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  4. Clinical Trials: The Patient Experience - 04/13/2023
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Workshop | Virtual

Event Title
Clinical Trials: The Patient Experience
April 13, 2023

April 13, 2023
11:00 AM - 3:00 PM ET

The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) hosted a public virtual workshop on Thursday, April 13, from 11:00 a.m. – 3:00 p.m. ET.

The workshop, Clinical Trials: The Patient Experience, featured a panel of rare disease patients, caregivers, and advocates who shared their experiences with gene therapy clinical trials. Through participation in clinical trials, patients can help advance the science of gene therapy and further the development of rare disease treatments.

FDA CBER OTP Gene Therapy Clinical Trials Workshop

Event Details

The workshop featured a variety of rare disease patients, caregivers, and advocates to discuss:

  • The experience of finding and participating in gene therapy clinical trials as both a patient and a caregiver
  • The risks and benefits of participating in a gene therapy clinical trial
  • How the FDA works to ensure that gene therapy clinical trials are as safe as possible for patients 

View a recording of the workshop here.

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 Series

This is the third annual patient engagement workshop hosted by FDA CBER OTP. You can access previous workshop materials, including event recordings and transcripts, here:

This workshop was part of an educational series, RegenMedEd: An FDA CBER OTP Series on Regenerative Medicine. The RegenMedEd series aims to bring together important stakeholders and FDA staff to discuss foundational information about regenerative medicine therapies, such as gene therapy and cell therapy products, and explore opportunities for patients, caregivers, and advocates to engage with FDA to help advance drug development. 

Event Materials

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