 26, 1:00-2:00 pm ET, and explore the latest trends in regulatory and development challenges for rare/orphan diseases.
Learning Objectives:
- Gain an in-depth understanding of regulatory and clinical development challenges for rare/orphan diseases
- Learn successful strategies for a proactive approach to gaining a rare/orphan disease designation
- Identify critical issues and steps to resolve them to achieve regulatory and development milestones
Presented By
Sharon Ayd
VP & Principal
Advarra Consulting – Regulatory Center of Excellence
Scott Oglesby
Managing Expert
Advarra Consulting – Regulatory Center of Excellence
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DATE: June 17, 2021
Time: 1 pm ET | 10 am PT
Duration: 1 hour