On-Demand Webinar

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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Who should watch?

This content applies to sponsors and CROs, growing biopharma and pharma companies - anyone involved in Regulatory, Clinical Development Operations, Quality, Procurement Director, Vendor Management, Outsourcing Manager, Strategic Sourcing, Proposal Manager, VP/Director of Quality, Manager of Quality Data Manager, IT Manager research, Safety monitoring, drug safety, Environmental Health and Safety Officer, Biosafety Officer, Laboratory Safety Officer Clinical Development Manager, Clinical Development Lead, Director of Regulatory Affairs, Clinical Trials Regulatory, Clinical Research, Regulatory Program Supervisor, VP Regulatory Affairs, and Regulatory Scientists.

CE Credits

The Society of Clinical Research Associates (SOCRA - www.SOCRA.org) accepts documentation of candidate participation in continuing education programs for recertification if the program is applicable to clinical research regulations, operations or management, or to the candidate’s clinical research therapeutic area. This program offers 1 hour of CE credit.

If you would like to obtain CE credits for a different credential, please check with your professional organization to determine if our webinars meet their continuing education requirements.

Certificate of Attendance

After watching this webinar in its entirety and completing the webinar survey, you will receive a certificate of attendance. This certificate will be emailed to you within one business day of watching the webinar. Please note that if your organization has other staff members who wish to get a certificate of attendance, each person must register, watch the webinar in its entirety, and complete the webinar survey. Visit our Webinar Help page for more information on certificates of attendance.

DATE: June 17, 2021
Time: 1 pm ET | 10 am PT
Duration: 1 hour

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