- March 9, 2022
- 11:00 AM - 12:00 PM ET
- Organized By:
The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) hosted a public webinar on Wednesday, March 9, 2022, from 11:00 a.m. - 12:00 p.m. ET.
The webinar, The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases, brought together patients, caregivers, advocates, and other important stakeholders to celebrate and recognize the contributions that patients with rare diseases have made to advancements in regenerative medicine. This event followed Rare Disease Day, an international day for awareness of rare diseases.
There are around 7,000 known rare diseases affecting more than 30 million people in the United States. Many rare diseases are life-threatening, and only a fraction of rare diseases have an available treatment. The FDA remains committed to supporting the development of treatments for rare diseases and engaging with the rare disease community.
About the Webinar
The webinar featured panelists to discuss:
- Perspectives from patients living with a rare disease and their experiences participating in clinical research
- Ways patients and caregivers can participate in and advance clinical research for rare diseases
- Why and how regenerative medicine may hold promise for patients with rare diseases
- FDA’s efforts and programs to support the development of rare disease treatments, including regenerative medicine like gene and cell therapies
- Debbie Drell, Director of Membership Services, National Organization for Rare Disorders
- Brian O’Mahony, Chief Executive, Irish Haemophilia Society
- Julienne Vaillancourt, Captain, U.S. Public Health Service Commission Corps & Rare Disease Liaison & Policy Advisor, FDA CBER
The webinar was moderated by Dr. Anne Rowzee, Associate Director for Policy, FDA CBER OTAT. This webinar was the second in an educational webinar series called RegenMedEd: An FDA CBER OTAT Webinar Series on Regenerative Medicine.
What is Regenerative Medicine?
Regenerative medicine therapies, such as cell and gene therapies, hold promise for transforming medicine and creating options for patients living with difficult, rare, and often incurable diseases. The FDA plays a vital role in facilitating the development of regenerative medicine therapies, as well as in ensuring approved products are safe and effective for patients.
About the RegenMedEd Webinar Series
To further engage patients, caregivers, and advocates on the topic of regenerative medicine, OTAT launched an educational webinar series called RegenMedEd: An FDA CBER OTAT Webinar Series on Regenerative Medicine. The RegenMedEd webinar series aims to bring together patients, caregivers, patient advocates, and FDA staff to discuss foundational information about regenerative medicine therapies, such as gene therapy and cell therapy, and explore opportunities for stakeholders to engage with the FDA to help advance drug development.
|Slides for The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases||pdf (4.81 MB)|
|Transcript for The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases||pdf (202.59 KB)|